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A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study of Cu(II)ATSM in Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease

Study Purpose:

Multicenter, randomized, double-blind, placebo controlled study to assess the tolerabilty and efficacy of CuATSM in patients with ALS/MND. Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment.

Disease:

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

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II ,Phase III

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

Dominic Rowe, MD Macquarie University

Clinicaltrials.gov ID (11 digit #):

NCT04082832

Neals Affiliated?

No

Coordinating Center Contact Information


Kay Noel, PhD / .(JavaScript must be enabled to view this email address) / 415-444 9600
Craig Rosenfeld, MD / .(JavaScript must be enabled to view this email address) / 415-444 9600
Australia

Full Study Summary:

Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment. Study drug is administered orally, once a day in fasted state (before breakfast). Assessments for safety (physical examination, vital signs, hematology, serum chemistry adverse events) will be conducted at baseline and following each cycle of treatment. Assessments for efficacy (Revised ALS Functional Rating Scale [ASLFRS-R] score, and Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen [ECAS] score, and seated slow vital capacity [SVC]) will be conducted at baseline and following 2, 4 and 6 cycles of treatment. Analysis of covariance (ANCOVA) will be used to compare efficacy endpoints between CuATSM and placebo groups.

Study Sponsor:

Collaborative Medicinal Development Pty Limited

Participant Duration:

Estimated Enrollment:

80

Estimated Study Start Date:

09/30/2019

Estimated Study Completion Date:

12/30/2020

Posting Last Modified Date:

11/12/2019

Date Study Added to alsconsortium.org:

11/12/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    75

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    • signed informed consent
    • familial or sporadic ALS/MNS by Awaji-shima Consensus Recommendations
    • not taking riluzole or on stable dose of riluzole for 4 weeks prior to screening visit
    • no prior exposure to agents other than riluzole for treatment of ALS
    • adequate bone marrow reserve, renal and liver function
    • women of childbearing potential must have a negative pregnancy test and be non-lactating
    • women and men with partners of childbearing potential must take effective contraception while on treatment

    Exclusion Criteria:

    • presence of a gastrointestinal disorder (eg, malabsorption) that might jeopardize intestinal absorption of study drug
    • inability to perform seated SVC
    • known immune compromising illness or treatment
    • drug abuse or alcoholism
    • clinically significant or active cardiovascular disease
    • acute or chronic infection
    • diagnosis of malignancy within 2 years prior to screening
    • dementia that may affect patient understanding and/or compliance with study requirements and procedures
    • current use of strong inducers or inhibitors of CYPs 2C19 and 2D6
    • current us of medications (other than riluzole) that are metabolized predominantly by CYP 1A2

  • Site Contact Information

    Macquarie University
    Macquarie, New South Wales
    Australia