A Phase 1b, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study, Followed by an Open-Label Extension, to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis

Study Purpose:

This is a Phase 1b, multicenter, randomized, placebo-controlled, double-blind study of 28 days, followed by an 18-month open-label extension, designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL343 in participants with amyotrophic lateral sclerosis (ALS)


Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:



Phase I

Study Chair(s)/Principal Investigator(s):

Richard Tsai, MD Denali Therapeutics Inc.

Clinicaltrials.gov ID (11 digit #):


Neals Affiliated?


Coordinating Center Contact Information

Centre for Human Drug Research (CHDR)
Project Manager / .(JavaScript must be enabled to view this email address) / +31 71 5246 400
.(JavaScript must be enabled to view this email address) Leiden, Netherlands

Full Study Summary:

Study Sponsor:

Denali Therapeutics Inc.

Participant Duration:

Estimated Enrollment:


Estimated Study Start Date:


Estimated Study Completion Date:


Posting Last Modified Date:


Date Study Added to alsconsortium.org:

  • Eligibility Criteria


    Female, Male

    Minimum Age:


    Maximum Age:


    Min Vital Capacity (% predicted normal):


    Time since Symptom Onset:

    <18 months

    Time since Diagnosis:

    Can participants use Riluzole?


    Key Inclusion Criteria:

    • Diagnosis of sporadic or familial ALS
    • Less than 3 years since ALS symptom onset
    • Stable doses of approved ALS treatments (riluzole and/or edaravone) for at least 2 months prior to screening
    • Participants must be able to swallow the study intervention
    • Vital capacity >50% predicted at screening
    • Women must have been surgically sterilized or be postmenopausal
    • Men, and sex partner if a woman of childbearing potential, must use highly effective contraception

    Key Exclusion Criteria:

    • Any history of unstable or poorly controlled psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematologic, immunologic, or allergic disease, or other major disorders
    • Positive serum pregnancy test or currently lactating or breastfeeding
    • History of malignancy within 5 years
    • History of clinically significant neurologic disorders other than ALS

  • Site Contact Information