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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

Tracking Information
First Submitted Date  ICMJE June 1, 2020
First Posted Date  ICMJE June 18, 2020
Last Update Posted Date October 30, 2020
Actual Study Start Date  ICMJE July 29, 2020
Estimated Primary Completion Date October 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 17, 2020)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 17, 2020)
  • Respiratory Function [ Time Frame: 24 Weeks ]
    Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
  • Muscle Strength [ Time Frame: 24 Weeks ]
    Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).
  • Survival [ Time Frame: 24 Weeks ]
    Comparison of rate of occurrence between groups.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE HEALEY ALS Platform Trial - Regimen A Zilucoplan
Official Title  ICMJE HEALEY ALS Platform Trial - Regimen A Zilucoplan
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.

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE
  • 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

  • Drug: Matching Placebo

    Drug: Matching Placebo

    Administration: Subcutaneous injection

    Dosage: Daily subcutaneous injection

Study Arms  ICMJE
  • 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

    Intervention: Drug: Zilucoplan
  • Placebo Comparator: Matching Placebo

    Administration: Subcutaneous injection

    Dosage: Daily subcutaneous injection

    Intervention: Drug: Matching Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: June 17, 2020)
160
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2022
Estimated Primary Completion Date October 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04436497
Other Study ID Numbers  ICMJE 2019P003518A
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Merit E. Cudkowicz, MD, Massachusetts General Hospital
Study Sponsor  ICMJE Merit E. Cudkowicz, MD
Collaborators  ICMJE Ra Pharmaceuticals
Investigators  ICMJE
Principal Investigator: Merit Cudkowicz Massachusetts General Hospital
PRS Account Massachusetts General Hospital
Verification Date October 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP