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

NCT05740813 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 2019P003518F
• Study Type: interventional
• Target: 310
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  Baseline to 24 weeks

Secondary Outcomes (6)

• Function by ALSFRS-R Total Score

  Baseline to 24 weeks

• Respiratory Function

  Baseline to 24 Weeks

• Upper Limb Muscle Strength

  Baseline to 24 weeks

• Disease Progression Biomarker

  Baseline to 24 Weeks

• Activities of Daily Living

  Baseline to 24 weeks

Study Arms (3)

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

Interventions

ABBV-CLS-7262 Dose 1 DRUG

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

ABBV-CLS-7262 Dose 1

ABBV-CLS-7262 Dose 2 DRUG

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

ABBV-CLS-7262 Dose 2

Matching Placebo DRUG

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

Matching Placebo

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Merit E. Cudkowicz, MD: lead
• Calico Life Sciences LLC: collaborator

Study Sites (1)

Healey Center for ALS at Mass General

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Healey Center for ALS Project Management
• Organization: Healey Center for ALS at Massachusetts General Hospital

healeyalsplatform@mgh.harvard.edu

833-425-8257 (HALT ALS)

Study Officials

• Merit Cudkowicz, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief, Neurology Department

Study Record Dates

• First Submitted: February 12, 2023
• First Posted: February 23, 2023
• Study Start: March 23, 2023
• Primary Completion: October 3, 2024
• Study Completion: October 3, 2024
• Last Updated: November 14, 2025
• Results First Posted: November 14, 2025

Record last verified: 2025-10

Locations

US (1)