AMX0035 for Wolfram Syndrome

Not currently recruiting at 1 trial location

Overseen By Amy Gauger

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Amylyx Pharmaceuticals Inc.

Disqualifiers: Heart failure, Cancer history, Salt restriction, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 144 weeks

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. ^{1 2 3 4 5}

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. ^{1 3 6 7 8}

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

I have been diagnosed with Wolfram syndrome.

I have diabetes that requires insulin because of Wolfram syndrome.

I am 17 years old or older.

Exclusion Criteria

I haven't taken any experimental drugs or used experimental devices recently.

I have a history of heart failure.

I have conditions affecting bile acid movement due to gut surgery or inflammation.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

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

144 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

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?

Group I: AMX0035 Experimental Treatment 1 Intervention

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:

Approved in United States as Relyvrio for:

• Amyotrophic Lateral Sclerosis

Approved in Canada as Albrioza for:

• Amyotrophic Lateral Sclerosis

Who Is Running the Clinical Trial?

Amylyx Pharmaceuticals Inc.

Lead Sponsor

Trials

12

Recruited

1,600+

Published Research Related to This Trial

Relyvrio (sodium phenylbutyrate-taurursodiol) has been approved for treating amyotrophic lateral sclerosis (ALS) based on data from a small phase 2 trial, which showed that it slowed the loss of functioning compared to a placebo.

The drug carries risks for patients sensitive to salt intake and those with disorders related to the pancreas, intestines, or enterohepatic circulation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New Drug Approved For ALS. Aschenbrenner, DS.[2022]

The coformulation of sodium phenylbutyrate and ursodoxicoltaurine (ALBRIOZA™) received conditional approval in Canada for treating amyotrophic lateral sclerosis (ALS) based on the results of the phase II CENTAUR trial.

In the CENTAUR trial, sodium phenylbutyrate/ursodoxicoltaurine demonstrated a significant slowing of ALS progression compared to placebo, indicating its potential efficacy in managing this neurodegenerative disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sodium Phenylbutyrate and Ursodoxicoltaurine: First Approval. Heo, YA.[2022]

Citations

1. amylyx.com amylyx.com/news/amylyx-pharmaceuticals-announces-positive-long-term-results-from-phase-2-helios-clinical-trial-of-amx0035-in-people-with-wolfram-syndrome

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 ...

3. withpower.com withpower.com/trial/phase-2-wolfram-syndrome-1-2023-1dcd4

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.html

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 ...

5. amylyx.com amylyx.com/news/amylyx-pharmaceuticals-announces-topline-results-from-global-phase-3-phoenix-trial-of-amx0035-in-als

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 ...

6. amylyx.com amylyx.com/news/amylyx-pharmaceuticals-announces-positive-topline-results-from-phase-2-helios-clinical-trial-demonstrating-sustained-improvements-with-amx0035-in-people-living-with-wolfram-syndrome

October 17, 2024 ... Wolfram syndrome. The safety profile of AMX0035 in HELIOS was consistent with prior safety data. AMX0035 was generally well-tolerated. All ...

7. biospace.com biospace.com/amylyx-pharmaceuticals-announces-interim-data-from-ongoing-phase-2-helios-clinical-trial-demonstrating-improvements-in-pancreatic-function-and-glycemic-control-with-amx0035-in-people-with-wolfram-syndrome

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 ...

8. adisinsight.springer.com adisinsight.springer.com/drugs/800044439

Sodium phenylbutyrate/ursodoxicoltaurine - AdisInsight An orally bioavailable fixed-dose combination of sodium phenylbutyrate and ursodoxicoltaurine, designated as AMX 0035, is being developed by ...