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
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|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||160 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
Verdiperstat is administered twice daily p.o. for 24 weeks.
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
Dose: two tablets twice daily
- 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.
- 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.
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
|United States, Massachusetts|
|Healey Center for ALS at Mass General|
|Boston, Massachusetts, United States, 02114|
|Principal Investigator:||Merit Cudkowicz, MD||Massachusetts General Hospital|