HEALEY ALS Platform Trial - Master Protocol

April 12, 2024 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

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 A - Zilucoplan Regimen B - Verdiperstat Regimen C - CNM-Au8 Regimen D - Priodopidine Regimen E - SLS-005 Trehalose Regimen F - ABBV-CLS-7262 Regimen G - DNL343

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 Contact

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
      • Scottsdale, Arizona, United States, 85259
        • Recruiting
        • Mayo Clinic Scottsdale
        • Contact:
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • Recruiting
        • University of Arkansas for Medical Sciences
        • Contact:
    • California
      • Loma Linda, California, United States, 92354
        • Recruiting
        • Loma Linda University Health
        • Contact:
      • Los Angeles, California, United States, 90048
      • Los Angeles, California, United States, 90033
      • Orange, California, United States, 92868
      • San Francisco, California, United States, 94143
      • San Francisco, California, United States, 94115
        • Recruiting
        • Forbes Norris MDA/ALS Research Center, California Pacific Medical Center
        • Contact:
    • Colorado
    • Connecticut
      • New Britain, Connecticut, United States, 06053
        • Recruiting
        • Hospital for Special Care
        • Contact:
      • New Haven, Connecticut, United States, 06519
        • Recruiting
        • Yale University
        • Contact:
    • District of Columbia
      • Washington, District of Columbia, United States, 20007
      • Washington, District of Columbia, United States, 20037
    • Florida
      • Davie, Florida, United States, 33024
        • Recruiting
        • Nova Southeastern University
        • Contact:
      • Gainesville, Florida, United States, 32610
      • Jacksonville, Florida, United States, 32224
      • Miami, Florida, United States, 33136
      • Tampa, Florida, United States, 33612
        • Recruiting
        • University of South Florida
        • Contact:
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Completed
        • Emory University
      • Augusta, Georgia, United States, 30912
    • Idaho
    • Illinois
      • Chicago, Illinois, United States, 60637
      • Chicago, Illinois, United States, 60611
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Indiana University Health
        • Contact:
    • Iowa
      • Iowa City, Iowa, United States, 52242
    • Kansas
      • Fairway, Kansas, United States, 66205
        • Recruiting
        • University of Kansas Medical Center
        • Contact:
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • Recruiting
        • University of Kentucky
        • Contact:
    • Louisiana
      • New Orleans, Louisiana, United States, 70115
        • Active, not recruiting
        • Ochsner Health System
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Recruiting
        • Johns Hopkins University
        • Contact:
      • Baltimore, Maryland, United States, 21201
    • Massachusetts
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
      • Detroit, Michigan, United States, 48202
        • Recruiting
        • Henry Ford Health System
        • Contact:
      • Grand Rapids, Michigan, United States, 49525
    • Minnesota
      • Duluth, Minnesota, United States, 55805
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesota/Twin Cities ALS Research Consortium
        • Contact:
      • Rochester, Minnesota, United States, 55902
    • Missouri
      • Columbia, Missouri, United States, 65212
        • Not yet recruiting
        • University of Missouri Health Care
      • Saint Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University School of Medicine
        • Contact:
      • Saint Louis, Missouri, United States, 63104
    • Nebraska
      • Lincoln, Nebraska, United States, 68506
        • Recruiting
        • Neurology Associates, P.C./Somnos Clinical Research
        • Contact:
      • Omaha, Nebraska, United States, 68198
    • Nevada
      • Las Vegas, Nevada, United States, 89145
        • Active, not recruiting
        • Las Vegas Clinic
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
    • New York
      • Amherst, New York, United States, 14226
      • New York, New York, United States, 10032
        • Active, not recruiting
        • Columbia University
      • Syracuse, New York, United States, 13202
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
      • Charlotte, North Carolina, United States, 28207
      • Durham, North Carolina, United States, 27702
      • Winston-Salem, North Carolina, United States, 27157
    • Ohio
      • Cincinnati, Ohio, United States, 45219
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic
        • Contact:
      • Columbus, Ohio, United States, 43221
    • Oregon
      • Portland, Oregon, United States, 97213
    • Pennsylvania
      • Allentown, Pennsylvania, United States, 18103
      • Hershey, Pennsylvania, United States, 17033
      • Philadelphia, Pennsylvania, United States, 19107
      • Philadelphia, Pennsylvania, United States, 19107
      • Philadelphia, Pennsylvania, United States, 19140
      • Pittsburgh, Pennsylvania, United States, 15232
        • Recruiting
        • University of Pittsburg Medical Center
        • Contact:
    • Tennessee
      • Nashville, Tennessee, United States, 37232
    • Texas
      • Dallas, Texas, United States, 75214
      • Houston, Texas, United States, 77030
      • San Antonio, Texas, United States, 78229
    • Utah
      • Salt Lake City, Utah, United States, 84132
    • Virginia
    • Washington
      • Seattle, Washington, United States, 98195
        • Recruiting
        • University of Washington
        • Contact:
      • Seattle, Washington, United States, 98122
        • Recruiting
        • Swedish Medical Center
        • Contact:
        • Contact:
          • Phone Number: 206-320-2609
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Medical College of Wisconsin
        • Contact:

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 ≤ 36 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.
  10. Participants must either not take Relyvrio/ Albrioza or have started Relyvrio/ Albrioza ≥ 30 days prior to the Master Protocol Screening Visit.

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, untreated thyroid dysfunction), 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.
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.
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.
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.
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.
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.
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.
Drug: DNL343

Drug: DNL343

Administration: Oral

Dose: Once per day

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Disease Progression
Time Frame: 24 Weeks
Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
24 Weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Respiratory Function
Time Frame: 24 Weeks
Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
24 Weeks
Muscle Strength
Time Frame: 24 Weeks
Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).
24 Weeks
Survival
Time Frame: 24 Weeks
Comparison of rate of occurrence between groups.
24 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

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)

July 14, 2020

Primary Completion (Estimated)

July 1, 2025

Study Completion (Estimated)

April 1, 2026

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)

April 16, 2024

Last Update Submitted That Met QC Criteria

April 12, 2024

Last Verified

April 1, 2024

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

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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