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CompletedNCT05740813Updated Nov 14, 2025Results posted

HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262

A Phase 2/3 interventional study of ABBV-CLS-7262 Dose 1 and ABBV-CLS-7262 Dose 2 in Amyotrophic Lateral Sclerosis, sponsored by Merit E. Cudkowicz, MD. Completed at 1 site in United States. Open to participants aged 18 Years to 100 Years. Per ClinicalTrials.gov, last updated 2025-11-14.

Sponsored by Merit E. Cudkowicz, MD · Phase 2/3, Interventional, and Treatment

Phase
Phase 2/3
Study type
Interventional
Enrollment
310
Allocation
Randomized
Ages
18 Years to 100 Years
Sex
All
01

Study summary

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.

Read the detailed description

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.

02

Conditions studied

  • Amyotrophic Lateral Sclerosis

Keywords

  • ALS
  • Placebo-Controlled
  • Double-Blind
  • Regimen Specific Appendix
  • Lou Gehrig's Disease
  • ABBV-CLS-7262
  • Calico Life Sciences
03

In context

Amyotrophic Lateral Sclerosis

981 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 283 are open to participants now.

This study's enrollment of 310 is above the median of 36 across 667 interventional studies indexed under Amyotrophic Lateral Sclerosis.

Browse Amyotrophic Lateral Sclerosis studies →

Lead sponsor

Merit E. Cudkowicz, MD is the lead sponsor of 9 studies on the registry; none are open to participants now.

Of its 7 completed or terminated interventional studies of FDA-regulated products, 7 (100%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 100 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).

Exclusion criteria

Exclusion Criteria:

  • The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).

    1. Based on the metabolism of the compound, the concomitant use of certain inhibitors and inducers of cytochrome P450 enzymes.
    2. Any clinically significant ECG abnormalities.
    3. Clinically significant clinical laboratory abnormalities.
05

Study design

Phase
Phase 2 / Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
310 participants (actual)

Study arms

  • Experimental
    ABBV-CLS-7262 Dose 1

    Drug: ABBV-CLS-7262 Dose 1

  • Experimental
    ABBV-CLS-7262 Dose 2

    Drug: ABBV-CLS-7262 Dose 2

  • Placebo comparator
    Matching Placebo

    Drug: Matching Placebo

Interventions

  • DrugABBV-CLS-7262 Dose 1

    ABBV-CLS-7262 is administered orally once per day for 24 weeks.

  • DrugABBV-CLS-7262 Dose 2

    ABBV-CLS-7262 is administered orally once per day for 24 weeks.

  • DrugMatching Placebo

    Matching placebo is administered orally once per day for 24 weeks.

06

What researchers measure

Primary outcomes

  1. 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

  2. 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

Secondary outcomes

  1. 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

  2. Respiratory Function

    Change in respiratory function over time as measured by Slow Vital Capacity (SVC).

    Time frame: Baseline to 24 Weeks

  3. 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.

    Time frame: Baseline to 24 weeks

  4. Disease Progression Biomarker

    Change in log-transformed serum neurofilament light protein (NfL) concentration from baseline to Week 24.

    Time frame: Baseline to 24 Weeks

  5. 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

  6. 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

07

Results

Posted Nov 14, 2025

Participant flow

Participant flow — Overall Study
MilestoneABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
Started1557976
Completed1376671
Not completed18135

Outcome measures

PrimaryDisease 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
Reported as:
Mean · Points per month
Disease Progression as Assessed by the ALSFRS-R-Slope
Points per monthABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
Disease Progression as Assessed by the ALSFRS-R-Slope-1.00 ± 0.068-0.91 ± 0.078-0.95 ± 0.085
Statistical analysis
  • ABBV-CLS-7262 Dose 1 vs Matching Placebo · Bayesian shared-parameter mode · Disease rate ratio: 1.06 · 95% CI 0.850 to 1.324DDR \<1 imply slowing of disease progression by ABBV-CLS-7262 relative to placebo. Note: reported "Confidence Interval" is actually a Bayesian credible interval.
  • ABBV-CLS-7262 Dose 2 vs Matching Placebo · Bayesian shared-parameter model · Disease rate ratio: 0.96 · 95% CI 0.753 to 1.220DDR \<1 imply slowing of disease progression by ABBV-CLS-7262 relative to placebo. Note: reported "Confidence Interval" is actually a Bayesian credible interval.
SecondaryFunction 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
Reported as:
Least squares mean · Points
Function by ALSFRS-R Total Score
PointsABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
Function by ALSFRS-R Total Score-6.110 ± 0.3710-5.468 ± 0.5252-5.563 ± 0.4106
Statistical analysis
  • ABBV-CLS-7262 Dose 1 vs Matching Placebo · Mixed Models Analysis · p = 0.3325 · Mean difference (net): -0.547 · 95% CI -1.657 to 0.562ABBV-CLS-7262 Dose 1 24-week change from baseline relative to placebo 24-week change from baseline.
  • ABBV-CLS-7262 Dose 2 vs Matching Placebo · Mixed Models Analysis · p = 0.8878 · Mean difference (net): 0.095 · 95% CI -1.230 to 1.420ABBV-CLS-7262 Dose 2 24-week change from baseline relative to placebo 24-week change from baseline.
SecondaryRespiratory Function

Change in respiratory function over time as measured by Slow Vital Capacity (SVC).

