A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome

A35-008

This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.

2023-03-03

2024-07-17

2026-01-30

Active (not recruiting)

Early phase I

12

Key Inclusion Criteria: * Definitive diagnosis of Wolfram syndrome * Insulin dependent diabetes mellitus due to Wolfram syndrome * At least 17 years of age * Participant must be willing to wear a CGM device for the duration of the study Key Exclusion Criteria: * Presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis) * Any history of heart failure per New York Heart Association (NYHA) * History of or family history of breast and/or ovarian cancer * Participant under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the Investigator's judgment * Received treatment with any investigational drug or device within the 30 days (or 5 half-lives, whichever is longer) prior to first dose at Day 1 * Previous treatment with gene or cellular therapy

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'A 96 week open-label treatment period of AMX0035 administered twice daily', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 12, 'type': 'ESTIMATED'}}

2024-04-26