drugset / Trial / NCT04436497

HEALEY ALS Platform Trial - Regimen A Zilucoplan

NCT04436497 ↗

Phase 2/3 Completed 162 enrolled Merit E. Cudkowicz, MD Ra Pharmaceuticals · collab
RandomizedParallel-groupQuadruple-blindTreatment

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.

Timeline

Start
2020-07-29
Primary completion
2022-04-13
Completion
2022-05-04

Outcome

Missed primary endpoint

registry Disease Rate Ratio 1.08 (95% CI 0.874 to 1.307) NCT04436497 ↗

registry Posterior probability that the disease rate ratio (DRR) is less than 1 (i.e. zilucoplan slowed progression) was 0.2418. NCT04436497 ↗

Drugs

EvaluationDrugModalityDoseRoute
Subject Zilucoplan Peptide 0.3 mg/kg Subcutaneous