HEALEY ALS Platform Trial - Master Protocol

July 17, 2026 updated by: Merit E. Cudkowicz, MD

HEALEY ALS Platform 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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

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. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial.

In this trial, multiple investigational products for ALS will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen.

The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting.

Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized in a 3:1 ratio to either study drug or placebo.

The following regimens are active in the trial:

Regimen I - NUZ-001

New regimens will be continuously added as new investigational products become available. The HEALEY ALS Platform Trial will enroll additional participants as each new regimen is available.

Study Type

Interventional

Enrollment (Estimated)

1500

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Barrow Neurological Institute
      • Scottsdale, Arizona, United States, 85259
        • Mayo Clinic Scottsdale
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • University of Arkansas for Medical Sciences
    • California
      • La Jolla, California, United States, 92037
        • UC San Diego
      • Loma Linda, California, United States, 92354
        • Loma Linda University Health
      • Los Angeles, California, United States, 90033
        • University of Southern California
      • Los Angeles, California, United States, 90048
        • Cedars-Sinai Medical Center
      • Los Angeles, California, United States, 90027
        • Kaiser Permanente Los Angeles Medical Center
      • Orange, California, United States, 92868
        • University of California, Irvine
      • San Francisco, California, United States, 94143
        • University of California, San Francisco
      • San Francisco, California, United States, 94115
        • Forbes Norris MDA/ALS Research Center, California Pacific Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado
    • Connecticut
      • New Britain, Connecticut, United States, 06053
        • Hospital for Special Care
      • New Haven, Connecticut, United States, 06519
        • Yale University
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20007
        • Georgetown University
      • Washington D.C., District of Columbia, United States, 20037
        • George Washington University
    • Florida
      • Davie, Florida, United States, 33024
        • Nova Southeastern University
      • Gainesville, Florida, United States, 32610
        • University of Florida
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic Florida
      • Miami, Florida, United States, 33136
        • University of Miami
      • Tampa, Florida, United States, 33612
        • University of South Florida
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Augusta University
    • Idaho
      • Boise, Idaho, United States, 83704
        • Saint Alphonsus Regional Medical Center
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
      • Chicago, Illinois, United States, 60637
        • University of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University Health
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
    • Kansas
      • Fairway, Kansas, United States, 66205
        • University of Kansas Medical Center
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • University of Kentucky
    • Louisiana
      • New Orleans, Louisiana, United States, 70115
        • Ochsner Health System
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Beth Israel Deaconess Medical Center
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
      • Burlington, Massachusetts, United States, 01805
        • Lahey Hospital and Medical Center
      • North Worcester, Massachusetts, United States, 01655
        • University of Massachusetts Medical School
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan
      • Detroit, Michigan, United States, 48202
        • Henry Ford Health System
      • Grand Rapids, Michigan, United States, 49525
        • Spectrum Health/Corewell Health
    • Minnesota
      • Duluth, Minnesota, United States, 55805
        • Essentia Health
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota Medical School
      • Rochester, Minnesota, United States, 55902
        • Mayo Clinic - Rochester
    • Missouri
      • Columbia, Missouri, United States, 65212
        • University of Missouri Health Care
      • St Louis, Missouri, United States, 63110
        • Washington University School of Medicine
      • St Louis, Missouri, United States, 63104
        • Saint Louis University
    • Nebraska
      • Lincoln, Nebraska, United States, 68506
        • Neurology Associates, P.C./Somnos Clinical Research
      • Omaha, Nebraska, United States, 68198
        • University of Nebraska Medical Center
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Dartmouth-Hitchcock Medical Center
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack University Medical Center
    • New York
      • Amherst, New York, United States, 14226
        • Dent Neurologic Institute
      • New York, New York, United States, 10032
        • Columbia University
      • New York, New York, United States, 10003
        • Mount Sinai
      • Rochester, New York, United States, 14642
        • University of Rochester
      • Stony Brook, New York, United States, 11794
        • Stony Brook University Hospital
      • Syracuse, New York, United States, 13202
        • SUNY Upstate
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • University of North Carolina
      • Charlotte, North Carolina, United States, 28207
        • Atrium Health
      • Durham, North Carolina, United States, 27702
        • Duke University
      • Winston-Salem, North Carolina, United States, 27157
        • Wake Forest Health Science
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • University of Cincinnati
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
      • Columbus, Ohio, United States, 43221
        • The Ohio State University
      • Westerville, Ohio, United States, 43082
        • OhioHealth Research Institute
    • Oregon
      • Portland, Oregon, United States, 97213
        • Providence Brain and Spine Institute ALS Center
    • Pennsylvania
      • Allentown, Pennsylvania, United States, 18103
        • Lehigh Valley Health Network
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Hershey
      • Philadelphia, Pennsylvania, United States, 19140
        • Lewis Katz School of Medicine at Temple University
      • Philadelphia, Pennsylvania, United States, 19107
        • University of Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Jefferson Weinberg ALS Center, Thomas Jefferson University
      • Pittsburgh, Pennsylvania, United States, 15232
        • University of Pittsburg Medical Center
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center
    • Texas
      • Austin, Texas, United States, 78759
        • Austin Neuromuscular Clinic
      • Dallas, Texas, United States, 75214
        • Texas Neurology
      • Houston, Texas, United States, 77030
        • Baylor College of Medicine
      • Houston, Texas, United States, 77030
        • Houston Methodist
      • San Antonio, Texas, United States, 78229
        • UTHSCSA
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • University of Virginia
      • Henrico, Virginia, United States, 23233
        • Virginia Commonwealth University
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington
      • Seattle, Washington, United States, 98122
        • Swedish Medical Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Regimen A - Zilucoplan

