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Current Clinical Trials

Alzheimer's disease (AD)

 
Lilly (J4T-MC-OLAA)      > We are recruiting!

Official title

A single- and multiple-ascending dose study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of LY3954068 in patients with early symptomatic Alzheimer’s disease

 

Purpose of the study

A phase I, randomised, double-blind, placebo-controlled study to evaluate the safety of LY3954068 in participants with early symptomatic Alzheimer's Disease (AD). The study will also investigate how much LY3954068 gets into the bloodstream and will test the effects of LY3954068 on CSF tau protein and other markers of AD.

 

Participants

Patients with a diagnosis of early AD, with a MMSE score of 18-30, who are between 50 and 85 years of age. Participants must be in good general health, other than AD.

 

What is involved?

The study will be comprised of two parts, A and B. Part B is optional. Each enrolled participant in Part A will be randomly assigned to receive a single dose of LY3954068 or placebo (no active drug) into the spinal fluid. If conducted, each participant in Part B would receive 2 doses of either LY3954068 or placebo, which is salty water, administered into the spinal fluid. Participants will receive active drug or placebo in a 3:1 ratio. After screening, participants will be required to stay at the research site for 2-3 nights. The study will last up to approx 45 weeks (10 months) for Part A, and, if conducted, 73 weeks (17 months) for Part B, including the screening period. There may also be an optional Open Label Extension study for this trial.

Participants will visit us in Queen Square, London, for drug/placebo administration, and also for assessments including medical and neurological examinations, memory and thinking questionnaires, blood and urine tests, brain scans, and lumbar punctures.

NIO-SILK      > We are recruiting!

Official title
A Phase 1b randomized, participant and investigator blinded, placebo-controlled study to evaluate the ability of intrathecally (IT) administered NIO752 to lower cerebrospinal fluid (CSF) total tau synthesis in participants with Alzheimer’s Disease (AD) measured by stable isotope labelling kinetics.

Purpose of the study
To test the safety and efficacy of an antisense oligonucleotide therapy to block the synthesis of tau production by nerve cells in the brain. It is hoped that this will prevent the progression of clinical cognitive decline and rate of neuronal loss.

Participants
Patients with a diagnosis of early to moderate AD, including those with an autosomal dominant (or genetic) form of AD. Participants must be in good general health, other than AD.

What is involved
4 week screening period: consent and eligibility assessments. If passed, participants will be randomised into drug or placebo groups and enter the treatment period. All participants will be offered active drug at the second dose.

18 week treatment period: Intrathecal (via lumbar puncture) administration of drug or placebo followed by an overnight stay. Second overnight visit where a stable isotope (non radioactive label) will be administered for 16 hours overnight with regular blood tests. Four further lumbar punctures, assessments and safety monitoring.

12 week follow up safety period: continued assessments and safety monitoring.

TRAILBLAZER-ALZ 5      > Recruitment opening soon!

Official title

Global study to assess safety and efficacy of Donanemab in early symptomatic Alzheimer’s disease (AD).

 

Purpose of the study

A Phase 3, double-blind, placebo-controlled study to evaluate the safety and efficacy of donanemab (LY3002813) in participants with early symptomatic AD (prodromal AD and mild dementia due to AD) with the presence of brain tau pathology. This study will assess whether removal of amyloid plaques in the brain can slow progression of disease as assessed by clinical outcomes for cognition and function, and by imaging biomarker measures of disease pathology and neurodegeneration.

 

Participants

Patients with a diagnosis of early symptomatic AD, with a MMSE score of 20-28, who are 60-85 years of age. Participants must be in good general health, other than AD.

 

What is involved?

The study duration, including screening and follow-up, is up to 93 weeks (22 months) and will include 34 visits to our clinical trial site in Queen Square.

Screening period: up to 9 weeks. Assessments to determine eligibility for the trial.

Treatment phase: 76 weeks. Participants who pass screening will be randomised in a 1:1 ratio to receive active drug (donanemab) or placebo (salty water) by intravenous infusion, once every 4 weeks.

Follow-up: up to 8 weeks of continued assessments

Throughout all stages of the trial, participants will have assessments including medical and health checks, neurological examinations, brain scans (MRI and PET), blood and urine tests, and  memory and thinking questionnaires

GSK Progress AD      > recruitment paused

Official title

A phase 2, parallel group, randomized, double-blind, placebo-controlled, 3-arm, multicenter treatment study to evaluate the efficacy and safety of GSK4527226 [AL101] intravenous infusion compared with placebo in patients with early Alzheimer’s disease (AD)

 

Purpose of the study

To assess the efficacy and safety of GSK4527226 in participants with early AD (including Mild Cognitive Impairment (MCI) and mild dementia due to AD). GSK4527226 is an antibody which blocks sortilin and increases PGRN levels which may reduce the rate of neuronal loss and clinical decline.

 

Participants

Patients with a diagnosis of early AD, with a MMSE score of 21-29, who are between 50 and 85 years of age. Participants must be in good general health, other than AD.

