- Brain Repair & Rehabilitation
Short Title Full Title PI Phase Type INSPIRES-2 Improving sleep and learning in rehabilitation after stroke, part 2. Ajina, Sara Not Applicable Non-commercial non-portfolio ECST 2: The 2nd European Carotid Surgery Trial ECST 2: The 2nd European Carotid Surgery Trial Brown, Prof Martin Not Applicable Non-commercial portfolio CONVINCE - Protocol Version 2.1 29th November 2016 CONVINCE - COLchicine for preventioN of Vascular Inflammation in Non-CardioEmbolic stroke) - a randomised clinical trial of low-dose colchicine for secondary prevention after stroke. Chandratheva, Dr Arvind Phase III Non-commercial portfolio Protocol 18-513 Andexanet ICH A PHASE 4 RANDOMIZED CLINICAL TRIAL OF ANDEXANET ALFA [ANDEXANET ALFA FOR INJECTION] IN ACUTE INTRACRANIAL HEMORRHAGE IN PATIENTS RECEIVING AN ORAL FACTOR XA INHIBITOR Chandratheva, Dr Arvind Phase IV Commercial portfolio Mechanisms underlying spoken language production Mechanisms underlying spoken language production: facilitating frontal brain networks following aphasic stroke Crinion, Dr Jenny Not Applicable Non-commercial non-portfolio Natural history and biomarkers in autonomic disorders Natural history and biomarkers in autonomic disorders Iodice, Dr Valeria Not Applicable Non-commercial non-portfolio ATTEND ATTEND: Attentional Therapy for the TrEatment of Neglect Disorder (ATTEND). Leff, Prof Alexander Not Applicable Non-commercial portfolio PLORAS version 1 Predicting Language Outcome and Recovery After Stroke Leff, Prof Alexander Not Applicable Non-commercial portfolio Neural correlates of visual consciousness The neural correlates of conscious visual experience in normal and brain damaged human subjects Leff, Prof Alexander Not Applicable Non-commercial non-portfolio Digital Interventions in Neuro-Rehabilitation (DINR) Digital Interventions in Neuro-Rehabilitation: Two digital neuro interventions (DNIs)for word retrieval. The development and testing of two web-based therapy applications for people with naming difficulties caused by Stroke (iTALKbetter) or Mild-Moderate Dementia (Gotcha!) Leff, Prof Alexander Study Non-commercial non-portfolio BPL-PSILO An Exploratory Open-Label, Phase 1b, Ascending Dose Study to Evaluate the Effects of Oral 3 [2 (dimethylamino)ethyl]-1h-indol-4-yl (Psilocybin, BPL PSILO) on Cognition in Patients With Chronic Short-Lasting Unilateral Neuralgiform Headache Attacks (SUNHA) Matharu, Dr Manjit Phase Ib Commercial portfolio Headache and facial pain: a prospective clinical study Primary and secondary headaches and facial pain disorders: a prospective clinical study Matharu, Dr Manjit Not Applicable Non-commercial non-portfolio BASIC Study BASIC - Botulinum toxin type A blockade of the sphenopalatine ganglion in treatment-refractory chronic cluster headache Matharu, Dr Manjit Phase III Non-commercial portfolio Craniocervical imaging in joint hypermobility disorders (version 1) Headache disorders and imaging of the craniocervical junction in joint hypermobility disorders Matharu, Dr Manjit Not Applicable Non-commercial portfolio Cytokines in chronic headache disorders Cytokines and inflammatory biomarkers in chronic headache and facial pain disorders Matharu, Dr Manjit Not Applicable Non-commercial non-portfolio Lesion Function Mapping High-Dimensional Lesion-Function Mapping of the Human Brain Nachev, Dr Parashkev Study Non-commercial non-portfolio Neurophysiology evaluation of symptomatic sacral Tarlov cysts A comprehensive evaluation of the effects of sacral Tarlov cysts on pelvic organ functions and neurophysiological parameters of the pelvic floor Panicker, Dr Jalesh Not Applicable Non-commercial non-portfolio MultiStem® Administration for Stroke Treatment and Enhanced Recovery MultiStem® Administration for Stroke Treatment and Enhanced Recovery Study (MASTERS-2) Perry, Dr Richard Phase III Commercial portfolio Study Exploring The Impact of COVID-19 On Stroke (SETICOS) Study Exploring The Impact of COVID-19 On Stroke (SETICOS) Perry, Dr Richard Not Applicable Non-commercial portfolio ATTEST 2 Alteplase-Tenecteplase Trial Evaluation for Stroke Thrombolysis (ATTEST 2) Perry, Dr Richard Phase III Non-commercial portfolio Validation Of Bioarray In Stroke Validation Of Bioarray In Stroke (VOBIS). Perry, Dr Richard Not Applicable Commercial portfolio Mechanisms underlying limb apraxia Understanding motor processing pathways to guide therapy for limb apraxia Perry, Dr Richard Not Applicable Non-commercial portfolio 252LH301: Efficacy & Safety of IV BIIB093 (The CHARM Study) Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous BIIB093 (Glibenclamide) for Severe Cerebral Edema following Large Hemispheric Infarction Perry, Dr Richard Phase III Commercial portfolio PERCIVAL Periodontitis, COVID-19 and Cerebrovascular Disease Simister, Dr Robert Not Applicable Non-commercial portfolio Prehospital triage for stroke patients (PHOTONIC) (v1.0) PreHOspital Triage for potential stroke patients: lessONs from systems Implemented in response to COVID19 (PHOTONIC) Simister, Dr Robert Not Applicable Non-commercial portfolio TEMPO-2 Multicentre, prospective randomized open label, blinded-endpoint (PROBE) controlled trial of thrombolysis with low dose Tenecteplase (TNK-tPA) versus standard of care in the prevention of disability at 3 months in minor ischemic stroke with proven acute symptomatic occlusion Simister, Dr Robert Phase III Non-commercial portfolio prophylaxis of post-stroke epilepsy prevention of epilepsy in stroke patients at high risk of developing unprovoked seizures: anti-epileptogenic effects of eslicarbazepine acetate Simister, Dr Robert Phase II Commercial portfolio Biomarkers of early stroke recovery (BEST) study. Version 2. Biomarkers of early stroke recovery (BEST) Ward, Prof Nick Not Applicable Non-commercial portfolio INTENSIVE Is intensive upper limb rehabilitation effective in chronic stroke patients? A randomised controlled trial Ward, Prof Nick Not Applicable Non-commercial portfolio TRACS study Tracking Reticulospinal And Corticospinal pathways after Stroke TRACS study Ward, Prof Nick Not Applicable Non-commercial non-portfolio tDCS in post-stroke fatigue (TIPS) Does diminished motor cortical excitability cause post-stroke fatigue? Ward, Prof Nick Not Applicable Non-commercial portfolio MOtor Control After Stroke (MOCAS) Does the upper limb rehabilitation programme lead to a general improvement in motor control processes in chronic stroke patients? Ward, Prof Nick Not Applicable Academic/student STROKE ODYSSEYS Version 1 Evaluation of an interdisciplinary performance arts intervention that combines movement and voice to reduce anxiety
and depression among those living with the effects of a stroke.Ward, Prof Nick Not Applicable Non-commercial non-portfolio Re-opening the Critical Period after Stroke (ReCAPS). Version 1.0 Re-opening the critical period for plasticity after stroke with dose-controlled non-invasive brain stimulation Ward, Prof Nick Not Applicable Non-commercial portfolio TICH-3 Tranexamic acid for Hyperacute Spontaneous Intracerebral Haemorrhage (TICH-3) Werring, Dr David Phase III Non-commercial portfolio ENRICH-AF: Edoxaban for IntraCranial Haemorrhage survivors with AF EdoxabaN foR IntraCranial Haemorrhage survivors with atrial fibrillation (ENRICH-AF) Werring, Dr David Phase III Non-commercial portfolio PROHIBIT-ICH Prevention Of Hypertensive Injury to the Brain by Intensive Treatment after Intracerebral Haemorrhage: a pilot randomised trial of home telemetry-guided treatment Werring, Dr David Not Applicable Non-commercial portfolio Genetic risk factors for cerebral small vessel disease Genetic risk factors for cerebral small vessel disease Werring, Dr David Not Applicable Non-commercial portfolio Familial Cerebral Small Vessel Disease A UK Familial Cerebral Small Vessel Disease Study Werring, Dr David Not Applicable Non-commercial portfolio Tenecteplase in Wake-up Ischaemic Stroke Trial Tenecteplase in Wake-up Ischaemic Stroke Trial (TWIST) Werring, Dr David Phase III Non-commercial portfolio Prediction of stroke outcome using brain imaging machine-learning Optimizing prediction of stroke outcome using brain imaging machine-learning Werring, Dr David Not Applicable Non-commercial portfolio OPTIMAS Trial OPtimal TIMing of Anticoagulation after acute ischaemic Stroke: a randomised controlled trial (OPTIMAS Trial) Werring, Dr David Not Applicable Non-commercial portfolio R4VaD Rates, Risks and Routes to Reduce Vascular Dementia (R4VaD) Werring, Dr David Not Applicable Non-commercial portfolio
- Imaging Neuroscience
Short title Long title PI Phase Type Development of novel MRI techniques for neurological applications Development of novel MRI techniques for neurological applications Wheeler-Kingshott, Prof Claudia Not Applicable Non-commercial portfolio MODEL-COV Advanced Modeling Of Magnetic Resonance Imaging Data To Study The
Biophysical Underpinning Of NEurological Symptoms Of Long-COVIDWheeler-Kingshott, Prof Claudia Not Applicable Non-commercial portfolio
- Neurodegenerative Disease
Short title Long title PI Phase Type Memory, language and complex thinking skills Memory, language and complex thinking skills in people with and without neurological conditions Cipolotti, Prof Lisa Not Applicable Non-commercial non-portfolio Cognitive dysfunction in survivors of childhood medulloblastoma Neuroanatomical correlates of cognitive dysfunction in short- and long-term survivors of childhood medulloblastoma Clark, Chris Not Applicable Non-commercial portfolio Neuropsychology of posterior cortical atrophy version1 Neuropsychological investigation of visuospatial and literacy skills in posterior cortical atrophy Crutch, Prof Sebastian Not Applicable Non-commercial non-portfolio DIAN Dominantly Inherited Alzheimer’s network Fox, Prof Nick Not Applicable Non-commercial portfolio A novel cell model for the dementias Induced pluripotent stem cells derived from patients with familial Alzheimer's disease and other dementias as novel cell models for neurodegeneration Fox, Prof Nick Not Applicable Non-commercial portfolio Longitudinal research into Alzheimer’s disease and other dementias Longitudinal research into Alzheimer’s disease and other dementias Fox, Prof Nick Not Applicable Non-commercial non-portfolio Normal Pressure Hydrocephalus SILK Stable Isotope Labeling Kinetics in Normal Pressure Hydrocephalus: implications for neurodegeneration Fox, Prof Nick Not Applicable Non-commercial portfolio DREAMS START (Dementia RElAted Manual for Sleep) RCT A parallel multi-centre randomised controlled trial to determine the clinical and cost-effectiveness of DREAMS START (Dementia RElAted Manual for Sleep; STrAtegies for RelaTives)for people living with dementia and their carers Fox, Prof Nick Not Applicable Non-commercial portfolio ADDID ADDID: ADDitonal csf In Dementia Fox, Prof Nick Study Non-commercial non-portfolio hMRI in Alzheimer’s Disease Non-Invasive In-Vivo Histology in Alzheimer’s disease Using Magnetic Resonance Imaging (MRI) Fox, Prof Nick Not Applicable Non-commercial portfolio Tau targeted vaccines in patients with early Alzheimer’s Disease A Phase Ib/IIa Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Immunogenicity of Different Doses, Regimens and Combinations of Tau Targeted Vaccines in Subjects with Early Alzheimer’s Disease Mummery, Dr Catherine Phase I/II Commercial portfolio Phase 3 Study of AL001 in Patients with or at risk of FTD A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene Mummery, Dr Catherine Phase III Commercial portfolio 221AD304 - Phase 3 Study of Aducanumab in Early Alzheimer’s Disease Phase 3b Open-Label, Multicenter, Safety Study of BIIB037 (aducanumab) in Subjects with Alzheimer’s disease Who Had Previously Participated in the Aducanumab Studies 221AD103, 221AD301, 221AD302 and 221AD205. Mummery, Dr Catherine Phase III Commercial portfolio DIANTU001:Ph. II/III Randomized, Double-blind Placebo in Alzheimer's A Phase II/III Randomized, Double-Blind,Placebo-Controlled Multi-Center Study of 2 Potential Disease Modifying Therapies in Individuals at Risk for and with Dominantly Inherited Alzheimer’s Disease Mummery, Dr Catherine Phase II/III Non-commercial portfolio AL002 In Participants With Early Alzheimer's Disease A Phase 2 Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of AL002 in Participants with Early Alzheimer’s Disease Mummery, Dr Catherine Phase II Commercial portfolio Study E2814-G000-103 An Open-Label Phase 1b/2 Study to Assess Safety and Target Engagement of E2814 in Subjects with Mild to Moderate Cognitive Impairment due to Dominantly Inherited Alzheimer’s Disease Mummery, Dr Catherine Phase I/II Commercial portfolio Biomarkers and Rapid Imaging in Dementia Diagnosis: B-RAPIDD Biomarkers and Rapid Imaging in Dementia Diagnosis: B-RAPIDD Mummery, Dr Catherine Not Applicable Non-commercial portfolio ALN-APP-001 A Study to Evaluate the Safety and Tolerability of ALN-APP in
Patients with EOADMummery, Dr Catherine Phase I Commercial portfolio IBC-01-01 A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of IBC-Ab002 in Persons with Early Alzheimer's Disease Mummery, Dr Catherine Phase I Commercial portfolio CONFLuid study Healthy control biofluid study. Paterson, Dr Ross Not Applicable Non-commercial non-portfolio IRAS 276517 A Phase 1/2 Ascending Dose Study to Evaluate the Safety A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on
Progranulin Levels of PR006A in Patients with Fronto-Temporal Dementia
with Progranulin Mutations (FTD-GRN)Rhorer , Dr Jonathan Phase I/II Commercial portfolio GENFI Mindstrong study Genetic Frontotemporal Dementia Initiative Mindstrong study Rohrer, Dr Jonathan Not Applicable Non-commercial non-portfolio Genetic Frontotemporal dementia Initiative (GENFI) Genetic Frontotemporal dementia Initiative (GENFI) Rohrer, Dr Jonathan Not Applicable Non-commercial portfolio Depletion of Serum Amyloid P Component in Alzheimer's Disease (v1.0) DEpletion of Serum amyloid P component In Alzheimer’s Disease: DESPIAD. Double-blind placebo controlled randomised phase IIb trial of SAP depletion by CPHPC in mild Alzheimer’s disease. Rossor, Prof Martin Phase II Non-commercial portfolio Bedside Assessment of Eye Movements in the Cognitively Impaired V1.1 Comparison of Bedside Assessment of Eye Movements using Facial and Non-facial Stimuli in the Cognitively Rossor, Prof Martin Not Applicable Non-commercial non-portfolio Shared decision aid nutrition/hydration in dementia in acute hospitals Developing a shared decision aid regarding nutrition and hydration for people with severe dementia in acute hospitals Sampson, Dr Elizabeth Not Applicable Non-commercial non-portfolio Shared decision aid nutrition/hydration in dementia in acute hospitals Developing a shared decision aid regarding nutrition and hydration for people with severe dementia in acute hospitals Sampson, Dr Elizabeth Not Applicable Non-commercial non-portfolio Multi-centre MRI study of Post Mortem proven cases (Research Database) Multi-centre MRI study of Post Mortem proven cases (Research Database) Schott, Dr Jonathan Not Applicable CSF collection in patients with neurodegenerative diseases Cerebrospinal fluid collection in patients with neurodegenerative diseases Schott, Dr Jonathan Not Applicable Non-commercial portfolio Biomarkers and genetics in cognitive disorders Biomarkers and genetics in cognitive disorders Schott, Dr Jonathan Not Applicable Non-commercial portfolio MRC National Survey of Health and Development Neuroimaging substudy MRC National Survey of Health and Development Neuroimaging substudy Schott, Dr Jonathan Not Applicable Non-commercial portfolio Brain signature of auditory information processing Brain signature of auditory information processing in the degenerative dementias Warren, Dr Jason Not Applicable Non-commercial portfolio Cognitive outcomes in Phenylketonuria across ages Cognitive assessment of Phenylketonuria: Comparing outcomes across developmental ages Aitkenhead, Lynne Not Applicable Non-commercial portfolio Pompe Registry Pompe Registry; ongoing, observational database tracking the natural history and outcomes of patients with pompe disease Lachmann, Dr Robin Not Applicable Commercial non-portfolio MRS I Registry online Feb 2006 MRS I Registry; ongoing, observational database tracking natural history and outcomes of patients with MPS I Lachmann, Dr Robin Not Applicable Commercial non-portfolio Gaucher Registry, online feb 2006 Gaucher Registry, online feb 2006 Lachmann, Dr Robin Not Applicable Commercial non-portfolio Rare and Undiagnosed Diseases Study (RUDY) Rare and Undiagnosed Diseases Study (RUDY) Lachmann, Dr Robin Not Applicable Non-commercial portfolio Open Label Extension study (OLE) of the Modify study A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease Lachmann, Dr Robin Phase III Commercial portfolio Cognitive outcomes in Phenylketonuria when returning to diet Assessing possible cognitive, emotional and quality of life benefits of resuming a diet in adults with PKU Lachmann, Dr Robin Not Applicable Non-commercial portfolio Gaucherite- A study to classify Gaucher disease Predictive measures to stratify clinical outcomes in children and adults with Gaucher disease and responses to specific therapies Lachmann, Dr Robin Not Applicable Non-commercial portfolio INPDR International Niemann-Pick Disease Registry Lachmann, Dr Robin Not Applicable Non-commercial portfolio Phase 2 study of ARCT-810 in Participants with OTC Deficiency Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants with Ornithine Transcarbamylase Deficiency Lachmann, Dr Robin Phase II Commercial portfolio CHIL 5604 Arimoclomol prospective double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C Lachmann, Dr Robin Phase II/III Commercial portfolio XLH Registry An international, multicentre, prospective, non-interventional observational registry for patients with X-linked hypophosphatemia (XLH) Murphy, Dr Elaine Not Applicable Commercial portfolio E IMD European registry and network for intoxication type metabolic diseases Murphy, Dr Elaine Not Applicable Non-commercial non-portfolio Homocystinuria v1.0 A Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine β-Synthase (CBS) Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of ACN00177 Murphy, Dr Elaine Phase I/II Commercial portfolio CHIL3751 (110-504) Morquio A Registry Study Murphy, Dr Elaine Not Applicable Commercial portfolio RAPSODI GD Remote assessment of Parkinsonism supporting ongoing development of interventions in Gaucher's disease Murphy, Dr Elaine Not Applicable Non-commercial portfolio Longitudinal Physiological Changes in Inherited Metabolic Disorders Longitudinal study of aerobic capacity and skeletal muscle characteristics in patients with rare inherited metabolic disorders (IMD) Murphy, Dr Elaine Not Applicable Non-commercial non-portfolio LALDR An Observational Disease and Clinical Outcomes Registry of Patients with Lysosomal Acid Lipase (LAL) Deficiency Murphy, Dr Elaine Not Applicable Commercial portfolio GalNet Registry European Galactosemia Network Patient Registry (GalNET) (Version 1) Murphy, Dr Elaine Non-commercial non-portfolio Development of methods for diagnosis of neurometabolic disorders Metabolomic profiling of cerebrospinal fluid to improve diagnosis and treatment monitoring of patients with inborn or acquired errors of metabolism Pope, Dr Simon Memory, language and complex thinking skills Memory, language and complex thinking skills in people with and without neurological conditions Cipolotti, Prof Lisa Not Applicable Non-commercial non-portfolio Cognitive dysfunction in survivors of childhood medulloblastoma Neuroanatomical correlates of cognitive dysfunction in short- and long-term survivors of childhood medulloblastoma Clark, Chris Not Applicable Non-commercial portfolio Neuropsychology of posterior cortical atrophy version1 Neuropsychological investigation of visuospatial and literacy skills in posterior cortical atrophy Crutch, Prof Sebastian Not Applicable Non-commercial non-portfolio DIAN Dominantly Inherited Alzheimer’s network Fox, Prof Nick Not Applicable Non-commercial portfolio A novel cell model for the dementias Induced pluripotent stem cells derived from patients with familial Alzheimer's disease and other dementias as novel cell models for neurodegeneration Fox, Prof Nick Not Applicable Non-commercial portfolio Longitudinal research into Alzheimer’s disease and other dementias Longitudinal research into Alzheimer’s disease and other dementias Fox, Prof Nick Not Applicable Non-commercial non-portfolio Normal Pressure Hydrocephalus SILK Stable Isotope Labeling Kinetics in Normal Pressure Hydrocephalus: implications for neurodegeneration Fox, Prof Nick Not Applicable Non-commercial portfolio DREAMS START (Dementia RElAted Manual for Sleep) RCT A parallel multi-centre randomised controlled trial to determine the clinical and cost-effectiveness of DREAMS START (Dementia RElAted Manual for Sleep; STrAtegies for RelaTives)for people living with dementia and their carers Fox, Prof Nick Not Applicable Non-commercial portfolio ADDID ADDID: ADDitonal csf In Dementia Fox, Prof Nick Study Non-commercial non-portfolio hMRI in Alzheimer’s Disease Non-Invasive In-Vivo Histology in Alzheimer’s disease Using Magnetic Resonance Imaging (MRI) Fox, Prof Nick Not Applicable Non-commercial portfolio Tau targeted vaccines in patients with early Alzheimer’s Disease A Phase Ib/IIa Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Immunogenicity of Different Doses, Regimens and Combinations of Tau Targeted Vaccines in Subjects with Early Alzheimer’s Disease Mummery, Dr Catherine Phase I/II Commercial portfolio Phase 3 Study of AL001 in Patients with or at risk of FTD A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene Mummery, Dr Catherine Phase III Commercial portfolio 221AD304 - Phase 3 Study of Aducanumab in Early Alzheimer’s Disease Phase 3b Open-Label, Multicenter, Safety Study of BIIB037 (aducanumab) in Subjects with Alzheimer’s disease Who Had Previously Participated in the Aducanumab Studies 221AD103, 221AD301, 221AD302 and 221AD205. Mummery, Dr Catherine Phase III Commercial portfolio DIANTU001:Ph. II/III Randomized, Double-blind Placebo in Alzheimer's A Phase II/III Randomized, Double-Blind,Placebo-Controlled Multi-Center Study of 2 Potential Disease Modifying Therapies in Individuals at Risk for and with Dominantly Inherited Alzheimer’s Disease Mummery, Dr Catherine Phase II/III Non-commercial portfolio AL002 In Participants With Early Alzheimer's Disease A Phase 2 Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of AL002 in Participants with Early Alzheimer’s Disease Mummery, Dr Catherine Phase II Commercial portfolio Study E2814-G000-103 An Open-Label Phase 1b/2 Study to Assess Safety and Target Engagement of E2814 in Subjects with Mild to Moderate Cognitive Impairment due to Dominantly Inherited Alzheimer’s Disease Mummery, Dr Catherine Phase I/II Commercial portfolio Biomarkers and Rapid Imaging in Dementia Diagnosis: B-RAPIDD Biomarkers and Rapid Imaging in Dementia Diagnosis: B-RAPIDD Mummery, Dr Catherine Not Applicable Non-commercial portfolio ALN-APP-001 A Study to Evaluate the Safety and Tolerability of ALN-APP in
Patients with EOADMummery, Dr Catherine Phase I Commercial portfolio IBC-01-01 A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of IBC-Ab002 in Persons with Early Alzheimer's Disease Mummery, Dr Catherine Phase I Commercial portfolio CONFLuid study Healthy control biofluid study. Paterson, Dr Ross Not Applicable Non-commercial non-portfolio IRAS 276517 A Phase 1/2 Ascending Dose Study to Evaluate the Safety A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on
Progranulin Levels of PR006A in Patients with Fronto-Temporal Dementia
with Progranulin Mutations (FTD-GRN)Rhorer , Dr Jonathan Phase I/II Commercial portfolio GENFI Mindstrong study Genetic Frontotemporal Dementia Initiative Mindstrong study Rohrer, Dr Jonathan Not Applicable Non-commercial non-portfolio Genetic Frontotemporal dementia Initiative (GENFI) Genetic Frontotemporal dementia Initiative (GENFI) Rohrer, Dr Jonathan Not Applicable Non-commercial portfolio Depletion of Serum Amyloid P Component in Alzheimer's Disease (v1.0) DEpletion of Serum amyloid P component In Alzheimer’s Disease: DESPIAD. Double-blind placebo controlled randomised phase IIb trial of SAP depletion by CPHPC in mild Alzheimer’s disease. Rossor, Prof Martin Phase II Non-commercial portfolio Bedside Assessment of Eye Movements in the Cognitively Impaired V1.1 Comparison of Bedside Assessment of Eye Movements using Facial and Non-facial Stimuli in the Cognitively Rossor, Prof Martin Not Applicable Non-commercial non-portfolio Shared decision aid nutrition/hydration in dementia in acute hospitals Developing a shared decision aid regarding nutrition and hydration for people with severe dementia in acute hospitals Sampson, Dr Elizabeth Not Applicable Non-commercial non-portfolio Shared decision aid nutrition/hydration in dementia in acute hospitals Developing a shared decision aid regarding nutrition and hydration for people with severe dementia in acute hospitals Sampson, Dr Elizabeth Not Applicable Non-commercial non-portfolio Multi-centre MRI study of Post Mortem proven cases (Research Database) Multi-centre MRI study of Post Mortem proven cases (Research Database) Schott, Dr Jonathan Not Applicable CSF collection in patients with neurodegenerative diseases Cerebrospinal fluid collection in patients with neurodegenerative diseases Schott, Dr Jonathan Not Applicable Non-commercial portfolio Biomarkers and genetics in cognitive disorders Biomarkers and genetics in cognitive disorders Schott, Dr Jonathan Not Applicable Non-commercial portfolio MRC National Survey of Health and Development Neuroimaging substudy MRC National Survey of Health and Development Neuroimaging substudy Schott, Dr Jonathan Not Applicable Non-commercial portfolio Brain signature of auditory information processing Brain signature of auditory information processing in the degenerative dementias Warren, Dr Jason Not Applicable Non-commercial portfolio Genomic and expression profiling of brain tumours Genomic and expression profiling of brain tumours Brandner, Prof Sebastian Not Applicable Non-commercial non-portfolio HD Gene TRX2
Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease
Tabrizi, Prof Sarah/ Wild, Prof Edward
Phase I/II
Commercial non-portfolio
PIVOT-HD
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease
Tabrizi, Prof Sarah
Phase II
Commercial non-portfolio
Generation HD2
A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease.
Tabrizi, Prof Sarah
Phase II
Commercial non-portfolio
Enroll-HD
A Prospective Registry Study in Global Huntington’s Disease Cohort
Tabrizi, Prof Sarah
Not applicable
Non-Commercial portfolio
HD Clarity
A Multi-site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington’s Disease
Wild, Prof Edward
Not Applicable
Non-Commercial portfolio
HD-YAS 2.0
Huntington’s Disease Young Adult Study 2.0
Tabrizi, Prof Sarah
Not Applicable
Non-Commercial portfolio
- Neuromuscular Diseases
Short title Long title PI Phase Type Ultrasound to detect vocal fold movement in neurological disease A feasibility study to investigate the accuracy and viability of ultrasound in detecting impairments of vocal fold mobility in people with neurological disease Allen, Dr Jodi Not Applicable Non-commercial non-portfolio Skeletal Muscle Dysfunction in Aortic Stenosis Investigating Skeletal Muscle Dysfunction in Aortic Stenosis Astin, Dr Ronan Not Applicable Non-commercial non-portfolio ReSolve v.1.1 Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD Bugiardini, Dr Enrico Not Applicable Non-commercial non-portfolio 1821-FSH-301 A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled,
48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod
in Treating Patients with Facioscapulohumeral Muscular DystrophyBugiardini, Dr Enrico Phase III Commercial portfolio ADHERE A Phase 2 Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Carr, Dr Aisling Phase II Commercial portfolio ARDA A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, Multicenter Trial to Evaluate the Safety and Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 2 Dose Regimens of ARGX-117 in Adults With Multifocal Motor Neuropathy Carr, Dr Aisling Phase II Commercial portfolio SORD A RandomIzed, Double-Blind, Placebo-CoNtrolled, Two-Part Study to Evaluate the Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients with SoRbitol Dehydrogenase (SORD) Deficiency. Cortese, Dr Andrea Commercial portfolio SBMA Natural history study Natural history study of Spinal and Bulbar Muscular Atrophy Fratta, Dr Pietro Not Applicable Non-commercial non-portfolio MND GENETICS Next Generation Genetic Screening in Motor Neuron Disease Fratta, Dr Pietro Not Applicable Non-commercial portfolio A Multicentre Biomarker Study in Neurodegeneration A Multicentre Biomarker Study in Neurodegeneration Fratta, Dr Pietro Not Applicable Non-commercial portfolio Investigation of human ion channel disorders Investigation of human neurological ion channel or episodic neurological disorders Hanna, Prof Michael Not Applicable Non-commercial portfolio Molecular mechanisms in mitochondrial disease Investigation of nuclear and mitochondrial genes underlying mitochondrial and muscle-related genetic diseases Hanna, Prof Michael Pilot/Feasibility Non-commercial non-portfolio Biobank to facilitate future therapy of neuromuscular disorders. Setting up of a rare diseases biological samples bank (biobank) for research to facilitate pharmacological, gene and cell therapy trials in neuromuscular disorders (NMD) Hanna, Prof Michael Not Applicable Non-commercial non-portfolio MRI in Motor Neuropathies V1 Magnetic Resonance Imaging as an outcome measure in Motor Neuropathies Hanna, Prof Michael Not Applicable Molecular alterations in inclusion body myositis Genetic, transcriptomic and proteomic alterations in inclusion body myositis Hanna, Prof Michael Not Applicable Non-commercial non-portfolio International Sporadic Inclusion Body Myositis Genetic Study Using Next Generation Sequencing to Unravel the Pathogenesis of Sporadic Inclusion Body Myositis (IBM) International - IBM Consortium Genetic Study Hanna, Prof Michael Not Applicable Non-commercial portfolio ICGNMD INTERNATIONAL CENTRE FOR GENOMIC MEDICINE IN NEUROMUSCULAR DISEASES (ICGNND) Hanna, Prof Michael Not Applicable Non-commercial portfolio Mitocohort Medical Research Council Centre for the Neuromuscular diseases: Mitochondrial disease patient cohort study Hanna, Prof Michael Not Applicable Non-commercial portfolio Lupin PASS study An Observational Study to Describe the Long-term Safety and Effectiveness of Namuscla in the Symptomatic Management of Myotonia in Adult Patients with Non-dystrophic Myotonic Disorders Hanna, Prof Michael Not Applicable Commercial non-portfolio mitoPK [11C]PK11195 PET as a biomarker to diagnose and monitor natural history in mitochondrial disease Hanna, Prof Michael Not Applicable Non-commercial non-portfolio Nicotinamide Riboside in mitochondrial biogenesis To study the role of nicotinamide riboside in inducing mitochondrial biogenesis Hanna, Prof Michael Not Applicable Non-commercial portfolio Activity & sedentary behaviour in channelopathy and Myotonic Dystrophy Measuring activity and sedentary behaviour in people with Muscle Ion Channelopathy and Myotonic Dystrophy type 1, and assessing the use of individual goal setting to support increasing habitual physical activity activity. Holmes, Miss Sarah Not Applicable Non-commercial non-portfolio UK register for hereditary spastic paraplegia UK register for hereditary spastic paraplegia Houlden, Prof Henry Not Applicable Non-commercial portfolio International Genetics Collaboration (IGC) International Genetics Collaboration (IGC): An observational study in the UK only, with international collaborators, to identify disease genes, genetic and COVID-19 risk factors, disease biomarkers and fundamental genetic mechanisms Houlden, Prof Henry Not Applicable Non-commercial portfolio MRI in Inflammatory Neuropathies Diagnostic Magnetic Resonance Imaging Characteristics of Inflammatory Neuropathies Lunn, Prof Michael Not Applicable Non-commercial portfolio International Guillain–Barré Syndrome Outcome Study Clinical and biological determinants of disease course in Guillain-Barré syndrome: a prospective UK-wide observational study interfacing with the International Guillain-Barré syndrome Outcome Study -IGOS. Lunn, Prof Michael Not Applicable Non-commercial portfolio Creation and validation of biomarkers in inflammatory neuropathies A pilot study to develop and validate biomarkers to monitor disease progression in POEMS syndrome and inflammatory neuropathies using a disability scale designed using Rasch statistics and magnetic resonance imaging Lunn, Prof Michael Not Applicable Single molecule biomarker discovery in inflammatory neuropathy A pilot study to identify ultra low concentration biomarkers in the serum of patients with inflammatory neuropathy using the ultrasensitive Single Molecule Analyser (SIMOA) apparatus Lunn, Prof Michael Study Non-commercial non-portfolio The pathogenesis of idiopathic sensory ganglionopathy Investigations into the possible antibody mediated causes of idiopathic sensory ganglionopathy Lunn, Prof Michael Not Applicable Non-commercial non-portfolio OPTIC Trial Intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in CIDP Lunn, Prof Michael Phase III Non-commercial portfolio Advanced metrology in axial spondyloarthritis (ANDROID) An investigation into the responsiveness of Inertial Measurement Unit (IMU) sensor-based tests in measuring changes in spinal mobility and function in axial spondyloarthritis patients treated with biologic drugs Machado, Dr Pedro Not Applicable Non-commercial portfolio IBM Swallow Evaluation of dysphagia in inclusion body myositis (IBM) Machado, Dr Pedro Not Applicable Non-commercial non-portfolio PHOENIX Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients with Amyotrophic Lateral Sclerosis (ALS) Malaspina, Dr Andrea Phase III Commercial portfolio Lighthouse II RANDOMISED DOUBLE-BLIND PLACEBO-CONTROLLED PHASE 3 TRIAL OF TRIUMEQ IN
