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.Study Status:
Not recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
Cu(II)ATSM, Placebos
Placebo:
Yes
Phase:
Phase 2/Phase 3
Study Chair(s)/Principal Investigator(s):
Dominic Rowe, MD, Macquarie University
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
Kay Noel, PhD / email hidden; JavaScript is required / (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
Estimated Enrollment:
80
Estimated Study Start Date:
09 / 30 / 2019
Estimated Study Completion Date:
12 / 30 / 2020
Posting Last Modified Date:
11 / 06 / 2019
Date Study Added to neals.org:
09 / 09 / 2019
Minimum Age:
18 Years
Maximum Age:
75 Years
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
Macquarie University | Recruiting
Dominic Rowe, MD
Macquarie, New South Wales
Australia