Wolfram Syndrome Clinical Trial
Official title:
A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
Verified date | April 2024 |
Source | Amylyx Pharmaceuticals Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
Status | Active, not recruiting |
Enrollment | 12 |
Est. completion date | January 30, 2026 |
Est. primary completion date | July 17, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 17 Years and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Washington University | Saint Louis | Missouri |
Lead Sponsor | Collaborator |
---|---|
Amylyx Pharmaceuticals Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | To evaluate the effect of AMX0035 during a 0-240 minutes mixed-meal tolerance test (MMTT) | C-peptide AUC response at Week 24
Change from Baseline in C-peptide at Week 24 |
24 weeks | |
Primary | To assess the safety and tolerability of AMX0035in adult participants with Wolfram syndrome | Incidence and severity of Adverse Events and Serious Adverse Events
Incidence of abnormalities in clinical laboratory assessments |
100 weeks | |
Secondary | To assess the effect size of AMX0035 on visual acuity | Change from Baseline on best-corrected visual acuity (Logmar) at Week 24 | 24 weeks | |
Secondary | To evaluate the effect of AMX0035 on total daily insulin dose | Change from Baseline of exogenous insulin dose to Week 24 | 24 weeks | |
Secondary | To evaluate the effect of AMX0035 on glucose range | Change from Baseline to Week 24
Time in good range Time below range Time above range |
24 weeks | |
Secondary | To evaluate the effect of AMX0035 on HbA1c levels | Change from Baseline to Week 24 in HbA1c level | 24 weeks |
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