AMYOTROPHIC LATERAL SCLEROSISMalaspina, Dr Andrea Phase III Non-commercial portfolio MYO-Guide: a machine learning approach to the analysis of MRI Implementation of an artificial intelligence module on the online imaging portal MYO-Share for guiding the diagnosis of muscle disease Morrow, Dr Jasper Not Applicable Non-commercial non-portfolio ALS Biomarkers Study Characterisation of a panel of disease biomarkers in peripheral blood from individuals with amyotrophic lateral sclerosis/motor neuron disease Malaspina, Dr Andrea Not Applicable Non-commercial portfolio An RCT of ACT for people with MND A randomised controlled trial of acceptance and COMmitment therapy for people with Motor nEuroN Disease (COMMEND) Malaspina, Dr Andrea Not Applicable Non-commercial portfolio Adult SMA REACH Study Adult SMA REACH: Development and implementation of a standardised data set and data collection research study in the UK Adult SMA population Parton, Dr Matthew Not Applicable Non-commercial portfolio Clinical Trial Assessing KL1333 A Randomised, Double-blind, Parallel-group, Placebo-controlled, Phase Ia/Ib, Multiple-site Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of KL1333 after a Single Oral Dose and Multiple Ascending Oral Doses in Healthy Subjects and Patients with Primary Mitochondrial Disease Pitceathly, Dr Robert Phase I Commercial portfolio Mitox study The effects of oxygen in the context of mitochondrial dysfunction Pitceathly, Dr Robert Not Applicable Non-commercial portfolio Natural History Study of Pyruvate Dehydrogenase Deficiency Natural History Study of Pyruvate Dehydrogenase Deficiency Pitceathly, Dr Robert Not Applicable Non-commercial portfolio Phase 2b Safety and Efficacy Study of REN001 in Mitochondrial Myopathy A double-blind, placebo-controlled, study to evaluate the efficacy and safety of 24 weeks treatment with REN001 in patients with Primary Mitochondrial Myopathy (PMM) Pitceathly, Dr Robert Phase II Commercial portfolio ION-PROM Development of a patient reported outcome measure (PROM) for patients with
inherited optic neuropathy (ION)Pitceathly, Dr Robert Not Applicable Non-commercial portfolio 2020-104A - Abliva - Mitochondrial Disease An interventional, randomised, double-blind, parallel-group, placebo-controlled, flexible-dose, adaptive study of the efficacy of KL1333 in adult patients with primary mitochondrial disease (PMD) Pitceathly, Dr Robert Phase III Commercial portfolio REN001-201-DXA, a sub-study of the STRIDE study AN EXPLORATORY STUDY TO ASSESS CHANGE IN BONE MINERAL DENSITY (BMD) AFTER 24 WEEKS TREATMENT OF REN001 IN SUBJECTS TAKING PART IN THE STRIDE STUDY, USING DUAL-ENERGY X-RAY ABSORPTIOMETRY (DXA) Pitceathly, Dr Robert Phase II Commercial portfolio Open label study of REN001 in PMM subjects An open label, multi-centre study to evaluate the long-term safety and tolerability of REN001 in subjects with primary mitochondrial myopathy (PMM) Pitceathly, Dr Robert Phase II Commercial portfolio A Phase 1b Open Label Study of REN001 in McArdle Disease A PHASE 1B, OPEN-LABEL STUDY TO EVALUATETHE SAFETY AND TOLERABILITY OF 12 WEEKS TREATMENT WITH ORAL REN001 IN PATIENTS WITH MCARDLE DISEASE (GLYCOGEN STORAGE DISORDER TYPE 5) Quinlivan, Prof Rosaline Phase I Commercial portfolio A Natural History Study in Patients with Duchenne Muscular Dystrophy A Natural History Study in Patients with Duchenne Muscular Dystrophy Managed in Routine Clinical Practice. Quinlivan, Prof Rosaline Not Applicable Commercial portfolio Rare Diseases Translational Research- DMD Neuromuscular Rare Disease Translational Research in patients with Duchenne Muscular Dystrophy Quinlivan, Prof Rosaline Not Applicable Non-commercial portfolio MRI in McArdle Disease Muscle MRI in patients with McArdle Disease (Glycogen storage disease type V) Quinlivan, Prof Rosaline Not Applicable Non-commercial non-portfolio Survey about urinary symptoms and renal function in adults with DMD Survey about the prevalence of urinary symptoms and abnormal renal function in adults with Duchenne muscular dystrophy (DMD) who are on and off glucocorticoids (CSs) treatment Quinlivan, Prof Rosaline Not Applicable Non-commercial non-portfolio EDG5506BMD A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy Quinlivan, Prof Rosaline Phase II Commercial portfolio MRI and Gait in dHMN Exploring muscle structure, function and gait patterns in people with Distal Hereditary Motor Neuropathy: natural history and the effect of rehabilitation interventions Ramdharry, Dr Gita Not Applicable Non-commercial non-portfolio Fatigue in individuals infected with SARS-Cov-2 Identifying incidence and nature of Fatigue in Individuals infected with COVID-19 (SARS-Cov-2) in the United Kingdom Ramdharry, Dr Gita Not Applicable Non-commercial non-portfolio ADAPT-NMD study ADAPT NMD: A hybrid II study of the feasibility and implementation of a self-management programme for people with neuromuscular diseases Ramdharry, Dr Gita Not Applicable Non-commercial portfolio Supporting Consultations in Remote Physiotherapy (SCiP) Supporting Consultations in Remote Physiotherapy (SCiP): a mixed-methods study to develop guidance for video consultations in physiotherapy services Ramdharry, Dr Gita Not Applicable Non-commercial non-portfolio CMT: A Natural History Study Charcot-Marie-Tooth Disease and Related Disorders: A Natural History Study Reilly, Prof Mary Not Applicable Non-commercial portfolio Preventable Axon Degeneration In Human Disease Preventable Axon Degeneration In Human Disease Reilly, Prof Mary Not Applicable Non-commercial portfolio Muscle MRI in Charcot-Marie-Tooth Disease (CMT) Muscle MRI in Charcot Marie Tooth Disease: a Prospective Cohort Study Reilly, Prof Mary Not Applicable Non-commercial portfolio CMT-TREAT-NMD-ID Charcot Marie Tooth disease Translational Research in Europe for the Assessment and Treatment of Neuromuscular Diseases International Database (CMT-TREAT-NMD-ID) Reilly, Prof Mary Not Applicable Non-commercial non-portfolio Endemic neuropathy in UK African and Caribbean communities Investigating the genetic basis of nutritional optic and sensory neuropathy in African and Caribbean communities in the UK Rossor, Mr Alex Not Applicable Non-commercial portfolio MND Register for England, Wales and Northern Ireland MND Register for England, Wales and Northern Ireland Sidle, Dr Katie Not Applicable Non-commercial portfolio Immune Profiles in Myasthenia Gravis Comparison of lymphocyte subset, cytokine, and complement profiles in myasthenia gravis of different severity, disease time-points, and treatment history Spillane, Dr Jennifer Not Applicable Non-commercial non-portfolio Developing antigen specific immunotherapy for myasthenia gravis Identification of antigen processing independent epitopes for developing antigen specific immunotherapy in myasthenia gravis Spillane, Dr Jennifer Not Applicable Non-commercial portfolio MEND: MExiletine versus lamotrigine in Non-Dystrophic myotonias A phase III, randomised, double blinded, head-to-head, single-site, cross-over trial of lamotrigine versus mexiletine for non-dystrophic myotonias. Vivekanandam, Dr Vinojini Phase III Non-commercial portfolio END-DM1 Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 Turner, Dr Chris Not Applicable Non-commercial non-portfolio DYNE101-DM1-201 A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing
Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101
Administered to Participants with Myotonic Dystrophy Type 1Turner, Dr Chris Phase II Commercial portfolio A Phase 1/2 Study to Evaluate AOC 1020 in Adult Participants with FSHD A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD) Bugiardini, Dr Enrico Phase I/II Commercial Portfolio MANOEUVRE A PHASE II, MULTICENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO EVALUATE THE PHARMACODYNAMICS, SAFETY, TOLERABILITY, PHARMACOKINETICS, AND EFFICACY OF RO7204239 IN PARTICIPANTS WITH FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY Bugiardini, Dr Enrico Phase II Commercial portfolio Neurotoxicity of immune checkpoint inhibitor therapy Neurotoxicity of immune checkpoint inhibitor therapy: A UK-wide case series Carr, Dr Aisling Not applicable Non-commercial Non-portfolio