 

What is involved?

12 week screening period: consent and eligibility assessments. If passed, participants will be randomised into drug or placebo groups and enter the treatment period. 2 in 3 participants will receive active drug, and 1 in 3 participants will receive placebo.

76 weeks (1.5 years) of treatment: monthly IV administrations of drug or placebo.

12 week follow-up safety period: continued assessments and safety monitoring

24 month open-label extension (optional): Possible opportunity to receive the active drug long-term.

Throughout all stages of the trial, participants will visit us regularly in Queen Square for a number of assessments including medical and health checks, neurological examinations, brain scans, blood and urine tests, memory and thinking questionnaires, and optional lumbar punctures.

Alnylam-EOAD (ALN-APP-001)      > recruitment paused

Official title
A phase 1 study to evaluate the safety and tolerability of ALN-APP in patients with early onset Alzheimer’s disease (AD)

Purpose of the study
To evaluate the safety (side effects) of a drug called ALN- APP. This drug may have the potential to slow disease progression by reducing production of amyloid protein, which builds up in the brains of people with AD.

Participants
Patients with a diagnosis of early onset AD (onset <65 years), with a MMSE score of 21+, who are over the age of 18. Participants must be in good general health, other than AD.

What is involved?
In Part A of the study, participants will make a total of approx. 18 visits over a period of just over a year. Each visit will take approx. 3 to 6 hours depending on the procedures completed at each visit. An overnight stay may also be required. If a participant passes screening, a single dose of ALN-APP or placebo is administered, and they are closely monitored for several months afterwards. Assessments include medical and neurological examinations, memory and thinking questionnaires, blood and urine tests, brain scans (MRI and PET), and a lumbar puncture.

Part B will be a multi-dose open-label period including patients previously enrolled in Part A, as well as new participants. This will involve multiple administrations of ALN-APP (all patients will receive the active drug and there will be no placebo). The estimated duration of Part B for each participant is up to 2 years, including a 12 month dosing period (7 separate visits) and 6-12 month follow-up period (at least 2-3 visits).

Brain Shuttle (Trontinemab)      > recruitment paused

Official title

A phase 1b/2a, randomized, double-blind, placebo-controlled, multiple-ascending dose, parallel-group study to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of RO7126209 following intravenous infusion in patients with prodromal or mild to moderate Alzheimer’s disease (AD)

 

Purpose of the study

To assess the safety and side effects of Trontinemab (RO7126209), an anti-amyloid antibody with enhanced blood-brain barrier crossing, in patients with prodromal or mild to moderate AD.

 

Participants

Patients with a diagnosis of Mild Cognitive Impairment (MCI) due to AD, or mild to moderate AD, with a MMSE score of 18-28, who are between 50 and 85 years of age. Participants must be in good general health, other than AD.

 

What is involved?

Screening: consent and eligibility assessments. If passed, participants will be randomised into drug or placebo groups (with an allocation ratio of 4:1) and enter the treatment period.

Placebo-controlled treatment: Intravenous (IV) administration of gradually increasing doses of drug (or placebo) with close safety monitoring at each dose escalation, followed by continued treatment at the determined optimum dosing regimens (or placebo). There will be a treatment period of 28 weeks, followed by a period of safety monitoring, then another treatment period of 24 weeks followed by further safety monitoring.

Open-label extension: Eligible participants may be offered continued treatment with the active drug (no placebo group) and safety monitoring for approx. 2 years.

Throughout all stages of the trial, participants will visit us regularly in Queen Square for assessments including medical and health checks, neurological examinations, brain scans, blood and urine tests, memory and thinking questionnaires, and lumbar punctures.

Upcoming trials

We have a number of studies currently in set-up which are due to open to recruitment in the coming weeks and months. 

Ongoing trials      (closed to recruitment)

INVOKE (AL002-2): A phase 2 study evaluating safety and efficacy of an antibody targeting the immune system in early Alzheimer’s disease (AD)

DESPIAD: A phase 2b trial investigating the safety and efficacy of miridesap, a drug which reduces a protein called Serum Amyloid P Component, in mild AD

INFRONT trials AL001-2, AL001-3 and AL001-CS-302: phase 2 and phase 3 studies assessing the effects of increasing progranulin using the antibody drug AL001 on slowing disease progression and the onset of symptoms in progranulin gene mutation carriers with or at risk of Frontotemporal dementia (FTD).
 
Biogen CELIA (BIIB080): A phase 2 study assessing safety and efficacy of anti-tau drug BIIB080 in patients with Mild Cognitive Impairment (MCI) and mild AD. Biogen are investigating if reducing tau production will slow down the progression of cognitive dysfunction.
 
DIAN-TU-001 (E2814 Secondary Prevention Tau NexGen): A phase 2/3 trial evaluating biomarker, cognitive, and clinical endpoints following open-label lecanemab administration alongside another potential disease-modifying therapy, E2814 in patients with Dominantly Inherited Alzheimer’s Disease (DIAD).
 
