AMX0035 in Adult Patients With Wolfram Syndrome
← catalyst calendarSponsored by Amylyx Pharmaceuticals Inc. (industry) · AMLX — their whole pipeline →.
Phase
Phase 2
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
12actual
Sites
1
Country
United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2023-03-03 | actual | When the study began enrolling |
| Primary completion | 2024-07-23 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2028-05-31 | estimated | The whole study's end, after follow-up |
| First posted | 2023-01-09 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-04-14 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Wolfram Syndrome
Intervention
- Drug: AMX0035 — also filed as Proprietary formulation of taurursodiol and sodium phenylbutyrate
Primary outcomes
what the primary-completion date above is the date OFTo 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)
measured 24 weeks
To assess the safety and tolerability of AMX0035 administered orally for up to 208 weeks in adult participants with Wolfram syndrome
measured 208 weeks
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