AMX0035 for ALS

(Phoenix Trial)

Recruiting at 76 trial locations

Overseen By Amylyx Trial Operations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Amylyx Pharmaceuticals Inc.

Must be taking: Riluzole, Edaravone

Must not be taking: PB, Taurursodiol

Disqualifiers: Tracheostomy, Renal insufficiency, Heart failure, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 3

Duration: 48 weeks

664 Participants Needed

Trial Summary

What is the purpose of this trial?

The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS

Research Team

Amylyx Study Director

Principal Investigator

Amylyx Pharmaceuticals

Eligibility Criteria

This trial is for adults diagnosed with ALS within the last 2 years who can consent and follow study procedures. They must use birth control during the study and not plan to conceive. Excluded are pregnant or breastfeeding women, those with severe medical conditions, on salt restriction, enrolled in other trials, using prohibited meds, or have specific health issues like low lung capacity or poor kidney function.

Inclusion Criteria

I can and will follow all trial procedures and visit requirements.

My first ALS symptoms appeared less than 2 years ago.

I have been diagnosed with ALS.

Exclusion Criteria

I haven't taken PB or taurursodiol in the last 30 days.

Currently enrolled in another trial involving use of an investigational therapy

My kidney function is low, with an eGFR under 60.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive AMX0035 or placebo for 48 weeks to evaluate safety and efficacy in ALS treatment

48 weeks

Once daily for first 3 weeks, then twice daily

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term follow-up

Long-term survival will be monitored by assessing all-cause mortality

3 years from LPI

Treatment Details

Interventions

• AMX0035
• Placebo

Trial Overview The Phoenix Trial is testing AMX0035 against a placebo to see if it's safe and effective for treating ALS. This Phase III trial randomly assigns participants to either receive AMX0035 or a placebo without them knowing which one they're getting (double-blind).

Participant Groups

Group I: AMX0035 Experimental Treatment 1 Intervention

Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating

Group II: Placebo Placebo Group 1 Intervention

Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating

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+