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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)

Disease:

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

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase I

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

Richard Tsai, MD Denali Therapeutics Inc.

Clinicaltrials.gov ID (11 digit #):

NCT05006352

Neals Affiliated?

No

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, South Holland, Netherlands

Full Study Summary:

Study Sponsor:

Denali Therapeutics Inc.

Participant Duration:

Estimated Enrollment:

30

Estimated Study Start Date:

08/11/2021

Estimated Study Completion Date:

12/31/2023

Posting Last Modified Date:

05/27/2022

Date Study Added to alsconsortium.org:

11/03/2021
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    <18 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    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

    University of California at San Diego
    Rose Previte / .(JavaScript must be enabled to view this email address) / 858-246-1319
    San Diego, California 92093
    United States

    California Pacific Medical Center
    Marnina Murez / .(JavaScript must be enabled to view this email address) / 638-224-7736
    San Francisco, California 94115
    United States

    PPD Orlando
    Project Manager / .(JavaScript must be enabled to view this email address) / 689-216-3179
    Orlando, Florida 32806
    United States

    Emory University
    Clinical Research Nurse / .(JavaScript must be enabled to view this email address) / 404-727-1679
    Atlanta, Georgia 30322
    United States

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