The LWENC CRF currently has approximately 47 studies in the active recruitment or set-up stage. Most of our studies range from First-Time-in-Human (FTIH) Phase I to Phase IIb clinical trials. We also undertake experimental observational/longitudinal studies and our strengths are in performing translational clinical research.
Below are examples of studies currently running at the LWENC CRF:
IONIS MAPT Rx (Phase I, FIH)
This study is a Phase I trial investigating the safety and tolerability of a new drug in Alzheimer’s disease (AD). The study drug is administered every 4 weeks for 13 weeks via intrathecal injection (i.e. directly into the CSF via the spinal canal). As this drug is early in its development, the study is mainly interested in its effects on the body, and the body’s effect on the drug. If results are positive the study will move on to assess the efficacy of the drug in altering the progression of the disease rather than simply treating its symptoms.
VAL-1221 (Phase I, FIH)
This is a first-in-human study of the safety and efficacy of a novel therapy in patients with Pompe Disease, a rare lysosomal storage disorder. Patients with the disease have problems breaking down glycogen in their muscles leading to muscle dysfunction and morbidity. This study will examine the safety profile of the drug and whether it leads to improvements in clearance of glycogen and better functional outcomes (e.g. lung function, motor skills, etc.).
LEGATO-HD (Phase II)
Inflammatory reactions are part of the changes occurring in the brain in the course of HD. Laquinimod has been shown to attenuate immune responses in inflammatory processes within the body including the brain and has shown favourable effects in preclinical studies, suggesting it may have a benefit for patients with HD. The efficacy and safety of multiple doses of laquinimod (0.5, 1.0 and 1.5 mg/day) as a potential treatment for patients with HD is being tested in this trial.
Passport BIIB092 (Phase II)
Passport is a Phase II study in Progressive Supranuclear Palsy, a neurological condition related to Parkinson’s disease, caused by the premature loss of nerve cells in certain parts of the brain. The purpose of the study is to determine the efficacy and safety of intravenously administered BIIB092 with changes in a PSP-specific functional rating scale representing the primary outcome.
MIROCALS (Phase II)
This is a Phase II study of a new therapeutic agent for Amyotrophic Lateral Sclerosis (also known as Motor Neurone Disease). This study will determine whether enhancing regulatory T cells modifies the body’s immune response and changes the rate of ALS disease progression. Newly diagnosed participants will receive the study drug (Id-IL-2) or placebo and will be followed up over 18 months to examine the effects of the drug on survival and functional decline.
LRRK2 (experimental medicine)
This is an epidemiological study to gain insight and understanding of the molecular pathogenesis of Parkinson’s disease, including the downstream biochemical and signaling consequences of genetic risk factors and the identification of crucial molecular triggers in the pathogenesis of PD.
An MRC funded epidemiological longitudinal study is ongoing at the CRF to determine the causes and consequences of brain amyloidosis, atrophy and cerebrovascular disease in 500 individuals enrolled in the 1946 birth cohort.
DIAN-TU-001 (Phase II/III)
This is a landmark trial testing two potential disease modifying therapies (Gantenerumab and Solanezumab) in subjects known to have a genetic mutation causing AD or those at risk of an autosomal dominant Alzheimer’s disease (ADAD) mutation. This study represents the first attempt ever made to demonstrate that monoclonal antibodies are able to prevent build up of amyloid load prior to symptom onset and therefore prevent their development.
GN28525 (Phase II)
The long term safety and tolerability of crenezumab is being investigated in this open label study in order to assess its suitability as a potential treatment for patients with AD. Crenezumab is a monoclonal antibody to abeta; peptides which are believed to be causative in AD.