A Biomarker Study of ATH434 in Participants With MSA

An Open-Label Biomarker Study of ATH434 in Multiple System Atrophy

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05864365

Enrollment

Registered

2023-05-18

Start date

2023-05-23

Completion date

2025-02-26

Last updated

2025-04-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Multiple System Atrophy

Keywords

Multiple System Atrophy, ATH434, Neurodegenerative disease, Shy-Drager Syndrome, Movement disorders, Autonomic dysfunction, Synucleinopathies, Atypical parkinsonism, Biomarkers

Brief summary

This study will assess the safety and efficacy of ATH434 in participants with a clinical diagnosis of Multiple System Atrophy

Interventions

DRUGATH434

ATH434 taken by mouth two times per day

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

30 Years to 75 Years

Healthy volunteers

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 has biomarker evidence of MSA in biologic fluid and on MRI.

Exclusion criteria

1. Participant is unable to swallow study drug. 2. Participant is unable to attend study visits or complete study procedures. 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 brain volume as measured by MRI	Change from Baseline to Week 52

Secondary

Measure	Time frame
Change in Clinical Global Impression of Change (CGI-C)	Change from Baseline to Week 52
Change in Neurofilament Light Chain Levels	Change from Baseline to Week 52
Change in Patient Global Impression of Change (PGI-C)	Change from Baseline to Week 52
Change in iron content in substantia nigra as measured by MRI	Change from Baseline to Week 52
Change in Unified MSA Rating Scale (UMSARS) Score	Change from Baseline to Week 52

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026