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

• Status: Not yet recruiting
• Trial end date: 2024-02-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amylyx Pharmaceuticals Inc.
• Treatments: 4-phenylbutyric acid

Criteria

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