HEALEY ALS Platform Trial - Master Protocol
Purpose
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.
Condition
- Amyotrophic Lateral Sclerosis
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
- 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 Design
- Phase
- Phase 2/Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Intervention Model Description
- As new investigational products become available, additional regimens will be added to the HEALEY ALS Platform Trial.
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Masking Description
- Research participants, care providers, investigators and site staff (i.e. outcome assessors) will not be blinded to the regimen assignment, but they will be blinded to active product or matching placebo assignment and this blind will be maintained throughout the study. Quadruple masking remains consistent across all regimens which may start at different time points.
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Regimen A - Zilucoplan |
Participants are randomized to receive either active zilucoplan or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
|
|
Experimental Regimen B - Verdiperstat |
Participants are randomized to receive either active verdiperstat or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
|
|
Experimental Regimen C - CNM-Au8 |
Participants are randomized to receive either active CNM-Au8 or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
|
|
Experimental Regimen D - Pridopidine |
Participants are randomized to receive either active Pridopidine or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
|
|
Experimental Regimen E - SLS-005 Trehalose |
Participants are randomized to receive either active SLS-005 Trehalose or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
|
|
Experimental Regimen F- ABBV-CLS-7262 |
Participants are randomized to receive either active ABBV-CLS-7262 or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
|
|
Experimental Regimen G - DNL343 |
Participants are randomized to receive either active DNL343 or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
|
|
Experimental Regimen I - NUZ-001 |
Participants are randomized to receive either active NUZ-001 or matching placebo. |
|
Recruiting Locations
Barrow Neurological Institute
Phoenix, Arizona 85013
Phoenix, Arizona 85013
Mayo Clinic Scottsdale
Scottsdale, Arizona 85259
Scottsdale, Arizona 85259
University of Southern California
Los Angeles, California 90033
Los Angeles, California 90033
Forbes Norris MDA/ALS Research Center, California Pacific Medical Center
San Francisco, California 94115
San Francisco, California 94115
University of California, San Francisco
San Francisco, California 94143
San Francisco, California 94143
University of Colorado
Aurora, Colorado 80045
Aurora, Colorado 80045
Hospital for Special Care
New Britain, Connecticut 06053
New Britain, Connecticut 06053
Yale University
New Haven, Connecticut 06519
New Haven, Connecticut 06519
Georgetown University
Washington D.C., District of Columbia 20007
Washington D.C., District of Columbia 20007
Nova Southeastern University
Davie, Florida 33024
Davie, Florida 33024
Northwestern University
Chicago, Illinois 60611
Chicago, Illinois 60611
Indiana University Health
Indianapolis, Indiana 46202
Indianapolis, Indiana 46202
University of Iowa Hospitals and Clinics
Iowa City, Iowa 52242
Iowa City, Iowa 52242
University of Kansas Medical Center
Fairway, Kansas 66205
Fairway, Kansas 66205
Massachusetts General Hospital
Boston, Massachusetts 02114
Boston, Massachusetts 02114
Beth Israel Deaconess Medical Center
Boston, Massachusetts 02215
Boston, Massachusetts 02215
University of Massachusetts Medical School
North Worcester, Massachusetts 01655
North Worcester, Massachusetts 01655
University of Michigan
Ann Arbor, Michigan 48109
Ann Arbor, Michigan 48109
Henry Ford Health System
Detroit, Michigan 48202
Detroit, Michigan 48202
Spectrum Health/Corewell Health
Grand Rapids, Michigan 49525
Grand Rapids, Michigan 49525
Essentia Health
Duluth, Minnesota 55805
Duluth, Minnesota 55805
University of Minnesota Medical School
Minneapolis, Minnesota 55455
Minneapolis, Minnesota 55455
Mayo Clinic - Rochester
Rochester, Minnesota 55902
Rochester, Minnesota 55902
University of Missouri Health Care
Columbia, Missouri 65212
Columbia, Missouri 65212
Saint Louis University
St Louis, Missouri 63104
St Louis, Missouri 63104
Washington University School of Medicine
St Louis, Missouri 63110
St Louis, Missouri 63110
University of Nebraska Medical Center
Omaha, Nebraska 68198
Omaha, Nebraska 68198
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire 03756
Lebanon, New Hampshire 03756
Dent Neurologic Institute
Amherst, New York 14226
Amherst, New York 14226
Atrium Health
Charlotte, North Carolina 28207
Charlotte, North Carolina 28207
Wake Forest Health Science
Winston-Salem, North Carolina 27157
Winston-Salem, North Carolina 27157
University of Cincinnati
Cincinnati, Ohio 45219
Cincinnati, Ohio 45219
The Ohio State University
Columbus, Ohio 43221
Columbus, Ohio 43221
OhioHealth Research Institute
Westerville, Ohio 43082
Westerville, Ohio 43082
Lehigh Valley Health Network
Allentown, Pennsylvania 18103
Allentown, Pennsylvania 18103
Penn State Hershey
Hershey, Pennsylvania 17033
Hershey, Pennsylvania 17033
Jefferson Weinberg ALS Center, Thomas Jefferson University
Philadelphia, Pennsylvania 19107
Philadelphia, Pennsylvania 19107
University of Pennsylvania
Philadelphia, Pennsylvania 19107
Philadelphia, Pennsylvania 19107
Lewis Katz School of Medicine at Temple University
Philadelphia, Pennsylvania 19140
Philadelphia, Pennsylvania 19140
University of Pittsburg Medical Center
Pittsburgh, Pennsylvania 15232
Pittsburgh, Pennsylvania 15232
Vanderbilt University Medical Center
Nashville, Tennessee 37232
Nashville, Tennessee 37232
Austin Neuromuscular Clinic
Austin, Texas 78759
Austin, Texas 78759
University of Utah
Salt Lake City, Utah 84132
Salt Lake City, Utah 84132
University of Virginia
Charlottesville, Virginia 22908
Charlottesville, Virginia 22908
Swedish Medical Center
Seattle, Washington 98122
Seattle, Washington 98122
University of Washington
Seattle, Washington 98195
Seattle, Washington 98195
Medical College of Wisconsin
Milwaukee, Wisconsin 53226
Milwaukee, Wisconsin 53226
More Details
- NCT ID
- NCT04297683
- Status
- Recruiting
- Sponsor
- Merit E. Cudkowicz, MD
Study Contact
HEALEY Center for ALS at Massachusetts General Hospital833-425-8257 (HALT ALS)
healeyalsplatform@mgh.harvard.edu
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.