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CuATSM Compared With Placebo for Treatment of ALS/MND

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ClinicalTrials.gov Identifier: NCT04082832
Recruitment Status : Recruiting
First Posted : September 9, 2019
Last Update Posted : November 6, 2019
Sponsor:
Information provided by (Responsible Party):
Collaborative Medicinal Development Pty Limited

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Cu(II)ATSM Drug: Placebos Phase 2 Phase 3

Detailed Description:
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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: ANCOVA will be used to compare efficacy endpoints between CuATSM and placebo groups
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: placebo controlled
Primary Purpose: Treatment
Official Title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study of Cu(II)ATSM in Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease
Actual Study Start Date : September 30, 2019
Estimated Primary Completion Date : December 30, 2020
Estimated Study Completion Date : December 30, 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Cu(II)ATSM
Cu(II)ATSM Powder for Oral Suspension, 36 mg, to be reconstituted with Diluent (15 mL of sugar-free flavored pharmaceutical syrup) to provide an oral suspension for immediate consumption. Specified dose is 72 mg (2 bottles) taken fasting, before breakfast each day.
 Drug: Cu(II)ATSM
oral suspension
Placebo Comparator: Placebo Powder for Oral Suspension
Placebo Powder for Oral Suspension, to be reconstituted with Diluent (15 mL of sugar-free flavored pharmaceutical syrup) to provide an oral suspension for immediate consumption. Specified dose is 72 mg (2 bottles) taken fasting, before breakfast each day.
 Drug: Placebos
oral suspension


Outcome Measures
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Primary Outcome Measures :
  1. Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst]) [ Time Frame: 24 weeks ]
    assessment of disease severity

  2. Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst]) [ Time Frame: 24 weeks ]
    assessment of cognitive function


Secondary Outcome Measures :
  1. seated slow vital capacity (SVC) [ Time Frame: 24 weeks ]
    assessment of respiratory function

  2. rate of adverse events [ Time Frame: 24 weeks ]
    tolerability assessment


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04082832


Contacts
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Contact: Kay Noel, PhD (415) 444 9600 Kay.Noel@colMedDev.com
Contact: Craig Rosenfeld, MD (415) 444 9600 CraigR@ColMedDev.com

Locations
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Australia, New South Wales
Macquarie University Recruiting
Macquarie, New South Wales, Australia
Contact: Dominic Rowe, MD         
Sponsors and Collaborators
Collaborative Medicinal Development Pty Limited
Investigators
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Principal Investigator: Dominic Rowe, MD Macquarie University
More Information
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Responsible Party: Collaborative Medicinal Development Pty Limited
ClinicalTrials.gov Identifier: NCT04082832    
Other Study ID Numbers: CMD-2019-001
First Posted: September 9, 2019    Key Record Dates
Last Update Posted: November 6, 2019
Last Verified: November 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases