HEALEY ALS Platform Trial

Study 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.

Study Status:

Recruiting

Disease:

Amyotrophic Lateral Sclerosis (ALS) , Familial ALS , Sporadic ALS

Study Type:

Interventional

Type of Intervention:

Drug Trial

Intervention Name:

Zilucoplan, Verdiperstat, CNM-Au8, Pridopidine, SLS-005 Trehalose, ABBV-CLS-7262, and DNL343.

Placebo:

Yes

Phase:

Phase 1/Phase 2

Study Chair(s)/Principal Investigator(s):

Merit Cudkowicz, MD Massachusetts General Hospital

Clinicaltrials.gov ID:

NCT04297683-NEALS

Neals Affiliated?

Yes

Coordinating Center Contact Information

Healey Center for ALS at Massachusetts General Hospital

Platform Trial Research Navigator / email hidden; JavaScript is required / 833-425-8257 (HALT ALS)

Boston, Massachusetts, 02114 United States

Full Study 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. 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 Sponsor:

Merit E. Cudkowicz, MD

Estimated Enrollment:

800

Estimated Study Start Date:

07 / 01 / 2020

Estimated Study Completion Date:

11 / 30 / 2022

Posting Last Modified Date:

11 / 29 / 2022

Date Study Added to neals.org:

03 / 09 / 2020

Visit the Sean M. Healey & AMG Center for ALS at Mass General website for more information: www.massgeneral.org/neurology/als/research/platform-trial

Minimum Age:

Can participants use Riluzole?

Yes

Inclusion Criteria:

Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
Age 18 years or older.
Capable of providing informed consent and complying with study procedures, in the SI's opinion.
Time since onset of weakness due to ALS ≤ 36 months at the time of the Master Protocol Screening Visit.
SVC ≥ 50% of predicted capacity for age, height, and sex at the time of the Master Protocol Screening Visit.
Participants must either not take riluzole or be on a stable dose of riluzole for ≥ 30 days prior to the Master Protocol Screening Visit. Riluzole-naïve participants are permitted in the study.
Participants must either not take edaravone or have completed at least one cycle of edaravone prior to the Master Protocol Screening Visit. Edaravone-naïve participants are permitted in the study.
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.
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, untreated thyroid dysfunction, or clinically significant laboratory abnormality or EKG changes). Lab abnormalities include, but are not limited to: Hemoglobin < 10 g/dL, White Blood Cells < 3.0 x 103/mm3, Neutrophils, Absolute ≤ 1000/mm3, Eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter), low platelet counts (< 150 x 109 per liter), alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal (ULN), eGFR < 30 mL/min/1.73m2, thyroid-stimulating hormone (TSH) levels >10 mIU/L or <0.01 mIU/L.
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.
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.
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.
Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).
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 longer as specified in each RSA, after discontinuing study treatment.
If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 3 months, or longer as specified in each RSA, after discontinuing study treatment.
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.
If a participant is being re-screened, the disqualifying condition has not been resolved, or the mandatory wash-out duration has not occurred.
For those participating in the optional CSF collection, contraindication to undergoing a lumbar puncture (LP) in the SI's opinion. Participants undergoing the LP must not be currently taking anticoagulation medications such as warfarin that would be a contraindication to LP; aspirin and non-steroidal anti-inflammatories are allowed.

Barrow Neurological Institute | Recruiting

Annalee Boyle / email hidden; JavaScript is required

Phoenix, Arizona 85013
United States

Mayo Clinic Scottsdale | Recruiting

Alissa Bojko / 480-301-4902 / email hidden; JavaScript is required

Scottsdale, Arizona
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Cedars-Sinai Medical Center | Recruiting

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Los Angeles, California 90048
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San Francisco, California
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Loma Linda University Health | Recruiting

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Loma Linda, California 92354
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Kaiser, Los Angeles Medical Center | Recruiting

Mary H. Berganza / email hidden; JavaScript is required

Los Angeles, California 90027
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Forbes Norris MDA/ALS Research Center, California Pacific Medical Cente | Recruiting

Daniela Sanchez / 415-600-1734 / email hidden; JavaScript is required

San Francisco, California 94115
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University of California, Irvine | Recruiting

Pola Gaid / email hidden; JavaScript is required

Orange, California 92868
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University of Southern California | Recruiting

Salma Akhter / 323-442-6221 / email hidden; JavaScript is required

Los Angeles, California 90033
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University of Colorado Anschutz | Recruiting

