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Active, not recruiting Phase 2/3

HEALEY ALS Platform Trial - Master Protocol

NCT04297683 · tracked via the Priya Life Science Israel tracker
Sponsor
Merit E. Cudkowicz, MD
Phase
Phase 2/3
Started
2020-06-14
Last updated
2026-07-21

Condition(s) studied

Amyotrophic Lateral Sclerosis

Investigational drug(s) / intervention(s)

Zilucoplan →VerdiperstatCNM-Au8Pridopidine →SLS-005 TrehaloseABBV-CLS-7262 →DNL343NUZ-001

Zilucoplan: Drug: Zilucoplan Administration: Subcutaneous injection Dose: Minimum of .0.22 mg/kg daily to a maximum dose of 0.42 mg/kg daily, dependent on weight

Verdiperstat: Drug: Verdiperstat Administration: Oral Dose: 600mg twice daily

CNM-Au8: Drug: CNM-Au8 Administration: Oral Dose: 30 mg or 60 mg daily

Pridopidine: Drug: Pridopidine Administration: Oral Dose: 45mg twice daily

SLS-005 Trehalose: Drug: SLS-005 Trehalose Administration: Infusion Dose: 0.75 g/kg weekly

ABBV-CLS-7262: Drug: ABBV-CLS-7162 Administration: Oral Dose: Dose 1 or Dose 2

DNL343: Drug: DNL343 Administration: Oral Dose: Once per day

NUZ-001: Drug: NUZ-001. Administration: Oral. Dose: Once per day.

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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 ≤ 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: 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), 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.

Primary outcome measure(s)

  • Disease Progression — 36 Weeks
    Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival.

Trial sites (81)

FacilityCityRegionStatus
Barrow Neurological Institute Phoenix Arizona
Mayo Clinic Scottsdale Scottsdale Arizona
University of Arkansas for Medical Sciences Little Rock Arkansas
UC San Diego La Jolla California
Loma Linda University Health Loma Linda California
Kaiser Permanente Los Angeles Medical Center Los Angeles California
University of Southern California Los Angeles California
Cedars-Sinai Medical Center Los Angeles California
University of California, Irvine Orange California
Forbes Norris MDA/ALS Research Center, California Pacific Medical Center San Francisco California
University of California, San Francisco San Francisco California
University of Colorado Aurora Colorado
Hospital for Special Care New Britain Connecticut
Yale University New Haven Connecticut
Georgetown University Washington D.C. District of Columbia
George Washington University Washington D.C. District of Columbia
Nova Southeastern University Davie Florida
University of Florida Gainesville Florida
Mayo Clinic Florida Jacksonville Florida
University of Miami Miami Florida
University of South Florida Tampa Florida
Augusta University Augusta Georgia
Saint Alphonsus Regional Medical Center Boise Idaho
Northwestern University Chicago Illinois
University of Chicago Chicago Illinois
Indiana University Health Indianapolis Indiana
University of Iowa Hospitals and Clinics Iowa City Iowa
University of Kansas Medical Center Fairway Kansas
University of Kentucky Lexington Kentucky
Ochsner Health System New Orleans Louisiana
Johns Hopkins University Baltimore Maryland
Massachusetts General Hospital Boston Massachusetts
Beth Israel Deaconess Medical Center Boston Massachusetts
Lahey Hospital and Medical Center Burlington Massachusetts
University of Massachusetts Medical School North Worcester Massachusetts
University of Michigan Ann Arbor Michigan
Henry Ford Health System Detroit Michigan
Spectrum Health/Corewell Health Grand Rapids Michigan
Essentia Health Duluth Minnesota
University of Minnesota Medical School Minneapolis Minnesota

+ 41 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04297683 on ClinicalTrials.gov ↗ ← All trials in Israel