AMX0035 for Wolfram Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. However, if you have taken any investigational drug or device in the last 30 days, you may not be eligible to participate.
How is the drug AMX0035 unique for treating Wolfram Syndrome?
AMX0035 is unique because it combines sodium phenylbutyrate and ursodoxicoltaurine, which may help reduce stress in the endoplasmic reticulum (a part of the cell that helps process proteins), potentially addressing a key issue in Wolfram Syndrome. This approach is different from other treatments like liraglutide, which focuses on improving insulin production and stabilizing neurological symptoms.12345
What evidence supports the effectiveness of the drug AMX0035 for treating Wolfram Syndrome?
AMX0035, also known as sodium phenylbutyrate/ursodoxicoltaurine, has shown effectiveness in slowing the progression of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease, in a phase II trial. This suggests potential benefits for other neurodegenerative conditions, although specific data for Wolfram Syndrome is not available.678910
Who Is on the Research Team?
Camille Bedrosian, MD
Principal Investigator
Amylyx Pharmaceuticals
Are You a Good Fit for This Trial?
Adults with Wolfram Syndrome can join this trial. They must be at least 17 years old, have a definitive diagnosis, and need insulin for diabetes caused by the syndrome. Participants should also agree to wear a continuous glucose monitoring (CGM) device throughout the study.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
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
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- AMX0035
AMX0035 is already approved in United States, Canada for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Amylyx Pharmaceuticals Inc.
Lead Sponsor