Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.

CuATSM Compared With Placebo for Treatment of ALS/MND

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
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

Tracking Information
First Submitted Date  ICMJE September 2, 2019
First Posted Date  ICMJE September 9, 2019
Last Update Posted Date November 6, 2019
Actual Study Start Date  ICMJE September 30, 2019
Estimated Primary Completion Date December 30, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 6, 2019)
  • Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst]) [ Time Frame: 24 weeks ]
    assessment of disease severity
  • 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
Original Primary Outcome Measures  ICMJE
 (submitted: September 6, 2019)
  • Revised ALS Functional Rating Scale (ALSFRS-R) [ Time Frame: 24 weeks ]
    assessment of disease severity
  • Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen [ECAS] [ Time Frame: 24 weeks ]
    assessment of cognitive function
Change History Complete list of historical versions of study NCT04082832 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 6, 2019)
  • seated slow vital capacity (SVC) [ Time Frame: 24 weeks ]
    assessment of respiratory function
  • rate of adverse events [ Time Frame: 24 weeks ]
    tolerability assessment
Original Secondary Outcome Measures  ICMJE
 (submitted: September 6, 2019)
  • seated slow vital capacity [SVC] [ Time Frame: 24 weeks ]
    assessment of respiratory function
  • rate of adverse events [ Time Frame: 24 weeks ]
    tolerability assessment
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE CuATSM Compared With Placebo for Treatment of ALS/MND
Official Title  ICMJE A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study of Cu(II)ATSM in Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease
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.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE 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
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE
  • Drug: Cu(II)ATSM
    oral suspension
  • Drug: Placebos
    oral suspension
Study Arms  ICMJE
  • 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.
    Intervention: Drug: Cu(II)ATSM
  • 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.
    Intervention: Drug: Placebos
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 6, 2019)
80
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 30, 2020
Estimated Primary Completion Date December 30, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Kay Noel, PhD (415) 444 9600 Kay.Noel@colMedDev.com
Contact: Craig Rosenfeld, MD (415) 444 9600 CraigR@ColMedDev.com
Listed Location Countries  ICMJE Australia
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04082832
Other Study ID Numbers  ICMJE CMD-2019-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Collaborative Medicinal Development Pty Limited
Study Sponsor  ICMJE Collaborative Medicinal Development Pty Limited
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Dominic Rowe, MD Macquarie University
PRS Account Collaborative Medicinal Development Pty Limited
Verification Date November 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP