NCT05676034Active, not recruitingPhase 2
AMX0035 in Adult Patients With Wolfram Syndrome
A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
Source: ClinicalTrials.gov · Last updated 14 Apr 2026
Brief summary (registry, in English)
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 12 (actual)
- First submitted
- 22 Dec 2022
- Start date
- 3 Mar 2023
- Primary completion date
- 23 Jul 2024
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- NCT number
- NCT05676034
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 12
- From 3 Mar 2023 to the primary completion date, 23 Jul 2024
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis)
- 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
Other illness
- Any history of heart failure per New York Heart Association (NYHA)
- History of or family history of breast and/or ovarian cancer
Other
- Participant under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the Investigator's judgment
Who the study is looking for
- 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
Age in the registry: 17 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Washington University Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.