Open-Label Access to ATH434 for Patients Who Completed Study ATH434-201 in France

NCT07729852 | Not Yet Recruiting Phase 2 FDA Drug

• 2 other identifiers: ATH434-2032025-524317-88-00
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 4

Brief Summary

This multicenter open-label extension study conducted in France is designed to provide ATH434 orally BID to eligible patients who completed the Phase 2 study ATH434-201, and who may benefit from this treatment according to the evaluation by the Investigator.

Conditions

Multiple System Atrophy

Outcome Measures

Primary Outcomes (5)

• Incidence, Severity, and Relationship of Adverse Events (AEs) and Serious Adverse Events (SAEs)

  All AEs will be reviewed at every participant contact and documented in the CRF. All SAEs will be reported within 24 hours of awareness in accordance with local regulatory requirements.

  Baseline to completion of study treatment, an average of 1 year.

• Change From Baseline in Clinical Laboratory Parameters

  Clinical laboratory parameters include hematology and blood chemistry panels.

  Baseline to completion of study treatment, an average of 1 year.

• Change From Baseline in Vital Signs

  Vital signs include blood pressure, pulse, temperature, and respiration rate.

  Baseline to completion of study treatment, an average of 1 year.

• Duration of Exposure to ATH434

  Total duration of treatment with investigational product per participant.

  Baseline to completion of study treatment, an average of 1 year.

• Cumulative Dose of ATH434

  Total cumulative dose of investigational product administered per participant.

  Baseline to completion of study treatment, an average of 1 year.

Study Arms (1)

ATH434 75mg BID

EXPERIMENTAL

Participants who completed the prior ATH434-201 Phase 2 trial will receive ATH434 75 mg orally twice daily (BID). Treatment consists of an initial 12-month treatment phase, followed by an ongoing extension phase based on investigator assessment of continued clinical benefit, safety, and tolerability

Drug: ATH434

Interventions

ATH434 DRUG

ATH434 75 mg tablets administered orally twice daily (BID).

Also known as: PBT434

ATH434 75mg BID

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Completed Alterity protocol ATH434-201.
• Expected to benefit from treatment from treatment with ATH434, in the opinion of the Investigator.

You may not qualify if:

• Discontinued prior ATH434 treatment (ATH434-201) for any reason.
• Significant medical or psychiatric condition that may decrease the benefit-risk ratio of participation in this program to an unacceptable level, as per the Investigator's opinion.

Sponsors & Collaborators

• Alterity Therapeutics: lead

Study Sites (4)

Hôpital Pierre-Paul Riquet

Toulouse, Midi-Pyrenees, 31300, France

Location

Centre Hospitalier Universitaire de Bordeaux

Bordeaux, New Aquitaine, 33076, France

Location

Hôpital de la Timone

Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France

Location

Hôpital Universitaire Pitié Salpêtrière

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

Location

MeSH Terms

Conditions

Multiple System Atrophy

Interventions

ATH434; 8-hydroxyquinazolin-4(3H)-one

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Central Study Contacts

Alterity Clinical Trials

CONTACT

+1 888-556-7582; clinicaltrials@alteritytx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 16, 2026
• First Posted: July 28, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2029
• Last Updated: July 28, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

FR (4)