HEALEY ALS Platform Trial - Master Protocol
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|ClinicalTrials.gov Identifier: NCT04297683|
Recruitment Status : Recruiting
First Posted : March 5, 2020
Last Update Posted : December 8, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Zilucoplan Drug: Verdiperstat Drug: CNM-Au8 Drug: Pridopidine Drug: SLS-005 Trehalose||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 Regimen D - Priodopidine Regimen E - SLS-005 Trehalose
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 :||800 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|
|Actual Study Start Date :||July 14, 2020|
|Estimated Primary Completion Date :||April 2023|
|Estimated Study Completion Date :||December 2023|
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
Experimental: Regimen D - Pridopidine
Participants are randomized to receive either active Pridopidine or matching placebo.
Dose: 45mg twice daily
Experimental: Regimen E - SLS-005 Trehalose
Participants are randomized to receive either active SLS-005 Trehalose or matching placebo.
Drug: SLS-005 Trehalose
Drug: SLS-005 Trehalose
Dose: 0.75 g/kg weekly
- 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|
- Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
- Age 18 years or older.
- Capable of providing informed consent and complying with study procedures, in the SI's opinion.
- Time since onset of weakness due to ALS ≤ 36 months at the time of the Master Protocol Screening Visit.
- Vital Capacity ≥ 50% of predicted capacity for age, height, and sex at the time of the Master Protocol Screening Visit measured by Slow Vital Capacity (SVC), or, if required due to pandemic-related restrictions, Forced Vital Capacity (FVC).
- Participants must either not take riluzole or be on a stable dose of riluzole for ≥ 30 days prior to the Master Protocol Screening Visit. Riluzole-naïve participants are permitted in the study.
- Participants must either not take edaravone or have completed at least one cycle of edaravone prior to the Master Protocol Screening Visit. Edaravone-naïve participants are permitted in the study.
- Participants must have the ability to swallow pills and liquids at the time of the Master Protocol Screening Visit and, in the SI's opinion, have the ability to swallow for the duration of the study.
- Geographically accessible to the site.
Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to SI's judgment (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, or clinically significant laboratory abnormality or EKG changes).
Lab abnormalities include, but are not limited to: Hemoglobin < 10 g/dL, White Blood Cells < 3.0 x 103/mm3, Neutrophils, Absolute ≤ 1000/mm3, Eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter), low platelet counts (< 150 x 109 per liter), alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal (ULN), eGFR < 30 mL/min/1.73m2, thyroid-stimulating hormone (TSH) levels >10 mIU/L or <0.01 mIU/L.
- Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.
- Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.
- Use of investigational treatments for ALS (off-label use or active participation in a clinical trial) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Master Protocol Screening Visit.
- Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).
- If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception for the duration of the trial and for 3 months, or longer as specified in each RSA, after discontinuing study treatment.
- If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 3 months, or longer as specified in each RSA, after discontinuing study treatment.
- Anything that would place the participant at increased risk or preclude the participant's full compliance with or completion of the study, in the SI's opinion.
- If a participant is being re-screened, the disqualifying condition has not been resolved, or the mandatory wash-out duration has not occurred.
- For those participating in the optional CSF collection, contraindication to undergoing a lumbar puncture (LP) in the SI's opinion. Participants undergoing the LP must not be currently taking anticoagulation medications such as warfarin that would be a contraindication to LP; aspirin and non-steroidal anti-inflammatories are allowed.
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|
|Responsible Party:||Merit E. Cudkowicz, MD, Chief, Neurology Department, Massachusetts General Hospital|
|Other Study ID Numbers:||
|First Posted:||March 5, 2020 Key Record Dates|
|Last Update Posted:||December 8, 2022|
|Last Verified:||December 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|
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