HEALEY ALS Platform Trial - Master Protocol
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04297683|
Recruitment Status : Not yet recruiting
First Posted : March 5, 2020
Last Update Posted : June 19, 2020
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Zilucoplan Drug: Verdiperstat Drug: CNM-Au8||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.
In this trial, multiple investigational products for ALS will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen.
The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting.
Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized in a 3:1 ratio to either study drug or placebo.
The following regimens are active in the trial:
Regimen A - Zilucoplan Regimen B - Verdiperstat Regimen C - CNM-Au8
New regimens will be continuously added as new investigational products become available. The HEALEY ALS Platform Trial will enroll additional participants as each new regimen is available.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||480 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||As new investigational products become available, additional regimens will be added to the HEALEY ALS Platform Trial.|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||HEALEY ALS Platform Trial|
|Estimated Study Start Date :||June 2020|
|Estimated Primary Completion Date :||March 2021|
|Estimated Study Completion Date :||September 2021|
Experimental: Regimen A - Zilucoplan
Participants are randomized to receive either active zilucoplan or matching placebo.
Administration: Subcutaneous injection
Dose: Minimum of .0.22 mg/kg daily to a maximum dose of 0.42 mg/kg daily, dependent on weight
Experimental: Regimen B - Verdiperstat
Participants are randomized to receive either active verdiperstat or matching placebo.
Dose: 600mg twice daily
Experimental: Regimen C - CNM-Au8
Participants are randomized to receive either active CNM-Au8 or matching placebo.
Dose: 30 mg or 60 mg 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): NCT04297683
|Contact: HEALEY Center for ALS at Massachusetts General Hospital||833-425-8257 (HALT ALS)||email@example.com|
|Principal Investigator:||Merit Cudkowicz, MD||Massachusetts General Hospital|