Clinical Trials in the USA / NCT05676034
Active, not recruiting
Phase 2
AMX0035 in Adult Patients With Wolfram Syndrome
NCT05676034 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Wolfram Syndrome
Study summary
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
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
Primary outcome measure(s)
- To evaluate the effect of AMX0035 on residual beta-cell function by monitoring C-peptide levels during a 0-240 minutes mixed-meal tolerance test (MMTT) — 24 weeks
* C-peptide area under the curve (AUC) response at Week 24 using a 0-240 minute MMTT * Change from Baseline in area under the curve (AUC) in delta C-peptide at Week 24 using a 0-240 minute MMTT - To assess the safety and tolerability of AMX0035 administered orally for up to 208 weeks in adult participants with Wolfram syndrome — 208 weeks
* Incidence and severity of Adverse Events and Serious Adverse Events * Incidence of abnormalities in clinical laboratory assessments
Trial sites (1)
| Facility | City | Region | Status |
|---|---|---|---|
| Washington University | St Louis | Missouri |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05676034 on ClinicalTrials.gov ↗ ← All trials in the USA