AMYLYX Phase 1/2 Clinical Trial

Study Overview

Official Title

A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome

Brief Summary

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

Detailed 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.

Please Note

Amylyx employees will have no involvement in patient inclusion decisions for upcoming clinical trials. They do not know any identifying information about participants in their trials to ensure that their trials are performed in an unbiased, fair, and equitable manner.

Once Amylyx has the next HELIOS trial designed and ready to enroll, they will make a public announcement and share the announcement with Wolfram syndrome advocacy groups and doctors so that everyone is aware that the trial is starting and which doctors will be enrolling participants in the trial.

When enrollment begins, patients may discuss trial participation with the doctor who manages their Wolfram syndrome care; that doctor can assess if a patient meets the eligibility criteria for the trial or may refer a patient to the closest doctor who is enrolling in the trial.

At this point in time, details of the upcoming trial are being finalized, including when it will start and where it will be. We look forward to posting more information soon.