Research Studies

Study Title

Healey Platform Trial

Enrollment Status


Principal Investigators

Michael Benatar, MD, PhD

Study Type

Drug Trial


  • 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:


ALS patients


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Clinical diagnosis of ALS
Onset of weakness within the past 36 months
Breathing function (vital capacity) at least 50% of normal
Regimen-specific eligibility criteria may also apply

Study Involvement

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Multiple study visits over a period of 28 weeks
Study procedures include:

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

Option to continue to open label or re-randomized to receive a new experimental agent

Primary Funding Source

Healey ALS Center

Related Publications

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Paganoni S, Berry JD, Quintana M, Macklin E, Saville BR, Detry MA, Chase M, Sherman AV, Yu H, Drake K, Andrews J, Shefner J, Chibnik LB, Vestrucci M, Cudkowicz ME; Healey ALS Platform Trial Study Group. Adaptive platform trials to transform amyotrophic lateral sclerosis therapy development. Ann Neurol. 2022 Feb;91(2):165-175. Epub 2022 Jan 10.

Related Presentations & Lectures

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