Study of ATH434 in Participants with Multiple System Atrophy

NCT05109091 | Completed Phase 2 FDA Drug

• 1 other identifier: ATH434-201
• Study Type: interventional
• Target: 77
• Locations: 6 countries
• Sites: 23

Brief Summary

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

Conditions

Multiple System Atrophy

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Change in iron content as measured by brain MRI

  Change from Baseline to Week 52

Secondary Outcomes (3)

• 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

Study Arms (3)

ATH434 Arm 1

EXPERIMENTAL

Drug: ATH434 dose level 1

ATH434 Arm 2

EXPERIMENTAL

Drug: ATH434 dose level 2

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

ATH434 dose level 1 DRUG

ATH434 taken BID

Also known as: PBT434

ATH434 Arm 1

ATH434 dose level 2 DRUG

ATH434 taken BID

Also known as: PBT434

ATH434 Arm 2

Placebo DRUG

Placebo taken BID

Placebo

Eligibility Criteria

• Age: 30 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant has clinical features of parkinsonism.
• Participant has evidence of orthostatic hypotension and/or bladder dysfunction.
• Participant has ataxia and/or pyramidal signs on neurological examination.
• Participant is ambulatory.
• Participant has biomarker evidence of MSA in biologic fluid and on MRI.

You may not qualify if:

• Participant has motor symptoms for \> 4 years.
• Participant has advanced disease, as indicated by frequent falls or choking.
• Participant has structural brain abnormality on MRI.
• Participant has any significant neurological disorder other than MSA.
• Participant has an unstable medical or psychiatric illness.
• Participant has a contraindication to, or is unable to tolerate, MRI or lumbar puncture.

Sponsors & Collaborators

• Alterity Therapeutics: lead

Study Sites (23)

University of California San Diego

La Jolla, California, 92093, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

The Movement Disorder Clinic of Oklahoma

Tulsa, Oklahoma, 74136, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

St Vincent's Hospital (Sydney)

Darlinghurst, New South Wales, Australia

Location

Westmead Hospital

Westmead, New South Wales, Australia

Location

The Alfred Hospital

Melbourne, Victoria, Australia

Location

CHU de Toulouse/Hôpital Pierre-Paul Riquet

Toulouse, Haute-Garonne, 31059, France

Location

CHU de Bordeaux/Groupe Hospitalier Pellegrin

Bordeaux, 33076, France

Location

CHU de Marseille/Hôpital de la Timone

Marseille, 13005, France

Location

CHU/HU Pitié Salpêtrière

Paris, Île-de-France Region, 75013, France

Location

IRCCS Istituto Delle Scienze Neurologiche di Bologna

Bologna, Bologna, Italy

Location

Policlinico di Milano

Milan, Italy

Location

Azienda Ospedaliero-Universitaria Pisana

Pisa, Italy

Location

AOU San Giovanni di Dio Ruggi d'Aragona

Salerno, Italy

Location

New Zealand Brain Research Institute

Christchurch, New Zealand

Location

Auckland City Hospital

Grafton, New Zealand

Location

University College London

London, England, United Kingdom

Location

Newcastle University

Newcastle upon Tyne, England, United Kingdom

Location

Salford Royal Hospital

Salford, England, United Kingdom

Location

Queen Elizabeth University Hospital

Glasgow, Scotland, United Kingdom

Location

MeSH Terms

Conditions

Multiple System Atrophy; Neurodegenerative Diseases; Shy-Drager Syndrome; Movement Disorders; Primary Dysautonomias; Synucleinopathies

Interventions

8-hydroxyquinazolin-4(3H)-one

Condition Hierarchy (Ancestors)

Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Hypotension; Vascular Diseases; Cardiovascular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 22, 2021
• First Posted: November 5, 2021
• Study Start: July 1, 2022
• Primary Completion: November 28, 2024
• Study Completion: November 28, 2024
• Last Updated: December 13, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

IT (4); NZ (2); GB (4); FR (4); US (6); AU (3)