HEALEY ALS Platform Trial - Regimen G DNL343

NCT ID: NCT05842941

Last Updated: 2026-01-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 249 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-05-24
• Study Completion Date: 2024-10-29

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 G will evaluate the safety and efficacy of a single study drug, DNL343, 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 G DNL343, the participant will complete a screening visit to assess additional Regimen G eligibility criteria. Once Regimen G eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active DNL343 or matching placebo.

Regimen G will enroll by invitation, as participants may not choose to enroll in Regimen G. 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 G.

For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

DNL343

Group Type: EXPERIMENTAL

DNL343

Intervention Type: DRUG

DNL343 is administered orally once daily per day for 24 weeks.

Matching Placebo

Group Type: PLACEBO_COMPARATOR

Matching Placebo

Intervention Type: DRUG

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

Interventions

DNL343

DNL343 is administered orally once daily per day for 24 weeks.

Intervention Type: DRUG

Matching Placebo

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

Intervention Type: DRUG

Eligibility Criteria

Exclusion Criteria

1. Diagnosis of epilepsy or seizure within 6 months of randomization

2. Hypersensitivity to DNL343 or any of the excipients contained within the DNL343 drug product

3. The concomitant use of prescription or over-the-counter (OTC) medications that are inducers of certain cytochrome P450 enzymes, substrates of certain cytochrome P450 enzymes, or substrates of certain drug transporters.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 100 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Denali Therapeutics Inc.

INDUSTRY

Sponsor Role: collaborator

Merit E. Cudkowicz, MD

OTHER

Sponsor Role: lead

Responsible Party

Merit E. Cudkowicz, MD

Chief, Neurology Department

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Merit Cudkowicz, MD

Role: PRINCIPAL_INVESTIGATOR

Massachusetts General Hospital

Locations

Healey Center for ALS at Massachusetts General Hospital

Boston, Massachusetts, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2019P003518G

Identifier Type: -

Identifier Source: org_study_id