Study of ATH434 in Participants With Multiple System Atrophy
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled Study of ATH434 in Multiple System Atrophy
IRAS ID
1004373
Contact name
David Stamler
Contact email
Sponsor organisation
Alterity Therapeutics Limited
Eudract number
2021-003231-29
Clinicaltrials.gov Identifier
Research summary
This is a randomized, double-blind, placebo-controlled, multinational study in approximately 60 subjects with MSA. Subjects must provide written informed consent prior to undergoing study procedures. Subjects must have a study partner who lives with and interacts with them daily to observe their status, goes with them to study visits, and can report potential nocturnal seizures. After Screening and Baseline assessments are complete, eligible subjects will be randomly assigned to 12 months treatment with 1 of 2 dose levels of ATH434 or placebo. Study drug will be taken twice daily. Enrollment will occur in 2 consecutive cohorts for enhanced safety monitoring in the first 15 subjects (Cohort 1). Cohort 1 will be followed for at least 2 weeks. Their safety and PK data will be reviewed by a Data Monitoring Committee (DMC) before the remaining 45 subjects (Cohort 2) are enrolled. Subjects will be assessed at in-clinic and remote (video/phone) visits during the study. After Screening and Baseline, in-clinic visits for safety and disease-related clinical assessments will occur at Weeks 2, 6, 13, 26, 39 and 52. Remote visits will occur at Weeks 4, 9, 17, 21, 30, 34, 43 and 47. A safety evaluation clinic visit will occur 2 weeks after treatment completion. This study includes blood sampling, brain MRI, and lumbar puncture (i.e., spinal tap) for assessment of biomarkers and study drug levels. Subject participation will last about 60 weeks (up to a 6-week Screening period, up to a 52-week Treatment Period, up to a 2-week Follow-up period). A Seizure Adjudication Committee (SAC), comprised of clinical experts in epilepsy, will review all possible seizure-related AEs (PSAEs) during the study. The SAC will be independent from the sponsor and will review PSAEs in a blinded manner to determine whether or not such events should be classified as seizure-related. In addition to Cohort 1 data review, the DMC will meet regularly to review unblinded safety data during study conduct.
REC name
West of Scotland REC 1
REC reference
22/WS/0028
Date of REC Opinion
8 Apr 2022
REC opinion
Further Information Favourable Opinion