Time frame:
Baseline to 24 Weeks
Reported as:
Least squares mean · 24-week diff. of percents of normal VC
Respiratory Function
24-week diff. of percents of normal VCABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
Respiratory Function-10.882 ± 1.3311-7.202 ± 1.9469-9.462 ± 1.5259
Statistical analysis
  • ABBV-CLS-7262 Dose 1 vs Matching Placebo · Mixed Models Analysis · p = 0.5008 · Mean difference (net): -1.420 · 95% CI -5.563 to 2.723ABBV-CLS-7262 Dose 1 24-week change from baseline relative to placebo 24-week change from baseline.
  • ABBV-CLS-7262 Dose 2 vs Matching Placebo · Mixed Models Analysis · p = 0.3721 · Mean difference (net): 2.261 · 95% CI -2.714 to 7.236ABBV-CLS-7262 Dose 2 24-week change from baseline relative to placebo 24-week change from baseline.
PrimaryMortality 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
Reported as:
Mean · Events per month
Mortality Event Rate
Events per monthABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
Mortality Event Rate0.009 ± 0.00220.008 ± 0.00210.009 ± 0.0022
SecondaryUpper 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.

Time frame:
Baseline to 24 weeks
Reported as:
Least squares mean · Percent change
Upper Limb Muscle Strength
Percent changeABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
Upper Limb Muscle Strength-36.281 ± 2.6896-25.760 ± 3.8365-38.078 ± 3.0500
Statistical analysis
  • ABBV-CLS-7262 Dose 1 vs Matching Placebo · Mixed Models Analysis · p = 0.6707 · Mean difference (net): 1.796 · 95% CI -6.506 to 10.099ABBV-CLS-7262 Dose 1 24-week change from baseline relative to placebo 24-week change from baseline.
  • ABBV-CLS-7262 Dose 2 vs Matching Placebo · Mixed Models Analysis · p = 0.0144 · Mean difference (net): 12.318 · 95% CI 2.463 to 22.172ABBV-CLS-7262 Dose 2 24-week change from baseline relative to placebo 24-week change from baseline.
SecondaryDisease Progression Biomarker

Change in log-transformed serum neurofilament light protein (NfL) concentration from baseline to Week 24.

Time frame:
Baseline to 24 Weeks
Reported as:
Least squares mean · ln(ng/L)
Disease Progression Biomarker
ln(ng/L)ABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
Disease Progression Biomarker0.086 ± 0.02470.067 ± 0.0355-0.002 ± 0.0282
Statistical analysis
  • ABBV-CLS-7262 Dose 1 vs Matching Placebo · Mixed Models Analysis · p = 0.0253 · Mean difference (net): 1.092 · 95% CI 1.011 to 1.179Least squares mean difference between ABBV-CLS-7262 Dose 1 24-week change from baseline and placebo 24-week change from baseline.
  • ABBV-CLS-7262 Dose 2 vs Matching Placebo · Mixed Models Analysis · p = 0.1364 · Mean difference (net): 1.073 · 95% CI 0.978 to 1.175Least squares mean difference between ABBV-CLS-7262 Dose 2 24-week change from baseline and placebo 24-week change from baseline.
SecondaryActivities 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
Reported as:
Least squares mean · Points
Activities of Daily Living
PointsABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
Activities of Daily Living13.643 ± 1.296112.049 ± 1.861713.701 ± 1.5115
Statistical analysis
  • ABBV-CLS-7262 Dose 1 vs Matching Placebo · Mixed Models Analysis · p = 0.9782 · Mean difference (net): -0.058 · 95% CI -4.254 to 4.137ABBV-CLS-7262 Dose 1 24-week change from baseline relative to placebo 24-week change from baseline.
  • ABBV-CLS-7262 Dose 2 vs Matching Placebo · Mixed Models Analysis · p = 0.5108 · Mean difference (net): -1.652 · 95% CI -6.590 to 3.286ABBV-CLS-7262 Dose 2 24-week change from baseline relative to placebo 24-week change from baseline.
SecondaryNumber 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
Reported as:
Count of participants · Participants
Number of Participants That Experienced Death or Death Equivalent
ParticipantsABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
Number of Participants That Experienced Death or Death Equivalent845
Statistical analysis
  • ABBV-CLS-7262 Dose 1 vs Matching Placebo · Log Rank · p = 0.6481Dose 1. See Other Statistical Analysis for model adjustment details.
  • ABBV-CLS-7262 Dose 2 vs Matching Placebo · Log Rank · p = 0.6344Dose 2. See Other Statistical Analysis for model adjustment details.

Adverse events

Collected over Up to 35 weeks after participant signed Master Protocol consent. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
ABBV-CLS-7262 Dose 16/155 (3.9%)21/155 (13.5%)125/155 (80.6%)
ABBV-CLS-7262 Dose 23/79 (3.8%)7/79 (8.9%)59/79 (74.7%)
Matching Placebo1/76 (1.3%)6/76 (7.9%)62/76 (81.6%)
Most frequent serious events
Showing 10 of 32
Most frequent serious events
EventABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
PneumoniaInfections and infestations2/1552/790/76
Acute respiratory failureRespiratory, thoracic and mediastinal disorders3/1552/790/76
Complication associated with deviceGeneral disorders1/1551/791/76
Cholecystitis acuteHepatobiliary disorders0/1550/791/76
COVID-19Infections and infestations0/1550/791/76
Upper limb fractureInjury, poisoning and procedural complications0/1550/791/76
Pulmonary embolismRespiratory, thoracic and mediastinal disorders1/1550/791/76
Respiratory failureRespiratory, thoracic and mediastinal disorders2/1551/791/76
Device malfunctionProduct Issues2/1550/790/76
Cardiac ArrestCardiac disorders0/1551/790/76
Most frequent other events
Showing 10 of 32
Most frequent other events
EventABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching Placebo
FallInjury, poisoning and procedural complications47/15528/7931/76
Muscular weaknessNervous system disorders19/15513/7914/76
NeuromyopathyNervous system disorders11/15513/798/76
ConstipationGastrointestinal disorders23/15510/7912/76
HeadacheNervous system disorders21/1559/7912/76
DizzinessNervous system disorders11/1551/7910/76
DiarrhoeaGastrointestinal disorders17/1559/798/76
Post lumbar puncture syndromeInjury, poisoning and procedural complications12/1558/798/76
DyspnoeaRespiratory, thoracic and mediastinal disorders5/1557/798/76
FatigueGeneral disorders9/1556/797/76

Baseline characteristics

Baseline Analysis Population includes only placebo participants from the Regimen F Efficacy Regimen Only (ERO) sample; shared placebo participants from other regimens are not included in the Baseline Analysis Population.

Age, Continuous
Age, Continuous(Years)ABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching PlaceboTotal
Mean57.3 ± 11.258.2 ± 11.0258.0 ± 11.1357.7 ± 11.11
Sex: Female, Male
Sex: Female, Male(Participants)ABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching PlaceboTotal
Female603034124
Male954942186
Race (NIH/OMB)
Race (NIH/OMB)(Participants)ABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching PlaceboTotal
American Indian or Alaska Native0101
Asian41510
Native Hawaiian or Other Pacific Islander0000
Black or African American3036
White1457565285
More than one race0000
Unknown or Not Reported3238
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)ABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching PlaceboTotal
Hispanic or Latino128626
Not Hispanic or Latino1437170284
Unknown or Not Reported0000
ALS Diagnosis from R El Escorial Criteria
ALS Diagnosis from R El Escorial Criteria(Participants)ABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching PlaceboTotal
Clinically Possible ALS177731
Clinically Probable ALS - Laboratory Supported34101458
Clinically Probable ALS582833119
Clinically Definite ALS463422102
ALS Onset Location
ALS Onset Location(Participants)ABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching PlaceboTotal
Axial0202
Bulbar2781146
Generalized0101
Limb1256765257
Multiple1102
Respiratory2002
Time Since Symptom Onset at Baseline
Time Since Symptom Onset at Baseline(Months)ABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching PlaceboTotal
Mean22.2 ± 8.0719.6 ± 7.9419.7 ± 7.3820.93 ± 7.95
Delay in ALS Symptom Onset and Diagnosis
Delay in ALS Symptom Onset and Diagnosis(Months)ABBV-CLS-7262 Dose 1ABBV-CLS-7262 Dose 2Matching PlaceboTotal
Mean10.4 ± 5.569.3 ± 5.448.9 ± 4.749.71 ± 5.36

11 further baseline measures are reported on the registry.

08

Study locations

1 site
  • Healey Center for ALS at Mass General
    Boston, Massachusetts 02114, United States
09

References and documents

Study documents

  • Study protocol · Jul 23, 2024
  • Statistical analysis plan · Aug 13, 2024

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 14, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT05740813
Lead sponsor
Merit E. Cudkowicz, MD
Collaborators
Calico Life Sciences LLC
Responsible party
Merit E. Cudkowicz, MD (Chief, Neurology Department, Massachusetts General Hospital) — Sponsor-investigator
First posted
Feb 23, 2023
Start date
Mar 23, 2023
Primary completion
Oct 3, 2024
Completion
Oct 3, 2024
Results posted
Nov 14, 2025
Last update
Nov 14, 2025

Study contacts

Merit Cudkowicz, MD
principal investigator · Massachusetts General Hospital

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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