A Phase II/III Study of ABC008 to Evaluate Efficacy and Safety in IBM A Phase II/III Randomized, Double-blind, Placebo- controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis Machado, Dr Pedro Phase II/III Commercial, portfolio NEPTUNIA A Phase IIa, Randomized, Parallel, Double-Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of Enpatoran in Dermatomyositis and Polymyositis Participants receiving Standard of Care Machado, Dr Pedro Phase IIa Commercial portfolio The Natural History of Inclusion Body Myositis The Natural History of Inclusion Body Myositis Machado, Dr Pedro Not applicable Non-Commercial Portfolio A Study of Nipocalimab in Participants with Active Idiopathic Inflammatory Myopathies A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Nipocalimab in Participants with Active Idiopathic Inflammatory Myopathies Machado, Dr Pedro Phase II Commercial portfolio ARGX-113-2007 - Argenx SE - Myositis - 2682/0027 A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathies Machado, Dr Pedro Phase II/III Commercial portfolio Phoenix OLE A Phase IIIb, Open Label Extension Study Evaluating The Safety And Tolerability of AMX0035 Up To 108 Weeks In Adult Participants with Amyotrophic Lateral Sclerosis (ALS) Previously Enrolled In Study A35-004 (PHOENIX) Malaspina, Dr Andrea Phase IIIb Commercial portfolio MND SMART Motor Neurone Disease Systematic Multi-Arm Adaptive Randomised Trial Malaspina, Dr Andrea Phase II/III Non-commercial portfolio 2020-104A - Abliva - Mitochondrial Disease An interventional, randomised, double-blind, parallel-group, placebo-controlled, flexible-dose, adaptive study of the efficacy of KL1333 in adult patients with primary mitochondrial disease (PMD) Pitceathly, Dr Robert Phase III Commercial portfolio RYR1-RM Endpoints Study An Observational Study in Participants with Ryanodine Receptor 1-Related Myopathies (RYR1-RM) to Determine Optimal Endpoint Measurements. Quinlivan, Prof Rosaline Not Application Commercial portfolio Sense Randomised, double blind, placebo-controlled trial of L-serine in Hereditary Sensory Neuropathy type 1.
Reilly, Prof Mary Phase II Non-commercial portfolio An inherited peripheral neuropathy database for the United Kingdom
A registry of patients with CMT in the UK who are willing to be contacted for ethically approved research projects.
Rossor, Dr. Alex Not applicable Non-commercial non-portfolio Alexion A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel, Multicenter Study to Evaluate the Safety and Efficacy of ALXN1720 in Adults with Generalized Myasthenia Gravis
Spillane, Dr Jennifer Phase III Commercial portfolio CARE CDM CArdio-REspiratory function in adult patients with Congenital Myotonic Dystrophy Turner, Dr Chris Not applicable Non-commercial portfolio
- Clinical & Experimental Epilepsy
Short title Long title PI Phase Type PRISM Project The Prevention and Risk Identification of SUDEP (Sudden Unexpected Death in Epilepsy) Mortality - PRISM Project Diehl, Dr Beate Not Applicable Non-commercial non-portfolio Optimising cognitive outcomes in epilepsy surgery Optimising cognitive outcomes in epilepsy surgery with direct cortical stimulation: a pilot, feasibility study Diehl, Dr Beate Non-commercial non-portfolio Optimizing epilepsy surgery V1 Optimizing epilepsy surgery Duncan, Prof John Not Applicable Non-commercial portfolio The 7-TLE Study The 7-Tesla Temporal Lobe Epilepsy Study Duncan, Prof John Not Applicable Non-commercial non-portfolio Multicentre Epilepsy Lesion Detection Project: a collaborative cohort Multicentre Epilepsy Lesion Detection Project: a collaborative cohort for the analysis of focal epilepsies Duncan, Prof John Not Applicable Non-commercial non-portfolio Pilot Pharmacological-fMRI Study Pilot Pharmacological fMRI Study - The effects of drugs on cognition in epilepsy Koepp, Prof Matthias Non-commercial non-portfolio Epilepsy and neurodegeneration: disease mechanisms and early detection Epiphany: Epilepsy and neurodegeneration: disease mechanisms and early detection Koepp, Prof Matthias Not Applicable Non-commercial non-portfolio Longitudinal cognitive fMRI: Rehabilitation and Surgery in epilepsy Longitudinal cognitive fMRI: Rehabilitation and Surgery in epilepsy Sidhu, Dr Meneka Non-commercial non-portfolio EpiPGX Epilepsy Pharmacogenomics: delivering biomarkers for clinical use Sisodiya, Prof Sanjay Non-commercial portfolio Cortical excitability in genetic neurological disorders Cortical excitability in subjects with genetic neurological disorders, a Transcranial Magnetic Stimulation study Sisodiya, Prof Sanjay Academic/student Epilepsy surgical and post mortem tissue bank Epilepsy (surgical and post mortem) research tissue bank. Thom, Dr Maria Non-commercial non-portfolio Person centred risk communications about epilepsy Person centred risk communication - what works best for people with epilepsy and their clinicians (a qualitative study) Walker, Prof Matthew Not Applicable Non-commercial portfolio High frequency activity in epilepsy patients High frequency oscillations in epilepsy patients: an intracranial electroencephalography and single unit recording multicase study at the National Hospital for Neurology and Neurosurgery Walker, Prof Matthew Non-commercial non-portfolio The use of portable Magnetoencephalography (MEG) in epilepsy. v1.1 The use of portable Magnetoencephalography (MEG) in epilepsy. v1.1 Walker, Prof Matthew Non-commercial non-portfolio Biomarkers to distinguish epileptic from non-epileptic seizures II Biomarkers to distinguish epileptic from functional non-epileptic seizures II: Using bio-signals obtained from EEG and non-EEG sensors to distinguish epileptic from functional non-epileptic seizures, an observational study Yogarajah, Dr Mahinda Not Applicable Non-commercial portfolio SCN1A Horizons: a natural history study of SCN1A epilepsies in the UK SCN1A Horizons: A natural history study of SCN1A-related epilepsies in the United Kingdom Sisodiya, Prof Sanjay Not Applicable Non-commericial. Portfolio Anhedonia in epilepsy Anhedonia in epilepsy (part of Epilepsy surgery programme) Vivekananda, Dr Umesh Not Applicable Non-commericial ADIE-FS Aligning Dimensions of Interoceptive Experience in Functional Seizures: A Feasibility Pilot Study Yogarajah, Dr Mahinda Not Applicable Non-commericial. non-portfolio Vibes Volatile non-Invasive Biomarkers of Epileptic Seizures Walker, Prof Matthew Not Applicable Non-commericial. Portfolio EpilSurgDB UCLH Epilepsy Surgery Database Duncan, Prof John Not Applicable Non-commericial ATMS study on cortical excitability in epilepsy A Transcranial Magnetic Stimulation study to measure cortical excitability in people with epilepsy Chowdhury, Dr Fahmida Not Applicable Non-commericial
- Neuroinflammation
Short title Long title PI Phase Type UK MS Register The UK Multiple Sclerosis Register Braisher, Mrs Marie Non-commercial portfolio Safety of IxaZOmib targeting plasma cells in MS (SIZOMUS) Safety of ixazomib targeting plasma cells in multiple sclerosis: A phase 1b randomised, double-blind, placebo-controlled trial. Brownlee, Dr Wallace Phase Ib Non-commercial portfolio MS-PROACTIVE Preventing job loss using Acceptance and Commitment Therapy in Vocational Rehabilitation Brownlee, Dr Wallace Not Applicable Non-commercial portfolio BN42083 - OCREVUS HIGH DOSE PPMS STUDY A PHASE IIIB MULTICENTER, RANDOMIZED, DOUBLE-BLIND, CONTROLLED STUDY TO
