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A clinical study of ATH434 in patients with multiple system atrophy

Phase 1
Conditions
Multiple System Atrophy (MSA)
MedDRA version: 21.1Level: PTClassification code 10064060Term: Multiple system atrophySystem Organ Class: 10029205 - Nervous system disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Registration Number
EUCTR2021-003231-29-AT
Lead Sponsor
Alterity Therapeutics Limited
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
60
Inclusion Criteria

1. Participant is 30 to 75 years old at the time of Screening.
2. Participant has clinical features of parkinsonism.
3. Participant has evidence of orthostatic hypotension and/or bladder dysfunction.
4. Participant has ataxia and/or pyramidal signs on neurological examination.
5. Participant is ambulatory.
6. Participant has biomarker evidence of MSA in biologic fluid and on MRI
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 30

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 evidence of cognitive impairment.
4. Participant has structural brain abnormality on MRI.
5. Participant has any significant neurological disorder other than MSA.
6. Participant has an unstable medical or psychiatric illness.
7. Participant has a contraindication to, or is unable to tolerate, MRI or lumbar puncture.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: To assess the efficacy of ATH434 in subjects with MSA.;Secondary Objective: • To assess the safety and tolerability of ATH434 in subjects with MSA<br>• To evaluate the pharmacokinetics (PK) of ATH434 and potential metabolites in subjects with MSA;Primary end point(s): Change in iron content as measured by brain MRI;Timepoint(s) of evaluation of this end point: MRI will be performed at Screening, Week 26 and Week 52 visits.
Secondary Outcome Measures
NameTimeMethod
Secondary end point(s): Key Secondary Endpoint:<br>Change in Unified MSA Rating Scale (UMSARS Part I) Score<br>Additional Secondary Endpoints:-<br> Change in Motor Examination Score<br>- Change in Aggregating alpha-Synuclein Levels<br>- Change in Neurofilament Light Chain Levels<br>- Change in SF-36 Score ;Timepoint(s) of evaluation of this end point: Changes from Baseline (Day 1) to Week 52 (in-clinic visits Day 1, Week 13, 26, 39 and 52).
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