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Aurora, Colorado 80045
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Hospital for Special Care | Recruiting

Honora Dalamagas / 860-612-6356 / email hidden; JavaScript is required

New Britain, Connecticut 06053
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Georgetown University | Recruiting

Eli Wolfgang / email hidden; JavaScript is required

Washington, District of Columbia 20007
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George Washington University | Recruiting

Aspen Nelson / 202-741-2181 / email hidden; JavaScript is required

Washington, District of Columbia 20037
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Nova Southeastern University | Recruiting

Donovan Mott / 954-203-2829 / email hidden; JavaScript is required

Davie, Florida 33314
United States

University of Florida Gainesville | Active, Not recruiting

Jessica Spana / email hidden; JavaScript is required

Gainesville, Florida 32610
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Mayo Clinic Florida | Recruiting

Jany Paulett / email hidden; JavaScript is required

Jacksonville, Florida 32224
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University of Miami | Active, Not recruiting

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Miami, Florida 33136
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University of South Florida College of Medicine | Recruiting

Jessica Shaw / email hidden; JavaScript is required

Tampa, Florida 33612
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Augusta University | Recruiting

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Augusta, Georgia 30912
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Saint Alphonsus Regional | Recruiting

Jessica Kemmet / email hidden; JavaScript is required

Boise, Idaho
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Northwestern University | Recruiting

Emma Schmidt / email hidden; JavaScript is required

Chicago, Illinois 60611
United States

University of Chicago | Active, Not recruiting

Deekshitha Turaka / 773-702-4392 / email hidden; JavaScript is required

Chicago, Illinois 60637
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Indiana University | Recruiting

Sandy Guingrich / 317-963-7382 / email hidden; JavaScript is required

Indianapolis, Indiana 46202
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University of Iowa Hospitals and Clinics | Recruiting

Emily Anderson / email hidden; JavaScript is required

Iowa City, Iowa 52242
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Nick Staudenmier / email hidden; JavaScript is required

Fairway, Kansas 66205
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University of Kentucky | Recruiting

Sura Al Hilfi / 859-218-5064 / email hidden; JavaScript is required

Lexington, Kentucky 40536
United States

Ochsner Health System | Active, Not recruiting

Lisa Edmond / 985-989-7182 / email hidden; JavaScript is required

New Orleans, Louisiana 70115
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Johns Hopkins University | Recruiting

Kristen Riley / email hidden; JavaScript is required

Baltimore, Maryland 21205
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University of Maryland | Recruiting

N/A

Baltimore, Maryland 21201
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Beth Israel Deaconess Medical Center | Recruiting

Teresa Capella / email hidden; JavaScript is required

Boston, Massachusetts 02215
United States

Massachusetts General Hospital | Recruiting

Ludjie Jean Pierre / 617-643-3902 / email hidden; JavaScript is required

Boston, Massachusetts 02114
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University of Massachusetts Medical School | Recruiting

Catherine Douthwright / email hidden; JavaScript is required

North Worcester, Massachusetts 01655
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University of Michigan | Recruiting

Jayna Duell / email hidden; JavaScript is required

Ann Arbor, Michigan 48109
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Henry Ford Health System | Recruiting

Maria Stotland / 313-916-3359 / email hidden; JavaScript is required

Detroit, Michigan 48202
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Spectrum Health | Recruiting

Sehrish Khan / email hidden; JavaScript is required

Grand Rapids, Michigan 49525
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Mayo Clinic Rochester | Recruiting

Brenda Nelson / 507-293-0545 / email hidden; JavaScript is required

Rochester, Minnesota 55902
United States

University of Minnesota/Twin Cities ALS Research Consortium | Recruiting

Valerie Ferment / email hidden; JavaScript is required

Minneapolis, Minnesota 55455
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Essentia Health | Recruiting

Brent Gavin / 218-786-1220 / email hidden; JavaScript is required

Duluth, Minnesota
United States

Saint Louis University | Active, Not recruiting

Elizabeth (Bitsy) Egan / 314-977-4900 / email hidden; JavaScript is required

Saint Louis, Missouri 63104
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University of Missouri Health Care | Active, Not recruiting

N/A / N/A

Columbia, Missouri 65212
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Washington University School of Medicine | Recruiting

Rebecca Livigni / email hidden; JavaScript is required

Saint Louis, Missouri 63110
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University of Nebraska Medical Center | Recruiting

Rachel Harper / 402-552-6233 / email hidden; JavaScript is required

Omaha, Nebraska 68198
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Neurology Associates, P.C./Somnos Clinical Research | Active, Not recruiting

