Amyotrophic Lateral SclerosisPhase 2/3NCT05740813
HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262
A Phase 2/3 study testing ABBV-CLS-7262 Dose 1 and ABBV-CLS-7262 Dose 2 in people with Amyotrophic Lateral Sclerosis, run by Merit E. Cudkowicz, MD at 1 site in United States.
Trial 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 more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- ABBV-CLS-7262 Dose 1 and ABBV-CLS-7262 Dose 2
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 310 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Mar 2023to 3 Oct 2024
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
Who cannot join
- The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
- Based on the metabolism of the compound, the concomitant use of certain inhibitors and inducers of cytochrome P450 enzymes.
- Any clinically significant ECG abnormalities.
- Clinically significant clinical laboratory abnormalities.
Trial locations and contacts
1 site in 1 country.
United States1 site
Healey Center for ALS at Mass General
Boston, Massachusetts, 02114
About this study
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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- ABBV-CLS-7262 Dose 1, ABBV-CLS-7262 Dose 2, Matching Placebo
- Keywords
- ALS, Placebo-Controlled, Double-Blind, Regimen Specific Appendix, Lou Gehrig's Disease, ABBV-CLS-7262, Calico Life Sciences
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ABBV-CLS-7262 Dose 1Drug
ABBV-CLS-7262 is administered orally once per day for 24 weeks.
ABBV-CLS-7262 Dose 2Drug
ABBV-CLS-7262 is administered orally once per day for 24 weeks.
Matching PlaceboDrug
Matching placebo is administered orally once per day for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| ABBV-CLS-7262 Dose 1 | Experimental | Not described Receives: Drug: ABBV-CLS-7262 Dose 1 |
| ABBV-CLS-7262 Dose 2 | Experimental | Not described Receives: Drug: ABBV-CLS-7262 Dose 2 |
| Matching Placebo | Placebo comparator | Not described Receives: Drug: Matching Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Baseline to 24 weeks
Secondary outcomes (6)
Function by ALSFRS-R Total Score
Change from baseline to Week 24 in function as assessed by ALSFRS-R total score.
Measured: Baseline to 24 weeks
Respiratory Function
Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
Measured: 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.
Measured: Baseline to 24 weeks
Disease Progression Biomarker
Change in log-transformed serum neurofilament light protein (NfL) concentration from baseline to Week 24.
Measured: 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.
Measured: 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.
Measured: Baseline to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Feb 2023
- Study start23 Mar 2023
- Primary completion3 Oct 2024
- Study completion3 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 310 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merit E. Cudkowicz, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Calico Life Sciences LLC
- Responsible party
- Sponsor-investigator, Merit E. Cudkowicz, MD, Massachusetts General Hospital
- Investigators
- Merit Cudkowicz, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05740813
- Sponsor's protocol ID
- 2019P003518F
- Status last verified
- Oct 2025
- First submitted
- 12 Feb 2023
- Last updated
- 14 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262
- ClinicalTrials.gov ID
- NCT05740813
- Acronym / sponsor ID
- 2019P003518F
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Placebo-Controlled, Double-Blind, Regimen Specific Appendix, Lou Gehrig's Disease, ABBV-CLS-7262, Calico Life Sciences
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 310 participants (actual)
- Lead sponsor
- Merit E. Cudkowicz, MDUniversity, hospital or other
- Collaborators
- Calico Life Sciences LLC
- Study start
- 23 Mar 2023 (actual)
- Primary completion
- 3 Oct 2024 (actual)
- Study completion
- 3 Oct 2024 (actual)
- First posted
- 23 Feb 2023 (actual)
- Results
- Posted 14 Nov 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ABBV-CLS-7262 Dose 1 | Experimental | Drug: ABBV-CLS-7262 Dose 1 |
| ABBV-CLS-7262 Dose 2 | Experimental | Drug: ABBV-CLS-7262 Dose 2 |
| Matching Placebo | Placebo comparator | Drug: Matching Placebo |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
Key exclusion criteria (4 listed)
- The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ABBV-CLS-7262 Dose 1ABBV-CLS-7262 is administered orally once per day for 24 weeks. | Drug | ABBV-CLS-7262 Dose 1 |
| ABBV-CLS-7262 Dose 2ABBV-CLS-7262 is administered orally once per day for 24 weeks. | Drug | ABBV-CLS-7262 Dose 2 |
| Matching PlaceboMatching placebo is administered orally once per day for 24 weeks. | Drug | Matching Placebo |
Primary outcome
Disease Progression as Assessed by the ALSFRS-R-Slope
Time frame: Baseline to 24 Weeks
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.
Mortality Event Rate
Time frame: Baseline to 24 weeks
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.
6 secondary outcomes
Function by ALSFRS-R Total Score
Time frame: Baseline to 24 weeks
Respiratory Function
Time frame: Baseline to 24 Weeks
Upper Limb Muscle Strength
Time frame: Baseline to 24 weeks
Disease Progression Biomarker
Time frame: Baseline to 24 Weeks
Activities of Daily Living
Time frame: Baseline to 24 weeks
Number of Participants That Experienced Death or Death Equivalent
Time frame: Baseline to 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Merit Cudkowicz, MD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing ABBV-CLS-7262 Dose 1 and ABBV-CLS-7262 Dose 2 in people with Amyotrophic Lateral Sclerosis, run by Merit E. Cudkowicz, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Nov 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease Progression as Assessed by the ALSFRS-R-Slope (time frame: Baseline to 24 Weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Nov 2025; AzTrials last refreshed its copy on 26 Oct 2026.