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HEALEY ALS Platform Trial - Regimen A Zilucoplan

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ClinicalTrials.gov Identifier: NCT04436497
Recruitment Status : Enrolling by invitation
First Posted : June 18, 2020
Last Update Posted : October 30, 2020
Sponsor:
Collaborator:
Ra Pharmaceuticals
Information provided by (Responsible Party):
Merit E. Cudkowicz, MD, Massachusetts General Hospital

Brief 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.


Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Zilucoplan Drug: Matching Placebo Phase 2 Phase 3

Detailed 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: HEALEY ALS Platform Trial - Regimen A Zilucoplan
Actual Study Start Date : July 29, 2020
Estimated Primary Completion Date : October 2021
Estimated Study Completion Date : December 2022


Arm Intervention/treatment
Experimental: Zilucoplan

Drug: Zilucoplan Administration: Subcutaneous injection

Dosage: Minimum of .0.22 mg/kg daily to a maximum dose of 0.42 mg/kg daily, dependent on weight

Drug: Zilucoplan

Drug: Zilucoplan Administration: Subcutaneous injection

Dosage: Minimum of .0.22 mg/kg daily to a maximum dose of 0.42 mg/kg daily, dependent on weight


Placebo Comparator: Matching Placebo

Administration: Subcutaneous injection

Dosage: Daily subcutaneous injection

Drug: Matching Placebo

Drug: Matching Placebo

Administration: Subcutaneous injection

Dosage: Daily subcutaneous injection





Primary Outcome Measures :
  1. Disease Progression [ Time Frame: 24 Weeks ]
    Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function 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.


Secondary Outcome Measures :
  1. Respiratory Function [ Time Frame: 24 Weeks ]
    Change in respiratory function over time as measured by Slow Vital Capacity (SVC).

  2. Muscle Strength [ Time Frame: 24 Weeks ]
    Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).

  3. Survival [ Time Frame: 24 Weeks ]
    Comparison of rate of occurrence between groups.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04436497


Locations
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United States, Massachusetts
Healey Center for ALS at Mass General
Boston, Massachusetts, United States, 02114
Sponsors and Collaborators
Merit E. Cudkowicz, MD
Ra Pharmaceuticals
Investigators
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Principal Investigator: Merit Cudkowicz Massachusetts General Hospital
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Responsible Party: Merit E. Cudkowicz, MD, Chief, Neurology Department, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT04436497    
Other Study ID Numbers: 2019P003518A
First Posted: June 18, 2020    Key Record Dates
Last Update Posted: October 30, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Merit E. Cudkowicz, MD, Massachusetts General Hospital:
ALS
Placebo-Controlled
Double-Blind
Regimen-Specific Appendix
Lou Gehrig's Disease
Zilucoplan
Ra Pharmaceuticals
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases