HEALEY ALS Platform Trial - Regimen A Zilucoplan

Merit E. Cudkowicz, MD

Status and phase: Completed; Phase 3; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Zilucoplan; Drug: Matching Placebo

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT04436497

2019P003518A

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 162 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• The following inclusion criterion is in addition to the inclusion criteria specified in the Master Protocol (NCT NCT04297683).

  1. 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).

  1. History of meningococcal disease.
  2. Prior treatment with a complement inhibitor.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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162 participants in 2 patient groups, including a placebo group

Zilucoplan

Experimental group

Description:

Drug: Zilucoplan Administration: Subcutaneous injection Dosage: 0.3mg/kg administered daily

Treatment:

Drug: Zilucoplan

Matching Placebo

Placebo Comparator group

Description:

Administration: Subcutaneous injection Dosage: Daily subcutaneous injection

Treatment:

Drug: Matching Placebo