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 F will evaluate the safety and efficacy of a single study drug, ABBV-CLS-7262, in participants with ALS.
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 F ABBV-CLS-7262, the participant will complete a screening visit to assess additional Regimen F eligibility criteria. Once Regimen F eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in an overall 3:1 ratio to either active ABBV-CLS-7262 or matching placebo. The first 240 participants will be assigned in a 2:1:1 allocation ratio to Dose 1 ABBV-CLS-7262, Dose 2 ABBV-CLS-7262, or placebo. The final approximately 60 participants will be assigned in a 3:1 allocation ratio to Dose 1 ABBV-CLS-7262 or placebo. Regimen F will enroll by invitation, as participants may not choose to enroll in Regimen F. 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 F. For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
310
ABBV-CLS-7262 is administered orally once per day for 24 weeks.
ABBV-CLS-7262 is administered orally once per day for 24 weeks.
Matching placebo is administered orally once per day for 24 weeks.
Healey Center for ALS at Mass General
Boston, Massachusetts, United States
Disease Progression as Assessed by the ALSFRS-R-Slope
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4(normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Participants with higher scores have more physical function. Note that only participants who survived to their Week 24 visit contribute to the estimate.
Time frame: Baseline to 24 Weeks
Mortality Event Rate
Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.
Time frame: Baseline to 24 weeks
Function by ALSFRS-R Total Score
Change from baseline to Week 24 in function as assessed by ALSFRS-R total score.
Time frame: Baseline to 24 weeks
Respiratory Function
Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
Time frame: Baseline to 24 Weeks
Upper Limb Muscle Strength
Change in upper limb muscle strength over time as measured isometrically using hand-held dynamometry and grip strength, calculated as the average percent change from baseline of the following muscles/maneuvers: shoulder flexion, elbow flexion, elbow extension, wrist extension, abductor pollicis brevis contraction, abductor digiti minimi contraction, first dorsal interosseous contraction, and grip strength. Note that only those with measurable strength at baseline were included.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: Baseline to 24 weeks
Disease Progression Biomarker
Change in log-transformed serum neurofilament light protein (NfL) concentration from baseline to Week 24.
Time frame: Baseline to 24 Weeks
Activities of Daily Living
Change from baseline to Week 24 in the activities of daily living (ADL)/independence domain score as assessed by the Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40). The ALSAQ-40, a patient-self reported outcome, consists of 40 questions that are used to measure the subjective well-being of participants, and each question is scored from 0 (never) to 5 (always or cannot do at all). The ADL/independence domain score is based on 10 out of the 40 questions, with a maximum ADL/independence domain score of 50 and minimum score of 0. Higher domain scores indicate a worse subjective well-being or less independence completing ADLs.
Time frame: Baseline to 24 weeks
Number of Participants That Experienced Death or Death Equivalent
The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24visit window (generally 175 days after baseline). The death equivalent criterion is use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.
Time frame: Baseline to 24 weeks