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Quick Info
Currently Recruiting
2/ 3
Trial Type
Treatment Type
Start Date
Contact Information
United States, Arizona
Barrow Neurological Institute, Phoenix, AZ, 85013, United States
Contact: Jessie Duncan
United States, California
Loma Linda University Health, Loma Linda, CA, 92354, United States
Contact: David Borg
University of Southern California, Los Angeles, CA, 90033, United States
Contact: Salma Akhter
Cedars-Sinai Medical Center, Los Angeles, CA, 90048, United States
Contact: Sofia Mostowy
University of California, Irvine, Orange, CA, 92868, United States
Contact: Jeanette Overton
Forbes Norris MDA/ALS Research Center, California Pacific Medical Cente, San Francisco, CA, 94115, United States
Contact: Marguerite Engel
United States, Colorado
University of Colorado, Aurora, CO, 80045, United States
Contact: Brenna Baines
United States, Connecticut
Hospital for Special Care, New Britain, CT, 06053, United States
Contact: Natalie Cartwright
United States, Florida
Phil Smith Neuroscience Institute at Holy Cross Hospital, Fort Lauderdale, FL, 33308, United States
Contact: Donovan Mott
University of Florida, Gainesville, FL, 32610, United States
Contact: Jennifer Steshyn
Mayo Clinic Florida, Jacksonville, FL, 32224, United States
Contact: Jany Paulett
University of Miami, Miami, FL, 33136, United States
Contact: Wendy Levy   888-413-9315
University of South Florida, Tampa, FL, 33612, United States
Contact: Jessica Shaw
United States, Georgia
Emory University, Atlanta, GA, 30322, United States
Contact: Arish Jamil
United States, Illinois
Northwestern University, Chicago, IL, 60611, United States
Contact: Ben Joslin
University of Chicago, Chicago, IL, 60637, United States
Contact: Shail Bhatnagar
United States, Iowa
University of Iowa Hospitals and Clinics, Iowa City, IA, 52242, United States
Contact: Harika Anedal Kekinagath
United States, Kansas
University of Kansas Medical Center, Fairway, KS, 66205, United States
Contact: Katheryn Jennens
United States, Kentucky
University of Kentucky, Lexington, KY, 40536, United States
Contact: Ryan Blood
United States, Louisiana
Ochsner Health System, New Orleans, LA, 70115, United States
Contact: Lisa Edmond
United States, Maryland
University of Maryland, Baltimore, MD, 21201, United States
Contact: Vikram Nambiar
Johns Hopkins University, Baltimore, MD, 21205, United States
Contact: Kristen Riley
United States, Massachusetts
Massachusetts General Hospital, Boston, MA, 02114, United States
Contact: Danny Hevert   617-643-3902
Beth Israel Deaconess Medical Center, Boston, MA, 02215, United States
Contact: Hilda Gutierrez
University of Massachusetts Medical School, North Worcester, MA, 01655, United States
Contact: Catherine Douthwright
United States, Michigan
University of Michigan, Ann Arbor, MI, 48109, United States
Contact: Jayna Duell
Henry Ford Health System, Detroit, MI, 48202, United States
Contact: Beverley Duthie
Spectrum Health, Grand Rapids, MI, 49525, United States
Contact: Jessica Gallavin
United States, Minnesota
University of Minnesota/Twin Cities ALS Research Consortium, Minneapolis, MN, 55455, United States
Contact: Valerie Ferment
Mayo Clinic - Rochester, Rochester, MN, 55902, United States
Contact: Carol Denny
United States, Missouri
University of Missouri Health Care, Columbia, MO, 65212, United States
Contact: Natalie Taylor
Saint Louis University, Saint Louis, MO, 63104, United States
Contact: Susan Brown
Washington University School of Medicine, Saint Louis, MO, 63110, United States
Contact: Rebecca Livigni
United States, Nebraska
Neurology Associates, P.C./Somnos Clinical Research, Lincoln, NE, 68506, United States
Contact: Rachel Harper
University of Nebraska Medical Center, Omaha, NE, 68198, United States
Contact: Katelyn Hilz
United States, New Hampshire
Dartmouth-Hitchcock Medical Center, Lebanon, NH, 03756, United States
Contact: Allison Breen
United States, New York
Columbia University, New York, NY, 10032, United States
Contact: Brixhilda Dedi
SUNY Upstate, Syracuse, NY, 13202, United States
Contact: Lena Deb
United States, North Carolina
Duke University, Durham, NC, 27702, United States
Contact: Michelle Ward
Wake Forest Health Science, Winston-Salem, NC, 27157, United States
Contact: Mozhdeh Marandi
United States, Ohio
The Ohio State University, Columbus, OH, 43221, United States
Contact: Mackenzie Kaschalk
United States, Oregon
Providence Brain and Spine Institute ALS Center, Portland, OR, 97213, United States
Contact: Ashley Adamo
United States, Pennsylvania
Penn State Hershey, Hershey, PA, 17033, United States
Contact: Heidi Runk
Jefferson Weinberg ALS Center, Thomas Jefferson University, Philadelphia, PA, 19107, United States
Contact: Stephanie Hansen
University of Penn, Philadelphia, PA, 19107, United States
Contact: Adreeja Guharay
Lewis Katz School of Medicine at Temple University, Philadelphia, PA, 19140, United States
Contact: Kathleen Hatala
United States, Tennessee
Vanderbilt University Medical Center, Nashville, TN, 37232, United States
Contact: Diana Davis
United States, Texas
Texas Neurology, Dallas, TX, 75214, United States
Contact: Todd Morgan
Houston Methodist, Houston, TX, 77030, United States
Contact: Rachel Applegate
UTHSCSA, San Antonio, TX, 78229, United States
Contact: Pamela Kittrell
United States, Virginia
University of Virginia, Charlottesville, VA, 22908, United States
Contact: Mary Wagoner
United States, Washington
University of Washington, Seattle, WA, 98195, United States
Contact: Laura Sissons-Ross
United States, Wisconsin
Medical College of Wisconsin, Milwaukee, WI, 53226, United States
Contact: Marie Mejaki
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
≥ 50%
Months Since Onset
Number of months since first symptoms of ALS.
<36 months
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Open Label
Update Notes
New site updated
Site contact updated
Site contact updates
Site contacts updated
Updates to site contact information
New sites recruiting
New sites recruiting, regimen D now active
Site contacts updated
New sites recruiting
Now recruiting at some sites
Trial locations updated
Trial locations updated
Updated anticipated trial end date.
No significant changes.
No Significant Updates
New Trial Added

Other Information

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.
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 ≤ 36 months at the time of the Master Protocol Screening Visit.
5. Vital Capacity ≥ 50% of predicted capacity for age, height, and sex 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).
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. Riluzole-naïve participants are permitted in the study.
7. 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.
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, 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.
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 longer 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 longer 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.
10. 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.
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 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 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.
Trial Protocol as Published on
NCT04297683 (First Published: 3/3/2020)