URGENT: The COVID-19 epidemic is severely impacting the progress of ALS research at the ALS Therapy Development Institute. If you are able to give, will you please provide an immediate gift to fund our lab?
Quick Info
Status
Currently Recruiting
Estimated Enrollment
480
Phase
2/3
Treatment Type
Multiple
Trial Type
Primary Investigator
Merit E. Cudkowicz, MD
Contact Information
Locations
United States, Arizona
Barrow Neurological Institute, Phoenix, AZ, 85013, United States
Contact: Jessie Duncan   fulton.research@dignityhealth.org
United States, California
Loma Linda University Health, Loma Linda, CA, 92354, United States
Contact: Imran Qasim   sqasim@llu.edu
Cedars-Sinai Medical Center, Los Angeles, CA, 90048, United States
Contact: Carolyn Prina   carolyn.prina@cshs.org
University of California, Irvine, Orange, CA, 92868, United States
Contact: Vivian Li   vcli1@hs.uci.edu
Forbes Norris MDA/ALS Research Center, California Pacific Medical Cente, San Francisco, CA, 94115, United States
Contact: Marguerite Engel   engelm@cpmcri.org
United States, Colorado
University of Colorado, Aurora, CO, 80045, United States
Contact: Brianna Blume   Brianna.Blume@CUAnschutz.edu
United States, Connecticut
Hospital for Special Care, New Britain, CT, 06053, United States
Contact: Natalie Schmitt   nschmitt@hfsc.org
United States, Florida
Phil Smith Neuroscience Institute at Holy Cross Hospital, Fort Lauderdale, FL, 33308, United States
Contact: Donovan Mott   donovan.mott@holy-cross.com
University of Florida, Gainesville, FL, 32610, United States
Contact: Jennifer Steshyn   jennifer.steshyn@neurology.ufl.edu
Mayo Clinic Florida, Jacksonville, FL, 32224, United States
Contact: Jany Paulett   Paulett.Jany@mayo.edu
University of Miami, Miami, FL, 33136, United States
Contact: Wendy Levy   888-413-9315   alsresearch@med.miami.edu
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
Contact: Arish Jamil   arish.jamil@emory.edu
United States, Illinois
Northwestern University, Chicago, IL, 60611, United States
Contact: Ben Joslin   ben.joslin@northwestern.edu
University of Chicago, Chicago, IL, 60637, United States
Contact: Shail Bhatnagar   sbhatnagar@neurology.bsd.uchicago.edu
United States, Iowa
University of Iowa Hospitals and Clinics, Iowa City, IA, 52242, United States
Contact: Jeri Sieren   jeri-sieren@uiowa.edu
United States, Kansas
University of Kansas Medical Center, Fairway, KS, 66205, United States
Contact: Andrew Heim   AHeim2@kumc.edu
United States, Kentucky
University of Kentucky, Lexington, KY, 40536, United States
Contact: Ghadah Karasneh   ghadah.karasneh@uky.edu
United States, Louisiana
Ochsner Health System, New Orleans, LA, 70115, United States
Contact: Kristina Brown   kristina.brown@ochsner.org
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
Contact: Kristen Riley   kriley15@jhmi.edu
United States, Massachusetts
Massachusetts General Hospital, Boston, MA, 02114, United States
Contact: Danny Hevert   617-643-3902   MGHsiteHealeyPlatform@mgh.harvard.edu
Beth Israel Deaconess Medical Center, Boston, MA, 02215, United States
Contact: Hilda Gutierrez   hgutier1@bidmc.harvard.edu
University of Massachusetts Medical School, North Worcester, MA, 01655, United States
Contact: Diane McKenna-Yasek   Diane.McKenna-Yasek@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: Anne Vallis   avallis1@hfhs.org
Spectrum Health, Grand Rapids, MI, 49525, United States
Contact: Nichole Roderique   nichole.roderique@spectrumhealth.org
United States, Minnesota
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: Valerie Ferment   ferm0016@umn.edu
United States, Missouri
University of Missouri Health Care, Columbia, MO, 65212, United States
Contact: Natalie Taylor   taylornat@health.missouri.edu
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: Hillary Herzog   herzog.hillary@wustl.edu
United States, Nebraska
Neurology Associates, P.C./Somnos Clinical Research, Lincoln, NE, 68506, United States
Contact: Desirae Eschiti   Desi@somnos.com
University of Nebraska Medical Center, Omaha, NE, 68198, United States
Contact: Renee Hogue   rhogue@unmc.edu
United States, New Hampshire
Dartmouth-Hitchcock Medical Center, Lebanon, NH, 03756, United States
Contact: Caren Saunders   Caren.H.Saunders@hitchcock.org
United States, New York
Columbia University, New York, NY, 10032, United States
Contact: Brixhilda Dedi   bd2577@cumc.columbia.edu
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: Lisa Harrison   Lisa.harrison@duke.edu
Wake Forest Health Science, Winston-Salem, NC, 27157, United States
Contact: Mozhdeh Marandi   mmarandi@wakehealth.edu
United States, Ohio
The Ohio State University, Columbus, OH, 43221, United States
Contact: Erin Cohen   erin.cohen@osumc.edu
United States, Oregon
Providence Brain and Spine Institute ALS Center, Portland, OR, 97213, United States
Contact: Arlena Cummings   arlena.cummings@providence.org
United States, Pennsylvania
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: Laura Oakley   laura.oakley@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
Contact: Kathleen Hatala   kathleen.hatala@tuhs.temple.edu
United States, South Carolina
Medical University of South Carolina, Charleston, SC, 29425, United States
Contact: John "Bo" Keller   kellej@musc.edu
United States, Tennessee
Wesley Neurology Clinic, Cordova, TN, 38018, United States
Contact: Cindy Benzel   cbenzel@wesleyneurology.com
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: Todd Morgan   tmorgan@texasneurology.com
Houston Methodist, Houston, TX, 77030, United States
Contact: Rachel Applegate   rgapplegate@houstonmethodist.org
UTHSCSA, San Antonio, TX, 78229, United States
Contact: Pamela Kittrell   kittrellp@uthscsa.edu
United States, Virginia
University of Virginia, Charlottesville, VA, 22908, United States
Contact: Mary Wagoner   MIW9B@hscmail.mcc.virginia.edu
United States, Washington
University of Washington, Seattle, WA, 98195, United States
Contact: Laura Sissons-Ross   lsissons@uw.edu
United States, Wisconsin
Medical College of Wisconsin, Milwaukee, WI, 53226, United States
Contact: Marie Mejaki   mmejaki@mcw.edu
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
≥ 50%
Months Since Onset
Number of months since first symptoms of ALS.
N/A
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?
Unknown
Update Notes
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 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)