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.
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 I - NUZ-001 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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
1,500
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 Administration: Oral Dose: 600mg twice daily
Drug: CNM-Au8 Administration: Oral Dose: 30 mg or 60 mg daily
Disease Progression
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival.
Time frame: 36 Weeks
Respiratory Function
Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
Time frame: 36 Weeks
Survival
Comparison of rate of occurrence between groups.
Time frame: 36 Weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Drug: Pridopidine Administration: Oral Dose: 45mg twice daily
Drug: SLS-005 Trehalose Administration: Infusion Dose: 0.75 g/kg weekly
Drug: ABBV-CLS-7162 Administration: Oral Dose: Dose 1 or Dose 2
Drug: DNL343 Administration: Oral Dose: Once per day
Drug: NUZ-001. Administration: Oral. Dose: Once per day.
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
UC San Diego
La Jolla, California, 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
...and 71 more locations