HEALEY ALS Platform Trial - Regimen A Zilucoplan
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.
Regimen A will evaluate the safety and efficacy of a single study drug, zilucoplan, in participants with ALS.
Disease:Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS
Study Type:Interventional Trial
Study Category:Drug Trial
Study Chair(s)/Principal Investigator(s):
Merit Cudkowicz, Massachusetts General Hospital
Clinicaltrials.gov ID (11 digit #):NCT04436497
Coordinating Center Contact InformationHealey Center for ALS at Massachusetts General Hospital
Full Study Summary:
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. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683.
Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen.
If a participant is randomized to Regimen A - Zilucoplan, the participant will complete a screening visit to assess additional Regimen A eligibility criteria. Once Regimen A eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active zilucoplan or matching placebo.
Regimen A will enroll by invitation, as participants may not choose to enroll in Regimen A. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen A.
For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.
Study Sponsor:Merit E. Cudkowicz, MD
Estimated Study Start Date:06/30/2020
Estimated Study Completion Date:09/30/2021
Posting Last Modified Date:11/12/2020
Date Study Added to alsconsortium.org:07/06/2020
Please refer to the Sean M. Healey & AMG Center for ALS website for the list of trial sites: https://www.massgeneral.org/neurology/als/research/platform-trial-sites
Time since Symptom Onset:
Time since Diagnosis:
Can participants use Riluzole?Yes
The following inclusion criterion is in addition to the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
- Vaccination with a quadrivalent meningococcal vaccine and meningococcal serotype B vaccine at least 14 days prior to the first dose of study drug at the Baseline (Day 0) visit. Meningococcal vaccines (including boosters) should be administered in accordance with the study's vaccination worksheet.
The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
- History of meningococcal disease.
- Prior treatment with a complement inhibitor.
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