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Hope for rare wolfram syndrome: drug aims to slow diabetes progression

NCT ID NCT05676034

Disease control Sponsor: Amylyx Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests an experimental drug called AMX0035 in 12 adults with Wolfram syndrome, a rare genetic disorder that causes diabetes and vision loss. The goal is to see if the drug can help preserve the pancreas's ability to make insulin and to check its safety over up to 4 years. Participants take the drug by mouth and wear a continuous glucose monitor throughout the study.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

12 people

The number who actually took part.

Started

Mar 2023

Expected to finish

May 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

17 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

Wolfram syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Wolfram syndrome

Contacts and locations

Locations

  • Washington University

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.