Multiple System Atrophy
Conditions
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.
Detailed description
This is a multicenter, open-label extension (OLE) study designed to provide continued access to the investigational product ATH434 for eligible participants with Multiple System Atrophy (MSA) who have successfully completed the prior Phase 2 clinical trial (ATH434-201) in France. Up to 5 participants will be enrolled. No new participants will be recruited or enrolled in this study. The primary objective is to monitor long-term safety, tolerability, and clinical response. Eligible participants will receive ATH434 at a dose of 75 mg twice daily (BID) to be taken with food. The study is divided into two operational phases: Initial Treatment Phase: Participants will receive treatment for a planned duration of 12 months. Extension Phase: Participants may continue treatment beyond the initial 12 months based on the investigator's clinical judgment of ongoing benefit, safety, and tolerability. Continued eligibility will be re-evaluated and documented every 3 months. Participants will undergo a combination of in-clinic and remote visits to perform safety monitoring, clinical evaluations, and investigational product accountability.
Interventions
DRUGATH434
ATH434 75 mg tablets administered orally twice daily (BID).
Sponsors
Alterity Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Completed Alterity protocol ATH434-201. * Expected to benefit from treatment from treatment with ATH434, in the opinion of the Investigator.
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence, Severity, and Relationship of Adverse Events (AEs) and Serious Adverse Events (SAEs) | Baseline to completion of study treatment, an average of 1 year. | 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. |
| Change From Baseline in Clinical Laboratory Parameters | Baseline to completion of study treatment, an average of 1 year. | Clinical laboratory parameters include hematology and blood chemistry panels. |
| Change From Baseline in Vital Signs | Baseline to completion of study treatment, an average of 1 year. | Vital signs include blood pressure, pulse, temperature, and respiration rate. |
| Duration of Exposure to ATH434 | Baseline to completion of study treatment, an average of 1 year. | Total duration of treatment with investigational product per participant. |
| Cumulative Dose of ATH434 | Baseline to completion of study treatment, an average of 1 year. | Total cumulative dose of investigational product administered per participant. |
Countries
France
Contacts
CONTACTAlterity Clinical Trials
Outcome results
None listed