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HEALEY ALS Platform Trial - Regimen B Verdiperstat

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ClinicalTrials.gov Identifier: NCT04436510
Recruitment Status : Enrolling by invitation
First Posted : June 18, 2020
Last Update Posted : October 30, 2020
Sponsor:
Collaborator:
Biohaven Pharmaceuticals, Inc.
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 B will evaluate the safety and efficacy of a single study drug, verdiperstat, in participants with ALS.


Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Matching Placebo Drug: Verdiperstat 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 B - Verdiperstat, the participant will complete a screening visit to assess additional Regimen B eligibility criteria. Once Regimen B eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active Verdiperstat or matching placebo.

Regimen B will enroll by invitation as participants may not choose to enroll in Regimen B. 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 B.

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 B Verdiperstat
Actual Study Start Date : July 28, 2020
Estimated Primary Completion Date : October 2021
Estimated Study Completion Date : November 2022


Arm Intervention/treatment
Experimental: Verdiperstat
Verdiperstat is administered twice daily p.o. for 24 weeks.
Drug: Verdiperstat

Drug: Verdiperstat

Administration: Oral

Dose: 600mg twice daily


Placebo Comparator: Matching Placebo
Matching placebo is administered twice daily p.o. for 24 weeks.
Drug: Matching Placebo

Drug: Matching Placebo

Administration: Oral

Dose: two tablets twice daily





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:

  • No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).

Exclusion Criteria:

  • The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).

    1. Participants who are taking strong inhibitors of CYP1A2 (i.e., ciprofloxacin, enoxacin, fluvoxamine, zafirlukast) for chronic/long-term use defined as more than two weeks.
    2. Participants who are taking strong inhibitors of CYP3A4 (i.e., conivaptan, itraconazole, ketoconazole, posaconazole, troleandomycin, voriconazole, clarithromycin, diltiazem, idelalisib, nefazodone, and certain antiviral agents [cobicistat, danoprevir, ritonavir, elvitegravir, indinavir, lopinavir, paritaprevir, ombitasavir, dasabuvir, saquinavir, tipranavir, nelfinavir]) for chronic/long-term use defined as more than two weeks. Note: Topical antifungal use is not exclusionary. Participants should not consume large quantities of grapefruit juice (more than 8oz per day) on a regular basis.

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): NCT04436510


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
Biohaven Pharmaceuticals, Inc.
Investigators
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Principal Investigator: Merit Cudkowicz, MD Massachusetts General Hospital
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Responsible Party: Merit E. Cudkowicz, MD, Chief, Neurology Department, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT04436510    
Other Study ID Numbers: 2019P003518B
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
Verdiperstat
Biohaven 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