A Multicenter, Randomized, Placebo-Controlled, Double-Blind, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL747 in Subjects With Amyotrophic Lateral Sclerosis

Study Purpose:

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple oral doses of DNL747 in subjects with Amyotrophic Lateral Sclerosis when administered for 29 days in a cross-over design

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):

Clinicaltrials.gov ID (11 digit #):

NCT03757351

Neals Affiliated?

No

Coordinating Center Contact Information


Melissa Leedom / .(JavaScript must be enabled to view this email address) / 913-624-4970
.(JavaScript must be enabled to view this email address)

Full Study Summary:

This is a Phase 1b randomized, placebo-controlled, double-blind, crossover study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL747 in subjects with Amyotrophic Lateral Sclerosis (ALS)

Study Sponsor:

Denali Therapeutics Inc.

Participant Duration:

Estimated Enrollment:

16

Estimated Study Start Date:

12/28/2018

Estimated Study Completion Date:

08/31/2019

Posting Last Modified Date:

09/12/2019

Date Study Added to alsconsortium.org:

07/30/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    21

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    <36 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    • Women of non-childbearing potential and men, aged 21−80 years
    • Willingness and ability to complete all aspects of the study; participant should be capable of completing assessments either alone or with help of a caregiver
    • Diagnosis of laboratory-supported probable, probable, or definite (sporadic or familial) ALS according to the El Escorial World Federation of Neurology revised research diagnostic criteria
    • Less than 3 years since symptom onset
    • Forced vital capacity (FVC) >50% predicted measured within 30 days of screening
    • If subject is taking approved ALS treatments (riluzole and/or edaravone), doses must be stable for ≥2 months prior to screening and subject is expected to stay on a stable regimen throughout the study

    Exclusion Criteria:

    • History of a clinically significant non-ALS neurologic disorder (other than frontal temporal lobe dementia), including, but not limited to, muscular dystrophy, spinal stenosis, peripheral neuropathy, inherited neuropathies, AD, Parkinson's disease, Lewy body dementia, vascular dementia, Huntington's disease, epilepsy, stroke, multiple sclerosis, brain tumor, or brain infection or abscess
    • Unstable or poorly controlled comorbid disease process of any organ system currently requiring active treatment or likely to require treatment adjustment during the study

  • Site Contact Information

    Bioclinica
    Matthew Bubalo / .(JavaScript must be enabled to view this email address) / Orlando, Florida 32806
    United States

    PRA Health Sciences
    Karena McDonald, RN / .(JavaScript must be enabled to view this email address) / 801-904-4640
    Salt Lake City, Utah 84124
    United States

    CHDR
    Leiden, South Holland
    Netherlands