HEALEY ALS Platform Trial

Plain English Summary

HEALEY ALS Platform Trial - Master Protocol is a Phase 3 clinical trial sponsored by Merit E. Cudkowicz, MD studying Amyotrophic Lateral Sclerosis. Tests new drugs like Zilucoplan and Pridopidine for ALS treatment. For adults diagnosed with ALS within 2 years, who can swallow pills. Participation involves regular visits and taking study drugs. Alternatives include standard care and other clinical trials. The trial aims to enroll 1500 participants.

Official Summary

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults with ALS, diagnosed within 2 years, can join. Excludes those with unstable medical conditions or active cancer. Must be able to swallow pills and be geographically accessible. No prior investigational ALS treatments within 30 days. This trial is studying Amyotrophic Lateral Sclerosis, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

Measures disease progression, helping patients understand how well the treatment works. The specific primary outcome measures are: Disease Progression (36 Weeks). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial fills a gap by testing multiple potential ALS treatments in a large, well-designed study. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Amyotrophic Lateral Sclerosis, where improved treatment options are needed.

Investor Insight

Largest ALS trial to date, with strong sponsors and a high chance of approval. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage. The large enrollment target of 1500 participants suggests significant investment in this program.

Is This Trial Right for Me?

Ask your doctor about your ALS diagnosis and if you qualify. Participation involves regular visits and taking study drugs. This trial is currently recruiting participants. The trial is being conducted at 20 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 1,500 participants

Interventions

• DRUG: Zilucoplan — Drug: Zilucoplan Administration: Subcutaneous injection Dose: Minimum of .0.22 mg/kg daily to a maximum dose of 0.42 mg/kg daily, dependent on weight
• DRUG: Verdiperstat — Drug: Verdiperstat Administration: Oral Dose: 600mg twice daily
• DRUG: CNM-Au8 — Drug: CNM-Au8 Administration: Oral Dose: 30 mg or 60 mg daily
• DRUG: Pridopidine — Drug: Pridopidine Administration: Oral Dose: 45mg twice daily
• DRUG: SLS-005 Trehalose — Drug: SLS-005 Trehalose Administration: Infusion Dose: 0.75 g/kg weekly

Primary Outcomes

• Disease Progression (36 Weeks)

Secondary Outcomes

• Respiratory Function (36 Weeks)
• Survival (36 Weeks)

Full Eligibility Criteria

Inclusion Criteria:

1. Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
2. Age 18 years or older.
3. Capable of providing informed consent and complying with study procedures, in the SI's opinion.
4. Time since onset of weakness due to ALS ≤ 24 months at the time of the Master Protocol Screening Visit.
5. Vital Capacity ≥ 50% of predicted capacity at the time of the Master Protocol Screening Visit measured by Slow Vital Capacity (SVC), or, if required due to pandemic-related restrictions, Forced Vital Capacity (FVC) measured in person.
6. Participants must either not take riluzole or be on a stable dose of riluzole for ≥ 30 days prior to the Master Protocol Screening Visit.
7. Participants must either not take edaravone or have completed at least one cycle (typically 14 days) of edaravone prior to the Master Protocol Screening Visit.
8. Participants must have the ability to swallow pills and liquids at the time of the Master Protocol Screening Visit and, in the SI's opinion, have the ability to swallow for the duration of the study.
9. Geographically accessible to the site.

Exclusion Criteria:

1. Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to SI's judgment (e.g., cardiovascular instability, systemic infection), or clinically significant laboratory abnormality or EKG changes. Clinically significant abnormal liver or kidney function is exclusionary. The following values \[alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 3 times the upper limit of normal (ULN) or estimated Glomerular Filtration Rate (eGFR) \< 30 mL/min/1.73m2\] are exclusionary regardless of clinical symptoms.
2. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.
3. Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.
4. Use of investigational treatments for ALS (off-label use or active participation in a clinical trial) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Master Protocol Screening Visit.
5. Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).
6. If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception, for the duration of the trial and for 3 months, or as specified in each RSA, after discontinuing study treatment.
7. If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 3 months, or as specified in each RSA, after discontinuing study treatment.
8. Anything that would place the participant at increased risk or preclude the participant's full compliance with or completion of the study, in the SI's opinion.
9. If a participant is being re-screened, the disqualifying condition has not been resolved, or the mandatory wash-out duration has not occurred.

Trial Locations

• Barrow Neurological Institute, Phoenix, Arizona, United States
• Mayo Clinic Scottsdale, Scottsdale, Arizona, United States
• University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States
• Loma Linda University Health, Loma Linda, California, United States
• Kaiser Permanente Los Angeles Medical Center, Los Angeles, California, United States
• University of Southern California, Los Angeles, California, United States
• Cedars-Sinai Medical Center, Los Angeles, California, United States
• University of California, Irvine, Orange, California, United States
• Forbes Norris MDA/ALS Research Center, California Pacific Medical Center, San Francisco, California, United States
• University of California, San Francisco, San Francisco, California, United States
• ...and 10 more locations

Frequently Asked Questions

Related Conditions

• ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neurodegenerative Disorders

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