RecruitingPhase 2Phase 3NCT04297683

HEALEY ALS Platform Trial - Master Protocol

HEALEY ALS Platform Trial

Sponsor

Merit E. Cudkowicz, MD

Enrollment

1,500 participants

Start Date

Jun 14, 2020

Study Type

INTERVENTIONAL

Conditions

Amyotrophic Lateral Sclerosis

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.

Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

The HEALEY ALS Platform Trial is a large research program designed to test multiple potential treatments for ALS (Amyotrophic Lateral Sclerosis, also called Lou Gehrig's disease) at the same time. ALS is a progressive disease that damages nerve cells controlling movement, eventually making it hard to walk, talk, swallow, and breathe. This platform trial uses a shared screening process so patients can be considered for several experimental therapies without having to re-qualify for each one individually. Participants will be matched to one or more investigational treatments (called 'regimens') being studied within the platform. The study collects safety data and measures how well treatments slow disease progression. By testing multiple drugs simultaneously in a coordinated way, the trial aims to find effective treatments faster than traditional single-drug trials. You may be eligible if you: - Are 18 years of age or older - Have been diagnosed with ALS (possible, probable, or definite) - Had onset of muscle weakness due to ALS within the last 24 months - Have adequate lung function (Vital Capacity at least 50% of predicted) - Are able to swallow pills and liquids - Are geographically accessible to a participating site - Are either not taking riluzole or have been on a stable dose for at least 30 days You may NOT be eligible if you: - Have a significant unstable medical condition other than ALS (such as heart instability, active serious infection, or significant liver/kidney problems) - Have unstable psychiatric illness, cognitive impairment, or active substance abuse - Have active cancer or a history of cancer in the past 3 years (with some exceptions for minor skin cancers) - Have used other investigational ALS treatments within 30 days or 5 half-lives prior to screening - Have ever received a gene therapy for ALS - Are pregnant, breastfeeding, or unwilling to use contraception during the study Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGZilucoplan

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

DRUGVerdiperstat

Drug: Verdiperstat Administration: Oral Dose: 600mg twice daily

DRUGCNM-Au8

Drug: CNM-Au8 Administration: Oral Dose: 30 mg or 60 mg daily

DRUGPridopidine

Drug: Pridopidine Administration: Oral Dose: 45mg twice daily

DRUGSLS-005 Trehalose

Drug: SLS-005 Trehalose Administration: Infusion Dose: 0.75 g/kg weekly

DRUGABBV-CLS-7262

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

DRUGDNL343

Drug: DNL343 Administration: Oral Dose: Once per day

DRUGNUZ-001

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

Locations(78)

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

Mount Sinai

New York, New York, United States

Columbia University

New York, New York, United States

University of Rochester

Rochester, 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

OhioHealth Research Institute

Westerville, 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

Austin Neuromuscular Clinic

Austin, Texas, 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

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NCT04297683

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