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Quick Info
Status
Currently Recruiting
Phase
2/ 3
Trial Type
Interventional
Treatment Type
Multiple
Randomization
3:1
Enrollment
800
Start Date
7/14/2020
Contact Information
Locations
United States, Arizona
Barrow Neurological Institute, Phoenix, AZ, 85013, United States
Contact: Annalee Boyle   fulton.research@dignityhealth.org
United States, California
Loma Linda University Health, Loma Linda, CA, 92354, United States
Contact: David Borg   dborg@llu.edu
University of Southern California, Los Angeles, CA, 90033, United States
Contact: Salma Akhter   salma.akhter@med.usc.edu
Cedars-Sinai Medical Center, Los Angeles, CA, 90048, United States
Contact: Sofia Mostowy   GroupNeuromuscularResearch@cshs.org
University of California, Irvine, Orange, CA, 92868, United States
Forbes Norris MDA/ALS Research Center, California Pacific Medical Cente, San Francisco, CA, 94115, United States
Contact: Marguerite Engel   engelm@cpmcri.org
University of California, San Francisco, San Francisco, CA, 94143, United States
Contact: Neha Madugala   neha.madugala@ucsf.edu
United States, Colorado
University of Colorado, Aurora, CO, 80045, United States
Contact: Brenna Baines   NeurologyResearchPartners@cuanschutz.edu
United States, Connecticut
Hospital for Special Care, New Britain, CT, 06053, United States
Contact: Natalie Cartwright   ncartwright@hfsc.org
United States, District of Columbia
Georgetown University, Washington, DC, 20007, United States
Contact: Eli Wolfgang   ew746@georgetown.edu
George Washington University, Washington, DC, 20037, United States
Contact: Grace Johnsonn   gjohnson@mfa.gwu.edu
United States, Florida
Nova Southeastern University, Davie, FL, 33024, United States
Contact: Donovan Mott   954-262-6387   Donovan.mott@nova.edu
Phil Smith Neuroscience Institute at Holy Cross Hospital, Fort Lauderdale, FL, 33308, United States
Contact: Ashley Stepler   954-542-3442   ashley.stepler@holy-cross.com
University of Florida, Gainesville, FL, 32610, United States
Mayo Clinic Florida, Jacksonville, FL, 32224, United States
Contact: Jany Paulett   Paulett.Jany@mayo.edu
University of Miami, Miami, FL, 33136, United States
University of South Florida, Tampa, FL, 33612, United States
Contact: Jessica Shaw   jessshaw@usf.edu
United States, Georgia
Emory University, Atlanta, GA, 30322, United States
Augusta University, Augusta, GA, 30912, United States
Contact: Brandy Quarles   706-721-0390   Neuromuscular_research@augusta.edu
United States, Idaho
Saint Alphonsus Regional Medical Center, Boise, ID, 83704, United States
Contact: Joshua Harrison   joshua.harrison@saintalphonsus.org
United States, Illinois
Northwestern University, Chicago, IL, 60611, United States
Contact: Emma Schmidt   emma.schmidt@northwestern.edu
University of Chicago, Chicago, IL, 60637, United States
United States, Indiana
Indiana University Health, Indianapolis, IN, 46202, United States
Contact: Sandy Guingrich   317-963-7382   sguingri@iu.edu
United States, Iowa
University of Iowa Hospitals and Clinics, Iowa City, IA, 52242, United States
Contact: Harika Anedal Kekinagath   harika-anedalkekinagath@uiowa.edu
United States, Kansas
University of Kansas Medical Center, Fairway, KS, 66205, United States
Contact: Katheryn Jennens   kjennens2@kumc.edu
United States, Kentucky
University of Kentucky, Lexington, KY, 40536, United States
United States, Louisiana
Ochsner Health System, New Orleans, LA, 70115, United States
United States, Maryland
University of Maryland, Baltimore, MD, 21201, United States
Contact: Vikram Nambiar   vnambiar@som.umaryland.edu
Johns Hopkins University, Baltimore, MD, 21205, United States
United States, Massachusetts
Massachusetts General Hospital, Boston, MA, 02114, United States
Contact: Mia Resendes   617-643-3902   MGHsiteHealeyPlatform@mgh.harvard.edu
Beth Israel Deaconess Medical Center, Boston, MA, 02215, United States
Contact: Hilda Gutierrez   BIDMCHealeyALSPlatformTrial@bidmc.harvard.edu
University of Massachusetts Medical School, North Worcester, MA, 01655, United States
Contact: Catherine Douthwright   Catherine.Douthwright@umassmed.edu
United States, Michigan
University of Michigan, Ann Arbor, MI, 48109, United States
Contact: Jayna Duell   jkballar@med.umich.edu
Henry Ford Health System, Detroit, MI, 48202, United States
Contact: Beverley Duthie   BDUTHIE1@hfhs.org
Spectrum Health, Grand Rapids, MI, 49525, United States
Contact: Jessica Gallavin   HealeyALS@spectrumhealth.org
United States, Minnesota
Essentia Health, Duluth, MN, 55805, United States
Contact: Brent Gavin   Brent.Gavin@essentiahealth.org
University of Minnesota/Twin Cities ALS Research Consortium, Minneapolis, MN, 55455, United States
Contact: Valerie Ferment   ferm0016@umn.edu
Mayo Clinic - Rochester, Rochester, MN, 55902, United States
