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Completed

NCT Number: NCT04436497

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.

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Key information

Conditions

Amyotrophic Lateral Sclerosis Central Nervous System Diseases Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Healey Center for ALS at Mass General

Boston, Massachusetts, 02114, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Zilucoplan

Drug

Drug: Zilucoplan Administration: Subcutaneous injection

Dosage: 0.3mg/kg administered daily

Matching Placebo

Drug

Drug: Matching Placebo

Administration: Subcutaneous injection

Dosage: Daily subcutaneous injection

Primary outcomes

  1. Disease Progression as Assessed by the ALSFRS-R Total Score

    Time frame: Baseline through 24 Weeks

    Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.

  2. Mortality Event Rate

    Time frame: Baseline through 24 Weeks

    Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.

Secondary outcomes

  1. Respiratory Function

    Time frame: Baseline and 24 Weeks

    Change in respiratory function as assessed by slow vital capacity (SVC).

  2. Muscle Strength

    Time frame: Baseline and 24 Weeks

    Change in muscle strength as measured isometrically using hand-held dynamometry (HHD).

  3. Number of Participants That Experienced Death or Death Equivalent

    Time frame: Baseline through 24 Weeks

    The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24 visit window (generally 175 days after baseline). The death equivalent criterion is use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.

Sponsors and collaborators

Lead sponsor

Merit E. Cudkowicz, MD

Other

Collaborators

  • Ra Pharmaceuticals

Registry information

Important dates

Study start
2020
Primary completion
2022
Study completion
2022
First posted
Jun 18, 2020
Registry last updated
Jul 25, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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