Actively Recruiting

Phase 2
Phase 3
Age: 18Years +
All Genders
NCT04297683

HEALEY ALS Platform Trial - Master Protocol

Led by Merit E. Cudkowicz, MD · Updated on 2026-05-14

1500

Participants Needed

78

Research Sites

424 weeks

Total Duration

On this page

Sponsors

M

Merit E. Cudkowicz, MD

Lead Sponsor

M

Massachusetts General Hospital

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

HEALEY ALS Platform Trial - Master Protocol

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosed with sporadic or familial ALS as clinically possible, probable, lab-supported probable, or definite ALS by revised El Escorial criteria
  • Age 18 years or older
  • Able to provide informed consent and follow study procedures
  • Onset of weakness from ALS within 24 months before the screening visit
  • Vital capacity of 50% or higher of predicted capacity measured by Slow Vital Capacity or Forced Vital Capacity
  • Either not taking riluzole or on a stable riluzole dose for at least 30 days before screening
  • Either not taking edaravone or completed at least one cycle of edaravone before screening
  • Able to swallow pills and liquids at screening and expected to do so during the study
  • Geographically accessible to the study site
Not Eligible

You will not qualify if you...

  • Unstable medical conditions or significant lab abnormalities such as liver or kidney dysfunction
  • Unstable psychiatric disease, cognitive impairment, dementia, or substance abuse affecting consent ability
  • Active cancer or history of cancer except certain skin or curatively treated cancers with no recurrence for 3 years
  • Use of investigational ALS treatments within 5 half-lives or 30 days before screening
  • Any prior exposure to investigational gene therapies for ALS
  • For females, breastfeeding, pregnant, planning pregnancy, or unwilling to use contraception during and 3 months after the trial
  • For males with reproductive capacity, unwilling to use contraception during and 3 months after the trial
  • Any condition increasing risk or preventing full study compliance as judged by the investigator
  • Unresolved disqualifying conditions or insufficient wash-out period if re-screening

