Help us determine the safety and efficacy of multiple experimental drugs in a single trial (one drug per participant)

Active - Not Recruiting

Study Name
Healey ALS Platform Trial
Volkan Granit, MD, MSc and Michael Benatar, MD, PhD
Study Type
Interventional (Platform Clinical Trial). Randomized. Placebo-controlled.
Eligible Population
Individuals affected with ALS, onset of weakness in the past 36 months, and breathing function (vital capacity) at least 50% of normal. (Other, study drug-specific, eligibility criteria may also apply.)
Study Purpose/Goals
To evaluate the safety and efficacy of multiple experimental agents compared to shared placebo, under the auspice of a single platform trial

  • Previous agents evaluated include:
    • Regimen A: Zilucoplan
    • Regimen B: Verdiperstat
    • Regimen C: Bioenergetic nanocatalysis (CNM-Au8-nanocrystaline gold)
    • Regimen D: pridopidine
    • Regimen E: Trehalose (SLS-005)
    • Regimen F: ABBV-CLS-7262
    • Regimen G: DNL343
  • Current agents evaluated include:


  • 28 weeks of participation
    • Screening: up to 4 weeks
    • Double-blind treatment: 24 weeks
  • Option to continue open label or re-randomize to receive a new experimental agent


  • Medical history and medication review
  • Drug dosing with compliance tracking
  • Breathing tests
  • Neuropsychological testing
  • Questionnaires/surveys
  • Physical exam
  • Neurological exam
  • Electrocardiogram
  • Blood and urine collection
  • Optional DNA and CSF collection

Join our family - together, we can change the course of this disease.

Join our family

Together, we can change the course of this disease.