Clinical trial NCT05842941: HEALEY ALS Platform Trial - Regimen G DNL343
Official title: HEALEY ALS Platform Trial - Regimen G DNL343
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Placebo-Controlled
Double-Blind
Regimen Specific Appendix
Lou Gehrig's Disease
DNL343
Denali Therapeutics
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Denali Therapeutics Inc.
First posted: 2023-05-06
Enrollment: 249
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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).

  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.

Primary outcomes:
measure: Disease Progression
timeFrame: 24 Weeks
Secondary outcomes:
measure: Function
timeFrame: 24 Weeks
measure: Combined Assessment of Function and Survival (CAFS)
timeFrame: 24 Weeks
measure: Respiratory Function
timeFrame: 24 Weeks
measure: Muscle Strength
timeFrame: 24 Weeks
measure: Survival
timeFrame: 24 Weeks
measure: Survival
timeFrame: 24 Weeks
Other outcomes:
Locations:
city: Boston
country: United States
facility: Healey Center for ALS at Massachusetts General Hospital
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Merit Cudkowicz, MD
role: PRINCIPAL_INVESTIGATOR
References: