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RECRUITINGPhase 2 / Phase 3INTERVENTIONAL

HEALEY ALS Platform Trial - Master Protocol

HEALEY ALS Platform Trial

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: 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 willing to sign a consent form 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. Who Should NOT Join This Trial: 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

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

DRUG

Verdiperstat

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

DRUG

CNM-Au8

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

DRUG

Pridopidine

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

DRUG

SLS-005 Trehalose

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

DRUG

ABBV-CLS-7262

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

DRUG

DNL343

Drug: DNL343 Administration: Oral Dose: Once per day

DRUG

NUZ-001

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

Locations (20)

Barrow Neurological Institute
Phoenix, Arizona, United States
Mayo Clinic Scottsdale
Scottsdale, Arizona, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Loma Linda University Health
Loma Linda, California, United States
Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, United States
University of Southern California
Los Angeles, California, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
University of California, Irvine
Orange, California, United States
Forbes Norris MDA/ALS Research Center, California Pacific Medical Center
San Francisco, California, United States
University of California, San Francisco
San Francisco, California, United States
University of Colorado
Aurora, Colorado, United States
Hospital for Special Care
New Britain, Connecticut, United States
Yale University
New Haven, Connecticut, United States
Georgetown University
Washington D.C., District of Columbia, United States
George Washington University
Washington D.C., District of Columbia, United States
Nova Southeastern University
Davie, Florida, United States
University of Florida
Gainesville, Florida, United States
Mayo Clinic Florida
Jacksonville, Florida, United States
University of Miami
Miami, Florida, United States
University of South Florida
Tampa, Florida, United States