NCT05676034Phase 2Active not recruiting

AMX0035 in Adult Patients With Wolfram Syndrome

Study sponsor: Amylyx Pharmaceuticals Inc. · AMLX

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2024-07-23Actual

Enrollment · participants12Actual

Source updated2026-04-14Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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Posted 2026-04-14 · 1 field changed

Main study measure wording changed

Text changed; whether the study measures changed has not been verified. Saved text may be shortened.

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− Before

Measure: 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) Measure: To assess the safety and tolerability of AMX0035 administered orally for up to ~~144~~ weeks in adult participants with Wolfram syndrome

+ After

Measure: 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) Measure: To assess the safety and tolerability of AMX0035 administered orally for up to 208 weeks in adult participants with Wolfram syndrome

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Study details

Conditions studied

Wolfram Syndrome

Treatments or interventions studied

AMX0035DrugAlso known as Proprietary formulation of taurursodiol and sodium phenylbutyrate

Primary outcomes · main study measures

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measure: 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); description: * 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; timeFrame: 24 weeks; measure: To assess the safety and tolerability of AMX0035 administered orally for up to 208 weeks in adult participants with Wolfram syndrome; description: * Incidence and severity of Adverse Events and Serious Adverse Events * Incidence of abnormalities in clinical laboratory assessments; timeFrame: 208 weeks

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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