Desirae Eschiti / email hidden; JavaScript is required

Lincoln, Nebraska 68506
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Las Vegas Clinic | Active, Not-Recruiting

Kevin Daniels / 702-505-4230 / email hidden; JavaScript is required

Las Vegas, Nevada 89145
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Dartmouth-Hitchcock Medical Center | Active, Not recruiting

Gina Kersey / email hidden; JavaScript is required

Lebanon, New Hampshire 03756
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Hackensack University Medical Center | Recruiting

Rebecca Philip / email hidden; JavaScript is required

Michelle Williams / email hidden; JavaScript is required

Hackensack, New Jersey 07601
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Columbia University | Recruiting

Oneika Blackman / email hidden; JavaScript is required

New York, New York 10032
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SUNY Upstate | Recruiting

Lena Deb / email hidden; JavaScript is required

Syracuse, New York 13202
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Stony Brook University | Recruiting

Jas Kaur / 631-667-3098 / email hidden; JavaScript is required

Stony Brook, New York
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Duke University | Recruiting

Michelle Ward / email hidden; JavaScript is required

Durham, North Carolina 27702
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Wake Forest Health Science | Recruiting

Mozhdeh Marandi / email hidden; JavaScript is required

Winston-Salem, North Carolina 27157
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University of Cincinnati | Recruiting

Ashley Smith / email hidden; JavaScript is required

Cincinnati, Ohio
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Ohio State University | Recruiting

Ikram Hassan / email hidden; JavaScript is required

Columbus, Ohio 43221
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Cleveland Clinic | Recruiting

Irys Caristo / 216-444-0173 / email hidden; JavaScript is required

Cleaveland, Ohio 44195
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Providence Brain and Spine Institute ALS Center | Recruiting

Tyler Regan / email hidden; JavaScript is required

Portland, Oregon 97213
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University of Pennsylvania | Recruiting

Matthew Burst / email hidden; JavaScript is required

Philadelphia, Pennsylvania 19107
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Thomas Jefferson University, Jefferson Weinberg ALS Center | Recruiting

Gemma Krautzel / 267-582-6061 / email hidden; JavaScript is required

Philadelphia, Pennsylvania 19107
United States

University of Pittsburgh Medical Center | Recruiting

Research Coordinator / 412-864-2873 / email hidden; JavaScript is required

Pittsburgh, Pennsylvania 15232
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Temple University, Lewis Katz School of Medicine | Active, Not recruiting

Kathleen Hatala / email hidden; JavaScript is required

Philadelphia, Pennsylvania 19140
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Penn State Hershey | Recruiting

Michele Hare / email hidden; JavaScript is required

Hershey, Pennsylvania 17033
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Lehigh Valley Health Network | Recruiting

Andrew Orzel / 610-402-9543 / email hidden; JavaScript is required

Allentown, Pennsylvania 18103
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Vanderbilt University Medical Center | Recruiting

Diana Davis / email hidden; JavaScript is required

Nashville, Tennessee 37232
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Texas Neurology | Recruiting

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Dallas, Texas 75214
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Houston Methodist | Recruiting

Aramide Balogun / 346-238-8226 / email hidden; JavaScript is required

Houston, Texas 77030
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University of Texas Health Science Center at San Antonio (UTHSCSA) | Active, Not recruiting

Randee Kent-Baron / 210-450-0524 / email hidden; JavaScript is required

San Antonio, Texas 78229
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University of Utah | Recruiting

Crystal Neate / email hidden; JavaScript is required

Salt Lake City, Utah 84132
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Mary Wagoner / email hidden; JavaScript is required

Charlottesville, Virginia 22908
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Virginia Commonwealth University | Recruiting

Demetrius Carter / email hidden; JavaScript is required

Henrico, Virginia
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Swedish Medical Center | Recruiting

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Seattle, Washington
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University of Washington | Recruiting

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Seattle, Washington 98195
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Medical College of Wisconsin | Recruiting

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Milwaukee, Wisconsin 53226
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Yale University | Active, Recruiting

Erika Renkl / 475-254-9169 / email hidden; JavaScript is required

New Haven, Conneticut

Atrium Health (North Carolina) | Active, Recruiting

Cynthia Lary / 704-355-9434 / email hidden; JavaScript is required

Charlotte, North Carolina

University of North Carolina (North Carolina) | Active, Recruiting

Said Alhassan / 919-966-0515 / email hidden; JavaScript is required