Participants are randomized to receive either active zilucoplan or matching placebo.

NO LONGER RECRUITING; RESULTS REPORTED.
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
Experimental: Regimen B - Verdiperstat

Participants are randomized to receive either active verdiperstat or matching placebo.

NO LONGER RECRUITING; RESULTS REPORTED.
Drug: Verdiperstat
Drug: Verdiperstat Administration: Oral Dose: 600mg twice daily
Experimental: Regimen C - CNM-Au8

Participants are randomized to receive either active CNM-Au8 or matching placebo.

NO LONGER RECRUITING; RESULTS REPORTED.
Drug: CNM-Au8
Drug: CNM-Au8 Administration: Oral Dose: 30 mg or 60 mg daily
Experimental: Regimen D - Pridopidine

Participants are randomized to receive either active Pridopidine or matching placebo.

NO LONGER RECRUITING; RESULTS REPORTED.
Drug: Pridopidine
Drug: Pridopidine Administration: Oral Dose: 45mg twice daily
Experimental: Regimen E - SLS-005 Trehalose

Participants are randomized to receive either active SLS-005 Trehalose or matching placebo.

NO LONGER RECRUITING; RESULTS REPORTED.
Drug: SLS-005 Trehalose
Drug: SLS-005 Trehalose Administration: Infusion Dose: 0.75 g/kg weekly
Experimental: Regimen F- ABBV-CLS-7262

Participants are randomized to receive either active ABBV-CLS-7262 or matching placebo.

NO LONGER RECRUITING; RESULTS REPORTED.
Drug: ABBV-CLS-7262
Drug: ABBV-CLS-7162 Administration: Oral Dose: Dose 1 or Dose 2
Experimental: Regimen G - DNL343

Participants are randomized to receive either active DNL343 or matching placebo.

NO LONGER RECRUITING; RESULTS REPORTED.
Drug: DNL343
Drug: DNL343 Administration: Oral Dose: Once per day
Experimental: Regimen I - NUZ-001
Participants are randomized to receive either active NUZ-001 or matching placebo. ACTIVE, NOT RECRUITING
Drug: NUZ-001
Drug: NUZ-001. Administration: Oral. Dose: Once per day.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Disease Progression
Time Frame: 36 Weeks
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival.
36 Weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Respiratory Function
Time Frame: 36 Weeks
Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
36 Weeks
Survival
Time Frame: 36 Weeks
Comparison of rate of occurrence between groups.
36 Weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Merit E. Cudkowicz, MD

Collaborators

Massachusetts General Hospital

Investigators

  • Principal Investigator: Merit Cudkowicz, MD, Massachusetts General Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 14, 2020

Primary Completion (Estimated)

July 1, 2027

Study Completion (Estimated)

August 1, 2028

Study Registration Dates

First Submitted

March 3, 2020

First Submitted That Met QC Criteria

March 4, 2020

First Posted (Actual)

March 5, 2020

Study Record Updates

Last Update Posted (Actual)

July 21, 2026

Last Update Submitted That Met QC Criteria

July 17, 2026

Last Verified

July 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2019P003518

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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