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RECRUITING

NCT04297683

PHASE2/PHASE3

HEALEY ALS Platform Trial - Master Protocol

Sponsor: Merit E. Cudkowicz, MD

View on ClinicalTrials.gov

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.

Official title: HEALEY ALS Platform Trial

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

1500

Start Date

2020-06-14

Completion Date

2028-08

Last Updated

2026-04-09

Healthy Volunteers

Conditions

Amyotrophic Lateral Sclerosis

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

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.

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.

Locations (74)

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

Augusta University

Augusta, Georgia, United States

Saint Alphonsus Regional Medical Center

Boise, Idaho, United States

Northwestern University

Chicago, Illinois, United States

University of Chicago

Chicago, Illinois, United States

Indiana University Health

Indianapolis, Indiana, United States

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

University of Kansas Medical Center

Fairway, Kansas, United States

University of Kentucky

Lexington, Kentucky, United States

Ochsner Health System

New Orleans, Louisiana, United States

Johns Hopkins University

Baltimore, Maryland, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

University of Massachusetts Medical School

North Worcester, Massachusetts, United States

University of Michigan

Ann Arbor, Michigan, United States

Henry Ford Health System

Detroit, Michigan, United States

Spectrum Health/Corewell Health

Grand Rapids, Michigan, United States

Essentia Health

Duluth, Minnesota, United States

University of Minnesota Medical School

Minneapolis, Minnesota, United States

Mayo Clinic - Rochester

Rochester, Minnesota, United States

University of Missouri Health Care

Columbia, Missouri, United States

Saint Louis University

St Louis, Missouri, United States

Washington University School of Medicine

St Louis, Missouri, United States

Neurology Associates, P.C./Somnos Clinical Research

Lincoln, Nebraska, United States

University of Nebraska Medical Center

Omaha, Nebraska, United States

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, United States

Hackensack University Medical Center

Hackensack, New Jersey, United States

Dent Neurologic Institute

Amherst, New York, United States

Columbia University

New York, New York, United States

Stony Brook University Hospital

Stony Brook, New York, United States

SUNY Upstate

Syracuse, New York, United States

University of North Carolina

Chapel Hill, North Carolina, United States

Atrium Health

Charlotte, North Carolina, United States

Duke University

Durham, North Carolina, United States

Wake Forest Health Science

Winston-Salem, North Carolina, United States

University of Cincinnati

Cincinnati, Ohio, United States

Cleveland Clinic

Cleveland, Ohio, United States

The Ohio State University

Columbus, Ohio, United States

Providence Brain and Spine Institute ALS Center

Portland, Oregon, United States

Lehigh Valley Health Network

Allentown, Pennsylvania, United States

Penn State Hershey

Hershey, Pennsylvania, United States

Jefferson Weinberg ALS Center, Thomas Jefferson University

Philadelphia, Pennsylvania, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Lewis Katz School of Medicine at Temple University

Philadelphia, Pennsylvania, United States

University of Pittsburg Medical Center

Pittsburgh, Pennsylvania, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Texas Neurology

Dallas, Texas, United States

Houston Methodist

Houston, Texas, United States

UTHSCSA

San Antonio, Texas, United States

University of Utah

Salt Lake City, Utah, United States

University of Virginia

Charlottesville, Virginia, United States

Virginia Commonwealth University

Henrico, Virginia, United States

Swedish Medical Center

Seattle, Washington, United States

University of Washington

Seattle, Washington, United States

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Clinical Research Directory

HEALEY ALS Platform Trial - Master Protocol

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (74)