AMX0035 in Adult Patients With Wolfram Syndrome

Amylyx Pharmaceuticals

Status and phase: Active, not recruiting; Phase 2

Conditions: Wolfram Syndrome

Treatments: Drug: AMX0035

Study type: Interventional

Funder types: Industry

Identifiers

NCT05676034

A35-008

Details and patient eligibility

About

This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.

Full 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.

Enrollment: 12 patients

Sex: All

Ages: 17+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

12 participants in 1 patient group

AMX0035

Experimental group

Description:

AMX0035 administered by mouth for 144 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participant

Treatment:

Drug: AMX0035