Contact: Megan Means   means.megan@mayo.edu
United States, Missouri
University of Missouri Health Care, Columbia, MO, 65212, United States
Saint Louis University, Saint Louis, MO, 63104, United States
Contact: Susan Brown   susan.a.brown@health.slu.edu
Washington University School of Medicine, Saint Louis, MO, 63110, United States
Contact: Rebecca Livigni   als@wustl.edu
United States, Nebraska
Neurology Associates, P.C./Somnos Clinical Research, Lincoln, NE, 68506, United States
Contact: Rachel Harper   Desi@somnos.com
University of Nebraska Medical Center, Omaha, NE, 68198, United States
Contact: Katelyn Hilz   katelyn.hilz@unmc.edu
United States, Nevada
Las Vegas Clinic, Las Vegas, NV, 89145, United States
Contact: Richa Priya Jha   jha.r@lasvegasclinic.org
United States, New Hampshire
Dartmouth-Hitchcock Medical Center, Lebanon, NH, 03756, United States
United States, New York
Columbia University, New York, NY, 10032, United States
SUNY Upstate, Syracuse, NY, 13202, United States
Contact: Lena Deb   debl@upstate.edu
United States, North Carolina
Duke University, Durham, NC, 27702, United States
Contact: Michelle Ward   ALSResearch@duke.edu
Wake Forest Health Science, Winston-Salem, NC, 27157, United States
Contact: Mozhdeh Marandi   mmarandi@wakehealth.edu
United States, Ohio
University of Cincinnati, Cincinnati, OH, 45219, United States
Contact: Elizabeth Gehlmaem   gehlmaem@ucmail.uc.edu
Cleveland Clinic, Cleveland, OH, 44195, United States
Contact: Irys Caristo   caristi@ccf.org
The Ohio State University, Columbus, OH, 43221, United States
Contact: Elizabeth (Beth) Wiley   Elizabeth.Wiley@osumc.edu
United States, Oregon
Providence Brain and Spine Institute ALS Center, Portland, OR, 97213, United States
Contact: Ashley Adamo   ashley.adamo@providence.org
United States, Pennsylvania
Lehigh Valley Health Network, Allentown, PA, 18103, United States
Contact: Andrew Orzel   andrew.orzel@lvhn.org
Penn State Hershey, Hershey, PA, 17033, United States
Contact: Heidi Runk   hrunk@pennstatehealth.psu.edu
Jefferson Weinberg ALS Center, Thomas Jefferson University, Philadelphia, PA, 19107, United States
Contact: Mattison Worthy   mattison.worthy@jefferson.edu
University of Penn, Philadelphia, PA, 19107, United States
Contact: Adreeja Guharay   Adreeja.Guharay@pennmedicine.upenn.edu
Lewis Katz School of Medicine at Temple University, Philadelphia, PA, 19140, United States
University of Pittsburg Medical Center, Pittsburgh, PA, 15232, United States
Contact: Rebecca Molczan   molcrs@upmc.edu
United States, Tennessee
Vanderbilt University Medical Center, Nashville, TN, 37232, United States
Contact: Diana Davis   diana.davis@vumc.org
United States, Texas
Texas Neurology, Dallas, TX, 75214, United States
Contact: Mohamad Nasri, MD   mnasri@texasneurology.com
Houston Methodist, Houston, TX, 77030, United States
UTHSCSA, San Antonio, TX, 78229, United States
United States, Utah
University of Utah, Salt Lake City, UT, 84132, United States
Contact: Crystal Neate   crystal.neate@hsc.utah.edu
United States, Virginia
University of Virginia, Charlottesville, VA, 22908, United States
United States, Washington
Swedish Medical Center, Seattle, WA, 98122, United States
Contact: Janae Rahiman   janae.rahiman@swedish.org
University of Washington, Seattle, WA, 98195, United States
Contact: Amos Sahu   adsahu@uw.edu
United States, Wisconsin
Medical College of Wisconsin, Milwaukee, WI, 53226, United States
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?
N/A
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
N/A
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Yes
Open Label
Yes
Update Notes
New sites recruiting
12/8/2022
Site contacts updated
10/20/2022
Site contact updates, new sites recruiting
9/22/2022
Sites addedc
8/22/2022
Sites added
6/29/2022
New sites recruiting
4/29/2022
Sites added
3/23/2022
Recruiting, site updates
2/16/2022
Study no longer recruiting
2/4/2022
New site updated
10/22/2021
Site contact updated
8/31/2021
Site contact updates
8/6/2021
Site contacts updated
6/7/2021
Updates to site contact information
4/13/2021
New sites recruiting
3/17/2021
New sites recruiting, regimen D now active
12/29/2020
Site contacts updated
10/30/2020
New sites recruiting
9/25/2020
Now recruiting at some sites
8/31/2020
Trial locations updated
8/6/2020
Trial locations updated
7/22/2020
Updated anticipated trial end date.
6/22/2020
No significant changes.
6/2/2020
No Significant Updates
4/13/2020
New Trial Added
3/6/2020

Other Information

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.
Eligibility
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.
Details
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 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.
Collaborator(s)
Trial Protocol as Published on Clinicaltrials.gov
NCT04297683 (First Published: 3/3/2020)