Phase 3 N=162 Randomized Quadruple-blind Treatment

HEALEY ALS Platform Trial - Regimen A Zilucoplan

Amyotrophic Lateral Sclerosis

Bottom Line

View on ClinicalTrials.gov: NCT04436497 ↗

Enrolled (actual)

162

Serious AEs

17.3%

Results posted

Jul 2023

Primary outcome: Primary: Disease Progression as Assessed by the ALSFRS-R Total Score — -1.11; -1.03 ALSFRS-R total score points per month

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Zilucoplan (Drug); Matching Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Merit E. Cudkowicz, MD
Primary completion: Apr 2022

Outcome Measures

Outcome	Result	p-value
PRIMARY Disease Progression as Assessed by the ALSFRS-R Total Score	-1.11; -1.03	—
PRIMARY Mortality Event Rate	0.009; 0.008	—
SECONDARY Respiratory Function	-9.66; -8.57	0.5495
SECONDARY Muscle Strength	-26.20; -25.16	0.7602
SECONDARY Number of Participants That Experienced Death or Death Equivalent	6; 6	0.6891

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. Regimen A will evaluate the safety and efficacy of a single study drug, zilucoplan, in participants with ALS.

Eligibility Criteria

Inclusion Criteria

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.

Exclusion Criteria

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04436497). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.