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 78 locations

1

Barrow Neurological Institute

Phoenix, Arizona, United States, 85013

Actively Recruiting

2

Mayo Clinic Scottsdale

Scottsdale, Arizona, United States, 85259

Actively Recruiting

3

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States, 72205

Actively Recruiting

4

Loma Linda University Health

Loma Linda, California, United States, 92354

Active, Not Recruiting

5

Kaiser Permanente Los Angeles Medical Center

Los Angeles, California, United States, 90027

Active, Not Recruiting

6

University of Southern California

Los Angeles, California, United States, 90033

Actively Recruiting

7

Cedars-Sinai Medical Center

Los Angeles, California, United States, 90048

Actively Recruiting

8

University of California, Irvine

Orange, California, United States, 92868

Active, Not Recruiting

9

Forbes Norris MDA/ALS Research Center, California Pacific Medical Center

San Francisco, California, United States, 94115

Actively Recruiting

10

University of California, San Francisco

San Francisco, California, United States, 94143

Actively Recruiting

11

University of Colorado

Aurora, Colorado, United States, 80045

Actively Recruiting

12

Hospital for Special Care

New Britain, Connecticut, United States, 06053

Actively Recruiting

13

Yale University

New Haven, Connecticut, United States, 06519

Actively Recruiting

14

Georgetown University

Washington D.C., District of Columbia, United States, 20007

Actively Recruiting

15

George Washington University

Washington D.C., District of Columbia, United States, 20037

Actively Recruiting

16

Nova Southeastern University

Davie, Florida, United States, 33024

Actively Recruiting

17

University of Florida

Gainesville, Florida, United States, 32610

Active, Not Recruiting

18

Mayo Clinic Florida

Jacksonville, Florida, United States, 32224

Actively Recruiting

19

University of Miami

Miami, Florida, United States, 33136

Active, Not Recruiting

20

University of South Florida

Tampa, Florida, United States, 33612

Actively Recruiting

21

Augusta University

Augusta, Georgia, United States, 30912

Active, Not Recruiting

22

Saint Alphonsus Regional Medical Center

Boise, Idaho, United States, 83704

Active, Not Recruiting

23

Northwestern University

Chicago, Illinois, United States, 60611

Actively Recruiting

24

University of Chicago

Chicago, Illinois, United States, 60637

Actively Recruiting

25

Indiana University Health

Indianapolis, Indiana, United States, 46202

Actively Recruiting

26

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States, 52242

Actively Recruiting

27

University of Kansas Medical Center

Fairway, Kansas, United States, 66205

Actively Recruiting

28

University of Kentucky

Lexington, Kentucky, United States, 40536

Actively Recruiting

29

Ochsner Health System

New Orleans, Louisiana, United States, 70115

Active, Not Recruiting

30

Johns Hopkins University

Baltimore, Maryland, United States, 21205

Active, Not Recruiting

31

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Actively Recruiting

32

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States, 02215

Actively Recruiting

33

University of Massachusetts Medical School

North Worcester, Massachusetts, United States, 01655

Actively Recruiting

34

University of Michigan

Ann Arbor, Michigan, United States, 48109

Actively Recruiting

35

Henry Ford Health System

Detroit, Michigan, United States, 48202

Actively Recruiting

36

Spectrum Health/Corewell Health

Grand Rapids, Michigan, United States, 49525

Actively Recruiting

37

Essentia Health

Duluth, Minnesota, United States, 55805

Actively Recruiting

38

University of Minnesota Medical School

Minneapolis, Minnesota, United States, 55455

Actively Recruiting

39

Mayo Clinic - Rochester

Rochester, Minnesota, United States, 55902

Actively Recruiting

40

University of Missouri Health Care

Columbia, Missouri, United States, 65212

Actively Recruiting

41

Saint Louis University

St Louis, Missouri, United States, 63104

Actively Recruiting

42

Washington University School of Medicine

St Louis, Missouri, United States, 63110

Actively Recruiting

43

Neurology Associates, P.C./Somnos Clinical Research

Lincoln, Nebraska, United States, 68506

Active, Not Recruiting

44

University of Nebraska Medical Center

Omaha, Nebraska, United States, 68198

Actively Recruiting

45

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, United States, 03756

Actively Recruiting

46

Hackensack University Medical Center

Hackensack, New Jersey, United States, 07601

Active, Not Recruiting

47

Dent Neurologic Institute

Amherst, New York, United States, 14226

Actively Recruiting

48

Mount Sinai

New York, New York, United States, 10003

Actively Recruiting

49

Columbia University

New York, New York, United States, 10032

Actively Recruiting

50

University of Rochester

Rochester, New York, United States, 14642

Actively Recruiting

51

Stony Brook University Hospital

Stony Brook, New York, United States, 11794

Actively Recruiting

52

SUNY Upstate

Syracuse, New York, United States, 13202

Actively Recruiting

53

University of North Carolina

Chapel Hill, North Carolina, United States, 27599

Active, Not Recruiting

54

Atrium Health

Charlotte, North Carolina, United States, 28207

Actively Recruiting

55

Duke University

Durham, North Carolina, United States, 27702

Actively Recruiting

56

Wake Forest Health Science

Winston-Salem, North Carolina, United States, 27157

Actively Recruiting

57

University of Cincinnati

Cincinnati, Ohio, United States, 45219

Actively Recruiting

58

Cleveland Clinic

Cleveland, Ohio, United States, 44195

Actively Recruiting

59

The Ohio State University

Columbus, Ohio, United States, 43221

Actively Recruiting

60

OhioHealth Research Institute

Westerville, Ohio, United States, 43082

Actively Recruiting

61

Providence Brain and Spine Institute ALS Center

Portland, Oregon, United States, 97213

Active, Not Recruiting

62

Lehigh Valley Health Network

Allentown, Pennsylvania, United States, 18103

Actively Recruiting

63

Penn State Hershey

Hershey, Pennsylvania, United States, 17033

Actively Recruiting

64

Jefferson Weinberg ALS Center, Thomas Jefferson University

Philadelphia, Pennsylvania, United States, 19107

Actively Recruiting

65

University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19107

Actively Recruiting

66

Lewis Katz School of Medicine at Temple University

Philadelphia, Pennsylvania, United States, 19140

Actively Recruiting

67

University of Pittsburg Medical Center

Pittsburgh, Pennsylvania, United States, 15232

Actively Recruiting

68

Vanderbilt University Medical Center

Nashville, Tennessee, United States, 37232

Actively Recruiting

69

Austin Neuromuscular Clinic

Austin, Texas, United States, 78759

Actively Recruiting

70

Texas Neurology

Dallas, Texas, United States, 75214

Actively Recruiting

71

Houston Methodist

Houston, Texas, United States, 77030

Active, Not Recruiting

72

UTHSCSA

San Antonio, Texas, United States, 78229

Active, Not Recruiting

73

University of Utah

Salt Lake City, Utah, United States, 84132

Actively Recruiting

74

University of Virginia

Charlottesville, Virginia, United States, 22908

Actively Recruiting

75

Virginia Commonwealth University

Henrico, Virginia, United States, 23233

Active, Not Recruiting

76

Swedish Medical Center

Seattle, Washington, United States, 98122

Actively Recruiting

77

University of Washington

Seattle, Washington, United States, 98195

Actively Recruiting

78

Medical College of Wisconsin

Milwaukee, Wisconsin, United States, 53226

Actively Recruiting

Loading map...

Research Team

H

HEALEY Center for ALS at Massachusetts General Hospital

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

8

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HEALEY ALS Platform Trial - Master Protocol | DecenTrialz