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Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 2 / Phase 3INTERVENTIONAL

HEALEY ALS Platform Trial - Master Protocol

HEALEY ALS Platform Trial

HEALEY ALS Platform Trial - Master Protocol (NCT04297683) is a Phase 2 / Phase 3 interventional studying Amyotrophic Lateral Sclerosis, sponsored by Merit E. Cudkowicz, MD. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Amyotrophic Lateral Sclerosis and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 1,500 participants makes this one of the larger Amyotrophic Lateral Sclerosis trials currently registered. Trials at this scale are typically global, run across many sites, and designed to generate the definitive evidence package for an FDA approval submission or a label expansion.

Who May Be Eligible (Plain English)

Who May Qualify: 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 willing to sign a consent form 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. Who Should NOT Join This Trial: 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

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

DRUG

ABBV-CLS-7262

Drug: ABBV-CLS-7162 Administration: Oral Dose: Dose 1 or Dose 2

DRUG

DNL343

Drug: DNL343 Administration: Oral Dose: Once per day

DRUG

NUZ-001

Drug: NUZ-001. Administration: Oral. Dose: Once per day.

Locations (20)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

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
University of Colorado
Aurora, Colorado, United States
Hospital for Special Care
New Britain, Connecticut, United States
Yale University
New Haven, Connecticut, United States
Georgetown University
Washington D.C., District of Columbia, United States
George Washington University
Washington D.C., District of Columbia, United States
Nova Southeastern University
Davie, Florida, United States
University of Florida
Gainesville, Florida, United States
Mayo Clinic Florida
Jacksonville, Florida, United States
University of Miami
Miami, Florida, United States
University of South Florida
Tampa, Florida, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT04297683), the sponsor (Merit E. Cudkowicz, MD), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT04297683 clinical trial studying?

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. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT04297683?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT04297683?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT04297683. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT04297683. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.