EVALUATE THE EFFICACY, SAFETY AND PHARMACOKINETICS OF A HIGHER DOSE OF OCRELIZUMAB IN ADULTS WITH PRIMARY PROGRESSIVE MULTIPLE SCLEROSISBrownlee, Dr Wallace Phase III Commercial portfolio SATRALIZUMAB IN PATIENTS WITH NEUROMYELITIS OPTICA SPECTRUM DISORDER (NMOSD) A Multicenter, Single Arm, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Satralizumab in Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) Brownlee, Dr Wallace Phase IIIb Commercial portfolio CAMELOT-MS Cladribine tablets in Highly-Active Relapsing Multiple Sclerosis - Real-World Effectiveness in UK Clinical Practice Brownlee, Dr Wallace Not Applicable Commercial non-portfolio Misdiagnosis of MS in the UK Misdiagnosis of MS in the UK Brownlee, Dr Wallace Not Applicable Non-commercial non-portfolio Dynamics of grey matter & white matter pathology in multiple sclerosis The dynamics and clinical relevance of grey matter and periventricular white matter pathology in multiple sclerosis Chard, Dr Declan Not Applicable Non-commercial portfolio MN39158 - A Long Term Extension Study in Multiple Sclerosis A SINGLE ARM, OPEN LABEL MULTICENTRE EXTENSION STUDY TO EVALUATE THE EFFECTIVENESS AND SAFETY OF OCRELIZUMAB IN PATIENTS WITH MULTIPLE SCLEROSIS PREVIOUSLY ENROLLED IN A F. HOFFMANN-LA ROCHE SPONSORED OCRELIZUMAB PHASE IIIb/IV CLINICAL TRIAL Chataway, Prof Jeremy Phase III Commercial portfolio OCTOPUS - Optimal Clinical Trials Platform for Progressive Multiple Sclerosis A multicentre, interventional, multi-arm, multi-stage trial including randomisation, double blinding, placebo control evaluation of treatments for slowing the progression of disability in participants with Secondary Progressive Multiple Sclerosis (SPMS) and Primary Progressive Multiple Sclerosis (PPMS), together termed Progressive Multiple Sclerosis (PMS) Chataway, Prof Jeremy Phase III Non-commercial portfolio To evaluate safety and efficacy of ION373 in Alexander Disease A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease Chataway, Prof Jeremy Phase IIa Commercial portfolio MS-NIRS Near-infrared spectroscopy to investigate cortical oxygenation and metabolism in multiple sclerosis – an observational cohort pilot study (student study) Chataway, Prof Jeremy Non-commercial non-portfolio MS-Bio Blood Biomarkers in Multiple Sclerosis – a non interventional cohort study (Student Study) Chataway, Prof Jeremy Non-commercial non-portfolio StarMS A multicentre, randomised controlled trial to evaluate the efficacy of autologous haematopoietic stem cell transplantation versus alemtuzumab or ocrelizumab in relapsing remitting multiple sclerosis. Ciccarelli, Prof Olga Non-commercial portfolio PITMS Predicting Individual Treatment responses towards personalised medicine in Multiple
SclerosisCiccarelli, Prof Olga Not Applicable Non-commercial portfolio MS and COVID-19 (COVID-19) (Covid, Covid-19 , Coronavirus) Towards understanding the risk of SARS-CoV-2 infection and immune response to COVID-19 vaccines in individuals with immune-mediated conditions Ciccarelli, Prof Olga Not Applicable Non-commercial non-portfolio To study clinical radiological and genetic predictors of outcome in MS Understanding the relationship between genotype and neurodegeneration in response to disease modifying treatments in multiple sclerosis: A pilot exploratory study employing high-dimensional inference of existing datasets. Ciccarelli, Prof Olga Non-commercial non-portfolio ORATORIO HAND A PHASE IIIb MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF OCRELIZUMAB IN ADULTS WITH PRIMARY PROGRESSIVE MULTIPLE SCLEROSIS De Angelis, Dr Floriana Phase III Commercial portfolio Sodium Accumulation in Multiple sclerosis In vivo quantification of tissue sodium (23Na) content in the brain and spinal cord in Multiple Sclerosis Solanky, Dr Bhavana Non-commercial portfolio A new prospective Study in Clinically Isolated Syndrome A new prospective Clinical, Genetic and Imaging study of patients with Clinically Isolated Syndrome (CIS). Toosy, Dr Ahmed Non-commercial portfolio Neuroimmunology 1.4 Investigation of autoimmunity and its effects on the brain 1.4 Zandi, Dr Michael Not Applicable Non-commercial portfolio IVIG and rituximab in antibody-associated psychosis - SINAPPS2 A randomised phase II double-blinded placebo-controlled trial of intravenous immunoglobulins and rituximab in patients with antibody-associated psychosis (SINAPPS2) Zandi, Dr Michael Phase II Non-commercial portfolio
- Clinical & Movement Neurosciences
Short title Long title PI Phase Type Dystonia Coalition Projects Dystonia Coalition Projects Batla, Dr Amit Not Applicable Non-commercial non-portfolio Molecular genetic studies of familial movement disorders. Molecular genetic studies of familial movement disorders. Bhatia, Prof Kailash Non-commercial non-portfolio SV2a evaluation in neurodegenerative research SV2a evaluation in neurodegenerative research Bhatia, Prof Kailash Non-commercial portfolio Synaptic loss in MSA Molecular Imaging of Synaptic loss in Multiple System Atrophy Bhatia, Prof Kailash Not Applicable Non-commercial portfolio Molecular and functional imaging in monogenic PD Molecular and functional imaging in monogenic PD Bhatia, Prof Kailash Not Applicable Non-commercial portfolio Longitudinal Investigation of I2BS in PD Longitudinal Investigation of Imidazoline-2 Binding Site as a Novel Marker of Disease Progression in Parkinson’s Disease: An [11C]BU99008 PET Study Bhatia, Prof Kailash Not Applicable Non-commercial portfolio Open-Label TD study to evaluate VMAT2 blockade using PET An Open-label Positron Emission Tomography Study to Evaluate the Effects of Vesicular Monoamine Transporter 2 Receptor (VMAT2) Blockade on the Dopamine Neurotransmitter System in Subjects with Tardive Dyskinesia Bhatia, Prof Kailash Not Applicable Commercial non-portfolio DNMS Quest Clinical validation of the Dystonia NMS Questionnaire (DNMS Quest) in patients with primary dystonia Bhatia, Prof Kailash Non-commercial portfolio Imaging Neuromelanin and Iron in dystonia/parkinsonism Imaging Neuromelanin and Iron in dystonia/parkinsonism Bhatia, Prof Kailash Not Applicable Non-commercial non-portfolio ATH434-201 A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF ATH434 IN MULTIPLE SYSTEM ATROPHY Chelban , Dr Viorica Phase IIb Non-commercial portfolio Effort-based decision-making in depression in Parkinson's disease Effort-based decision-making, reward, and dopamine in depression in Parkinson’s disease Costello, Dr Harry Not Applicable Non-commercial non-portfolio PRISM Project The Prevention and Risk Identification of SUDEP (Sudden Unexpected Death in Epilepsy) Mortality - PRISM Project Diehl, Dr Beate Not Applicable Non-commercial non-portfolio Optimising cognitive outcomes in epilepsy surgery Optimising cognitive outcomes in epilepsy surgery with direct cortical stimulation: a pilot, feasibility study Diehl, Dr Beate Non-commercial non-portfolio Exenatide once-weekly as a treatment for Multiple System Atrophy. A Phase IIa, open label, single-site, 48 week randomised controlled trial evaluating the safety and efficacy of Exenatide once-weekly in the treatment of patients with Multiple System Atrophy Foltynie, Prof Thomas Phase IIa Non-commercial non-portfolio Phase 3 Trial of exenatide for Parkinson’s disease A randomised, double blind, parallel group, placebo controlled, Phase 3 trial of exenatide once weekly over 2 years as a potential disease modifying treatment for Parkinson's disease. Foltynie, Prof Thomas Phase III Non-commercial portfolio Kennedy's Disease Study and Register Kennedy's Disease Study and Register Giunti, Dr Paola Non-commercial non-portfolio EFACTS European Friedreich's Ataxia Consortium for Translational Studies Giunti, Dr Paola Non-commercial portfolio Detecting Retinal Changes in ARSACS & Other Ataxias Using OCT Detecting Retinal Changes in Autosomal Recessive Spastic Ataxia of Charlevoix Saguenay (ARSACS) and Other Causes of Ataxia Using Optical Coherence Tomography (OCT) Giunti, Dr Paola Non-commercial portfolio EUROSCA European integrated project on spinocerebellar ataxias (EUROSCA): pathogenesis, genetics, models and therapy Giunti, Dr Paola Non-commercial portfolio DRPLA Natural History and Biomarkers Study (DRPLA NHBS), v1.0, 26/4/21 Dentatorubral-pallidoluysian atrophy Natural History and Biomarkers Study (DRPLA NHBS) Giunti, Dr Paola Not Applicable Non-commercial portfolio ESMI European Spinocerebellar Ataxia Type 3/Machado-Joseph Disease Initiative Giunti, Dr Paola Non-commercial portfolio BP43176 - RO7486967 IN