BHV-3241 in patients with Multiple System Atrophy (M-STAR Study)
Research type
Research Study
Full title
Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy (M-STAR Study)
IRAS ID
264255
Contact name
Huw Morris
Contact email
Sponsor organisation
Biohaven Pharmaceuticals, Inc
Eudract number
2019-001100-38
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 9 months, 0 days
Research summary
MSA is an adult-onset fatal disease which causes neurodegeneration or the deterioration, destruction and loss of function of the neurons (nerve cells) in the brain/nervous system. Onset of MSA usually occurs during the sixth decade of life, and invariably leads to death after an average of 6 to 10 years from symptom onset. Causes of death for MSA patients are commonly infection and sudden death. Currently, no disease modifying treatment exists. Only symptomatic and palliative approaches are available for MSA patients, approaches which treat the symptoms of the disease and manage pain.
Biohaven Pharmaceuticals is developing a new drug, BHV-3241,for the treatment of MSA. This clinical trial will evaluate the safety, tolerability and efficacy of BHV-3241, as a possible treatment for multiple system atrophy (MSA).
The proposed trial is based on data gathered form other studies that implicate the activity of the enzyme MPO (myeloperoxidase) in the onset and progression of neurodegenerative diseases, including MSA. BHV-3241 is an inhibitor of MPO, suggesting treatment with BHV-3241 has the potential to slow neurodegeneration in MSA. The high unmet need for an effective treatment, together with the available data, provide rationale for the development of BHV-3241 as a treatment for MSA.
Approximately 252 MSA patients aged 40-75 years are expected to be enrolled into the study at 50 study centres in the USA and Europe. Eligible participants will receive either the investigational product, BHV-3241 or placebo. Participants will be randomised 1:1 to the study drug or placebo. The study will consist of a screening phase lasting a maximum of 6 weeks followed by a treatment phase of approximately 48 weeks. Participants will be assessed at clinic visits throughout their participation in the study.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
19/NW/0504
Date of REC Opinion
19 Sep 2019
REC opinion
Further Information Favourable Opinion