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HEALEY ALS Platform Trial - Regimen G DNL343

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Enrolling by invitation
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
DNL343
Matching Placebo
Sponsored by
Merit E. Cudkowicz, MD
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Placebo-Controlled, Double-Blind, Regimen Specific Appendix, Lou Gehrig's Disease, DNL343, Denali Therapeutics

Eligibility Criteria

18 Years - 100 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683). Exclusion Criteria: The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683). Diagnosis of epilepsy or seizure within 6 months of randomization Hypersensitivity to DNL343 or any of the excipients contained within the DNL343 drug product 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.

Sites / Locations

  • Healey Center for ALS at Massachusetts General Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

DNL343

Matching Placebo

Arm Description

Outcomes

Primary Outcome Measures

Disease Progression
Change in disease severity over time as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score and survival. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.

Secondary Outcome Measures

Function
Change in ALSFRS-R total score over time. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Combined Assessment of Function and Survival (CAFS)
Combined assessment of function and survival uses ALSFRS-R total score as the function measure and death or permanent assisted ventilation as the survival component. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score and survival. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Respiratory Function
Change in respiratory function over time as assessed by slow vital capacity (SVC)
Muscle Strength
Change in muscle strength over time as measured isometrically using hand-held dynamometry and grip strength
Survival
Survival evaluated as time to death or permanent assisted ventilation (PAV)
Survival
Survival evaluated as time to death

Full Information

First Posted
April 24, 2023
Last Updated
May 29, 2023
Sponsor
Merit E. Cudkowicz, MD
Collaborators
Denali Therapeutics Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05842941
Brief Title
HEALEY ALS Platform Trial - Regimen G DNL343
Official Title
HEALEY ALS Platform Trial - Regimen G DNL343
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
May 24, 2023 (Actual)
Primary Completion Date
July 2025 (Anticipated)
Study Completion Date
August 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Merit E. Cudkowicz, MD
Collaborators
Denali Therapeutics Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
ALS, Placebo-Controlled, Double-Blind, Regimen Specific Appendix, Lou Gehrig's Disease, DNL343, Denali Therapeutics

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
240 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
DNL343
Arm Type
Experimental
Arm Title
Matching Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
DNL343
Intervention Description
DNL343 is administered orally once daily per day for 24 weeks.
Intervention Type
Drug
Intervention Name(s)
Matching Placebo
Intervention Description
Matching placebo is administered orally once daily per day for 24 weeks.
Primary Outcome Measure Information:
Title
Disease Progression
Description
Change in disease severity over time as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score and survival. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Time Frame
24 Weeks
Secondary Outcome Measure Information:
Title
Function
Description
Change in ALSFRS-R total score over time. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Time Frame
24 Weeks
Title
Combined Assessment of Function and Survival (CAFS)
Description
Combined assessment of function and survival uses ALSFRS-R total score as the function measure and death or permanent assisted ventilation as the survival component. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score and survival. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Time Frame
24 Weeks
Title
Respiratory Function
Description
Change in respiratory function over time as assessed by slow vital capacity (SVC)
Time Frame
24 Weeks
Title
Muscle Strength
Description
Change in muscle strength over time as measured isometrically using hand-held dynamometry and grip strength
Time Frame
24 Weeks
Title
Survival
Description
Survival evaluated as time to death or permanent assisted ventilation (PAV)
Time Frame
24 Weeks
Title
Survival
Description
Survival evaluated as time to death
Time Frame
24 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
100 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683). Exclusion Criteria: The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683). Diagnosis of epilepsy or seizure within 6 months of randomization Hypersensitivity to DNL343 or any of the excipients contained within the DNL343 drug product 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Merit Cudkowicz, MD
Organizational Affiliation
Massachusetts General Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Healey Center for ALS at Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States

12. IPD Sharing Statement

Learn more about this trial

HEALEY ALS Platform Trial - Regimen G DNL343

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