ImmunoBrain (IBC-01-01): A phase I study investigating the safety of a new drug, IBC-Ab002, in patients with early AD. IBC-Ab002 is an antibody which may help suppress age-related immune system decline by blocking certain immune system pathways that cause age-related impairment, slowing progression of AD.

 

Dominantly Inherited Alzheimer’s Disease (DIAD), or familial Alzheimer’s disease (fAD)

NIO-SILK      > We are recruiting!

Official title
A Phase 1b randomized, participant and investigator blinded, placebo-controlled study to evaluate the ability of intrathecally (IT) administered NIO752 to lower cerebrospinal fluid (CSF) total tau synthesis in participants with Alzheimer’s Disease (AD) measured by stable isotope labelling kinetics.

Purpose of the study
To test the safety and efficacy of an antisense oligonucleotide therapy to block the synthesis of tau production by nerve cells in the brain. It is hoped that this will prevent the progression of clinical cognitive decline and rate of neuronal loss.

Participants
Patients with a diagnosis of early to moderate AD, including those with an autosomal dominant (or genetic) form of AD. Participants must be in good general health, other than AD.

What is involved
4 week screening period: consent and eligibility assessments. If passed, participants will be randomised into drug or placebo groups and enter the treatment period. All participants will be offered active drug at the second dose.

18 week treatment period: Intrathecal (via lumbar puncture) administration of drug or placebo followed by an overnight stay. Second overnight visit where a stable isotope (non radioactive label) will be administered for 16 hours overnight with regular blood tests. Four further lumbar punctures, assessments and safety monitoring.

12 week follow up safety period: continued assessments and safety monitoring.

Upcoming trials

We have studies currently in set-up which are due to open to recruitment in the coming weeks and months. 

  • A phase 1b anti-tau gene silencing trial looking at the effects of drug on tau production and clearance in Alzheimer’s disease (AD) and Dominantly Inherited Alzheimer’s Disease (DIAD)
  • An open label extension study evaluating impact of continued amyloid removal on disease progression in DIAD
Ongoing trials      (closed to recruitment)
  • DIAN-TU-001 (E2814 Secondary Prevention Tau NexGen): A phase 2/3 trial evaluating biomarker, cognitive, and clinical endpoints following open-label lecanemab administration alongside another potential disease-modifying therapy, E2814 in patients with Dominantly Inherited Alzheimer’s Disease (DIAD). 

Frontotemporal dementia (FTD)

INFRONT-2 (AL001-2)      > recruitment closed

Official title
A Phase 2 Open-Label Study to Evaluate the Safety and Tolerability of AL001 in Heterozygous Carriers of Granulin or C9orf72 Mutations Causative of Frontotemporal Dementia

Purpose of the study
To see how safe and well-tolerated the experimental drug AL001 is when given intravenously to participants with a mutation in the gene for Granulin (GRN) or C9orf72, which causes frontotemporal dementia (FTD). Mutations such as GRN and C9orf72 cause accumulation of a protein called TDP-43 in the brain, leading to disruption of brain activity, and neurodegeneration. AL001 prevents the breakdown of another protein, Progranulin, which has been found to reduce TDP-43 in non-clinical studies. Therapeutics targeted at reducing TDP-43 may slow the progression of disease in FTD.

Participants
This study will enrol symptomatic and asymptomatic participants with a GRN mutation who completed the AL001-1 study, as well as symptomatic patients with a GRN or C9orf72 mutation who have not had AL001 before.

What is involved
Participants will receive up to 13 doses of AL001 over a 48 week period. Study procedures will include blood sampling, adverse event monitoring, ECG, blood pressure, physical and neurological examinations, lumbar puncture, questionnaires, and MRI. 

INFRONT-3 (Alector, AL001-3)      > recruitment closed

Official title
A Phase 3, Randomized, 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

Purpose of the study
Alector, Inc. is studying AL001 as a new experimental drug for frontotemporal dementia (FTD) caused by mutations in the progranulin gene. These mutations reduce progranulin levels in the body and may lead to symptoms of FTD. The purpose of the phase 3 study is to learn whether increasing progranulin levels with treatment with AL001 will delay onset of symptoms or slow disease progression, when compared to a placebo (a solution that contains no active AL001 drug).

Participants
Open to participants who have been diagnosed with FTD and have a progranulin gene mutation OR have a progranulin gene mutation and are at risk of developing FTD symptoms as evidenced by a biomarker.

What is involved
AL001 or placebo will be administered every 4 weeks by an intravenous (IV) infusion. Assessments will include regular medical examinations, blood tests, brain imaging (MRI), and completion of questionnaires. All participants will be in the study about 2 years and will need to visit the study site at least once a month during this time. Participants who complete the study and who meet the criteria will be eligible to continue to the optional Open Label Extension, during which all participants will receive AL001. 

Taking part

If you would like more information about taking part in our clinical trials, please email drctrialenquiries@ucl.ac.uk. If you are unable to contact us by email, please call our team during office hours (9am-5pm, Mon-Fri) on 020 3448 3105.