AMX0035 for Wolfram Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests AMX0035, a new treatment for Wolfram syndrome, which affects the brain and pancreas and can lead to diabetes and vision problems, among other issues. The researchers aim to determine if AMX0035 is safe and effective when taken orally over a long period. Participants should have Wolfram syndrome with insulin-dependent diabetes and be comfortable using a continuous glucose monitor (CGM) throughout the study. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in Wolfram syndrome treatment.
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.
Is there any evidence suggesting that AMX0035 is likely to be safe for humans?
Research shows that AMX0035 is generally safe for people with Wolfram syndrome. Studies have found its safety aligns with earlier findings, indicating no unexpected issues. In past trials, few participants experienced side effects, and those were not serious. This treatment combines two compounds, sodium phenylbutyrate and ursodoxicoltaurine, taken orally. Although AMX0035 is not yet approved for Wolfram syndrome, its use in slowing the progression of amyotrophic lateral sclerosis (ALS) suggests it is safe for humans.12345
Why do researchers think this study treatment might be promising?
Unlike the standard treatments for Wolfram Syndrome, which mainly focus on managing symptoms like diabetes and vision loss, AMX0035 targets the underlying cellular stress that may contribute to the progression of the disease. This treatment is a combination of two compounds, sodium phenylbutyrate and tauroursodeoxycholic acid, which work together to protect brain cells from degeneration by reducing stress in the endoplasmic reticulum and mitochondria. Researchers are excited about AMX0035 because it not only offers a novel mechanism of action but also holds potential for slowing down or altering the disease progression, offering hope for a more comprehensive approach to treating Wolfram Syndrome.
What evidence suggests that AMX0035 might be an effective treatment for Wolfram syndrome?
Research has shown that AMX0035, which participants in this trial will receive, may help treat Wolfram syndrome. In one study, individuals with Wolfram syndrome who took AMX0035 for 48 weeks experienced stable or improved health in several areas. This suggests it might effectively manage the condition over time. Additionally, AMX0035 has successfully slowed the progression of another brain disease, amyotrophic lateral sclerosis (ALS). This supports the potential benefit of AMX0035 for neurological conditions like Wolfram syndrome. The available evidence is promising for those considering this treatment.13678
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
Trial Overview
The trial is testing AMX0035's safety and effectiveness in adults with Wolfram Syndrome. It's an open label Phase II study, which means everyone knows they're getting the treatment and there’s no placebo group.
How Is the Trial Designed?
1
Treatment groups
Experimental 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
AMX0035 is already approved in United States, Canada for the following indications:
- Amyotrophic Lateral Sclerosis
- Amyotrophic Lateral Sclerosis
Find a Clinic Near You
Who Is Running the Clinical Trial?
Amylyx Pharmaceuticals Inc.
Lead Sponsor
Published Research Related to This Trial
Citations
Amylyx Pharmaceuticals Announces Positive Long-Term ...
Data at Week 48 demonstrated that treatment with AMX0035 led to sustained stabilization or improvement in multiple outcomes related to ...
2.
thesnowfoundation.org
thesnowfoundation.org/positive-long-term-results-from-phase-2-helios-clinical-trial-of-amx0035-in-people-with-wolfram-syndrome/Positive Long-Term Results from Phase 2 HELIOS Clinical ...
Positive Long-Term Results from Phase 2 HELIOS Clinical Trial of AMX0035 in People with Wolfram Syndrome. Amylyx Pharmaceuticals, Inc. today ...
AMX0035 for Wolfram Syndrome · Info for Participants
AMX0035, also known as sodium phenylbutyrate/ursodoxicoltaurine, has shown effectiveness in slowing the progression of amyotrophic lateral sclerosis (ALS), a ...
4.
finance.yahoo.com
finance.yahoo.com/news/amylyx-pharmaceuticals-announces-positive-long-110000867.htmlAmylyx Pharmaceuticals Announces Positive Long-Term ...
Data at Week 48 demonstrated that treatment with AMX0035 led to sustained stabilization or improvement in multiple outcomes related to ...
Amylyx Pharmaceuticals Announces Topline Results From ...
Additionally, AMX0035 has been shown to also reduce markers associated with neurodegenerative diseases in clinical trials, including a reduction ...
October 17, 2024
... Wolfram syndrome. The safety profile of AMX0035 in HELIOS was consistent with prior safety data. AMX0035 was generally well-tolerated. All ...
Amylyx Pharmaceuticals Announces Interim Data From ...
... AMX0035 slows progression of diabetic, visual, and other measures in people living with Wolfram syndrome and to evaluate safety and tolerability ...
Sodium phenylbutyrate/ursodoxicoltaurine - AdisInsight
An orally bioavailable fixed-dose combination of sodium phenylbutyrate and ursodoxicoltaurine, designated as AMX 0035, is being developed by ...
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