HEALEY ALS Platform Trial - Regimen B Verdiperstat
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|ClinicalTrials.gov Identifier: NCT04436510|
Recruitment Status : Active, not recruiting
First Posted : June 18, 2020
Last Update Posted : June 28, 2022
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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)|
|Actual Enrollment :||167 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|
|Actual Primary Completion Date :||April 13, 2022|
|Estimated Study Completion Date :||April 2023|
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.
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.
|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
- 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.
- 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.
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|
|Responsible Party:||Merit E. Cudkowicz, MD, Chief, Neurology Department, Massachusetts General Hospital|
|Other Study ID Numbers:||
|First Posted:||June 18, 2020 Key Record Dates|
|Last Update Posted:||June 28, 2022|
|Last Verified:||June 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Regimen Specific Appendix
Lou Gehrig's Disease
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases