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

Recruiting patients with ALS

Study Name
Healey ALS Platform Trial
PI
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:

Procedures/Participation
Participation:

  • 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

Procedures:

  • 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.