PARTICIPANTS WITH PARKINSON’S DISEASE A PHASE 1B, ADAPTIVE, MULTI-CENTER, RANDOMIZED, DOUBLE BLIND, PLACEBO-CONTROLLED, PARALLEL DESIGN STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7486967 IN PARTICIPANTS WITH EARLY IDIOPATHIC PARKINSON’S DISEASE Korlipara, Dr Prasad Phase Ib Commercial portfolio Op-TICS Double blind comparison of optimised Deep Brain Stimulation for severe Tourette syndrome (Op-TICS) Limousin, Prof Patricia Not Applicable Non-commercial portfolio Neurophysiologic assessment of ET patients treated by Vim DBS Assessment of cerebellar ataxia associated with Ventral intermediate median nucleus deep brain stimulation in essential tremor: a multimodal physiologic study Limousin, Prof Patricia Non-commercial non-portfolio Brain Sensing in neurological and psychiatric Disorders Electrophysiologic brain sensing using implanted DBS systems in neurological and psychiatric disorders Limousin, Prof Patricia Not Applicable Non-commercial non-portfolio 254PD101: Phase 1 Study BIIB094 Administered Intrathecally to Adults A Phase 1 Single- and Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB094 Administered Intrathecally to Adults With Parkinson’s Disease Morris, Prof Huw Phase I Commercial portfolio 262SP101 Phase 1 Study of BIIB101 in Multiple System Atrophy A Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB101 Administered Intrathecally to Adults with Multiple System Atrophy (MSA). Morris, Prof Huw Phase I/II Commercial portfolio PSP CBD MSA Longitudinal Study UK PROgressive Supranuclear Palsy CorTicoBasal Syndrome Mulitple System Atrophy Longitudinal Study UK (PROSPECTMUK) Morris, Prof Huw Non-commercial portfolio Parkinson's and Movement Disorders Families Project (PFP) Parkinson's and Movement Disorders Families Project (PFP) Morris, Prof Huw Not Applicable Non-commercial portfolio Predict PD the 1000/100/100 pilot study A pilot study of an algorithm designed to identify Parkinson's disease in the early, nonmotor phase. Noyce, Dr Alastair Non-commercial portfolio CHIEF-PD STUDY CHIEF-PD (CHolinesterase Inhibitor to prEvent Falls in Parkinson’s Disease): A phase 3 randomised double-blind placebo-controlled trial of rivastigmine to prevent falls in Parkinson’s disease Saifee, Dr Tabish Phase III Non-commercial portfolio ADepT-PD A Randomised Placebo-Controlled Trial of Escitalopram and Nortriptyline with Standard Psychological Care for Depression in Parkinson’s Disease Saifee, Dr Tabish Phase III Non-commercial portfolio PPD Predicting Risk of Parkinson’s Disease in the Community Schrag, Dr Anette-Eleonore Non-commercial portfolio POP-PD Patterns of Perception in Parkinson's disease: Understanding the taboo of talking about dementia in Parkinson's Weil, Dr Rimona Not Applicable Non-commercial non-portfolio Vision in Parkinson's Disease Disruption of visual processing in Parkinson's Disease Weil, Dr Rimona Non-commercial portfolio Metacognitive function in dementia and Parkinson's disease Investigating metacognition in Alzheimer’s disease and Parkinson’s disease. Weil, Dr Rimona Non-commercial portfolio TOP HAT Version 1_19.11.19 Trial of Ondansetron as a Parkinson’s HAllucinations Treatment Weil, Dr Rimona Phase IIb Non-commercial portfolio The UCL BioResource version 1. The University College London (UCL) BioResource. Wood, Prof Nicholas Not Applicable Non-commercial non-portfolio NIHR BioResource - Rare Diseases National Institute for Health Research BioResource - Rare Diseases Wood, Prof Nicholas Non-commercial portfolio The effect of weak polarizing currents on the excitability of human motor cortex The effect of weak polarizing currents on the excitability of human motor cortex Rothwell, Prof John Non-commercial non-portfolio Effect of short bursts of high frequency, low intensity TMS on cortical excitability Assessment of the effect of short bursts of high frequency, low intensity TMS on cortical excitability, the corticospinal pathway, and behaviour in healthy subjects and patients with movement disorders Rothwell, Prof John Non-commercial non-portfolio
- Neurosurgery
Short title Long title PI Phase Type ROAR Study Risk of Aneurysm Rupture Study Grover, Mr Patrick Not Applicable Non-commercial portfolio CARE pilot trial Cavernomas A Randomised Effectiveness (CARE) pilot trial, to address the effectiveness of medical management versus medical and surgical management (with neurosurgery or stereotactic radiosurgery) in people with symptomatic brain cavernoma Grover, Mr Patrick Not Applicable Non-commercial portfolio AIP Genetics of Endocrine tumours Marcus, Dr Hani Not Applicable Non-commercial portfolio Axonal regeneration using nasal mucosa derived olfactory cells Sourcing specialized olfactory ensheathing cells from nasal mucosal lining of adults to repair spinal cord injuries Marcus, Dr Hani Not Applicable Non-commercial portfolio AI in endoscopic transsphenoidal surgery The application of Artificial Intelligence to patients undergoing endoscopic transsphenoidal surgery: a single-site prospective feasibility study. Marcus, Dr Hani Not Applicable Non-commercial non-portfolio Electrocorticographic Monitoring of Brain Retraction Injury Electrocorticography as a Neurophysiological Marker for Intraoperative Monitoring of Brain Retraction Marcus, Dr Hani Not Applicable Non-commercial non-portfolio Robotic guidance of SEEG electrodes and brain biopsies Use of Autoguide and EpiNav for directing SEEG electrodes and cranial biopsies McEvoy, Mr Andrew Not Applicable Non-commercial portfolio Electrical Impedance Tomography & selective stimulation of vagus nerve Electrical Impedance Tomography imaging of functional anatomy and selective stimulation of fascicles within the vagus nerve McEvoy, Mr Andrew Not Applicable Non-commercial non-portfolio Cellular neurobiology of the human brain in health and disease Exploring the physiology of brain neurons, glia and vasculature in health, and their dysfunction in ischaemia and neurodegenerative disease Sethi, Dr Huma Not Applicable Non-commercial non-portfolio THE SOCIAL CONSEQUENCES OF NEW TECHNOLOGIES IN BRAIN TUMOURS Treatment in a new era of molecular and personalised medicine: Understanding and anticipating the social consequences for people affected by brain tumours Thorne, Mr Lewis Not Applicable Non-commercial portfolio ICP dynamics Intracranial Pressure Monitoring in Cerebrospinal Fluid Dynamics disorders: a retrospective observational study Toma, Dr Ahmed Not Applicable Non-commercial non-portfolio ICP position related changes Continuous body position monitoring in patients undergoing continuous intracranial pressure monitoring, a feasibility study Toma, Dr Ahmed Not Applicable Non-commercial non-portfolio SC IL-1Ra in SAH - phase III trial Does Interleukin-1 Receptor Antagonist Improve Outcome following aneurysmal Subarachnoid Haemorrhage (aSAH)? A Phase III trial Toma, Dr Ahmed Phase III Non-commercial portfolio A new prospective Study in Clinically Isolated Syndrome A new prospective Clinical, Genetic and Imaging study of patients with Clinically Isolated Syndrome (CIS). Toosy, Dr Ahmed Not Applicable Non-commercial portfolio UK Chiari 1 Study UK Chiari 1 Study Watkins, Mr Laurence Not Applicable Non-commercial non-portfolio Deep Brain Activity in Cluster Headache and Mood Disorder Ventral Tegmental Area Local Field Potentials and Brain Magnetoencephalography Measurement in Cluster Headache and Mood Disorder Zrinzo, Mr Ludvic Not Applicable Non-commercial non-portfolio SVP and ICP Association Spontaneous Retinal Venous Pulsation (SVP) and its association with Intracranial Pressure (ICP) in different body
positionsBremner, Dr Fion Not Applicable Non-commercial non-portfolio Spontaneous Retinal Venous Pulsation and its correlation with ICP Spontaneous retinal venous pulsation and its correlation with intracranial pressure in patients with cerebrospinal fluid dynamics disturbances Bremner, Dr Fion Not Applicable Non-commercial non-portfolio