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Quick Info
Status
Currently Recruiting
Phase
2/ 3
Trial Type
Interventional
Treatment Type
Drug Trials
Randomization
1:1
Enrollment
80
Start Date
9/30/2019
Sponsor
Collaborative Medicinal Development Pty Limited
Contact Information
Locations
Australia, Other
Macquarie University, Macquarie, Australia
Contact: Dominic Rowe, MD
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
N/A
Months Since Onset
Number of months since first symptoms of ALS.
N/A
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
N/A
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
N/A
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
No
Open Label
No
Update Notes
Updated recruitment status
11/7/2019
Added New Trial
9/12/2019

Other Information

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.
Eligibility
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
Details
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.
Collaborator(s)
  • Collaborative Medicinal Development Pty Limited
Trial Protocol as Published on Clinicaltrials.gov
NCT04082832 (First Published: 9/2/2019)