Multiple System Atrophy
Conditions
Keywords
Multiple System Atrophy, ATH434, Neurodegenerative disease, Shy-Drager Syndrome, Movement disorders, Autonomic dysfunction, Synucleinopathies, Atypical parkinsonism
Brief summary
This study will assess the safety and efficacy of ATH434 in participants with Multiple System Atrophy
Interventions
DRUGATH434 dose level 1
ATH434 taken BID
DRUGATH434 dose level 2
ATH434 taken BID
DRUGPlacebo
Placebo taken BID
Sponsors
Alterity Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
30 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
1. Participant has clinical features of parkinsonism. 2. Participant has evidence of orthostatic hypotension and/or bladder dysfunction. 3. Participant has ataxia and/or pyramidal signs on neurological examination. 4. Participant is ambulatory. 5. Participant has biomarker evidence of MSA in biologic fluid and on MRI.
Exclusion criteria
1. Participant has motor symptoms for \> 4 years. 2. Participant has advanced disease, as indicated by frequent falls or choking. 3. Participant has structural brain abnormality on MRI. 4. Participant has any significant neurological disorder other than MSA. 5. Participant has an unstable medical or psychiatric illness. 6. Participant has a contraindication to, or is unable to tolerate, MRI or lumbar puncture.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in iron content as measured by brain MRI | Change from Baseline to Week 52 |
Secondary
| Measure | Time frame |
|---|---|
| Change in Neurofilament Light Chain Levels | Change from Baseline to Week 52 |
| Change in Unified MSA Rating Scale (UMSARS) Score | Change from Baseline to Week 52 |
| Change in SF-36 Score | Change from Baseline to Week 52 |
Countries
Australia, France, Italy, New Zealand, United Kingdom, United States
Outcome results
None listed