Research Studies
Study Title
Healey Platform Trial
Enrollment Status
Active - Not Recruiting
Principal Investigators
Michael Benatar, MD, PhD
Study Type
Drug Trial
Goals
- 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:
Enrolling
ALS patients
Eligibility
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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
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:
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
Contact
Links
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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