AMX0035 in Adult Patients With Wolfram Syndrome

Last updated: March 28, 2025
Sponsor: Amylyx Pharmaceuticals Inc.
Overall Status: Active - Not Recruiting

Phase

2

Condition

Retinitis Pigmentosa

Treatment

AMX0035

Clinical Study ID

NCT05676034
A35-008
  • Ages > 17
  • All Genders

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 Criteria

Inclusion

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

Exclusion

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 totreatment would put the patient at risk, in the Investigator's judgment

  • Received treatment with any investigational drug or device within the 30 days (or 5half-lives, whichever is longer) prior to first dose at Day 1

  • Previous treatment with gene or cellular therapy

Study Design

Total Participants: 12
Treatment Group(s): 1
Primary Treatment: AMX0035
Phase: 2
Study Start date:
March 03, 2023
Estimated Completion Date:
December 31, 2026

Study Description

AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and to evaluate the effect of AMX0035 on residual beta cell functions by monitoring c-peptide levels during a 0-240 minute mixed-meal tolerance test. The trial will also assess the effects of AMX0035 on changes to diabetic measurements including daily insulin dose, time in good glucose range, and HbA1c levels. Effect on best-corrected visual acuity in both eyes will also be evaluated.

Connect with a study center

  • Washington University

    Saint Louis, Missouri 63110
    United States

    Site Not Available

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