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CuATSM Compared With Placebo for Treatment of ALS/MND

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Unknown status
Phase
Phase 2
Locations
Australia
Study Type
Interventional
Intervention
Cu(II)ATSM
Placebos
Sponsored by
Collaborative Medicinal Development Pty Limited
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Macquarie UniversityRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Cu(II)ATSM

Placebo Powder for Oral Suspension

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst])
assessment of disease severity
Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst])
assessment of cognitive function

Secondary Outcome Measures

seated slow vital capacity (SVC)
assessment of respiratory function
rate of adverse events
tolerability assessment

Full Information

First Posted
September 2, 2019
Last Updated
November 4, 2019
Sponsor
Collaborative Medicinal Development Pty Limited
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1. Study Identification

Unique Protocol Identification Number
NCT04082832
Brief Title
CuATSM Compared With Placebo for Treatment of ALS/MND
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
Study Type
Interventional

2. Study Status

Record Verification Date
November 2019
Overall Recruitment Status
Unknown status
Study Start Date
September 30, 2019 (Actual)
Primary Completion Date
December 30, 2020 (Anticipated)
Study Completion Date
December 30, 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Collaborative Medicinal Development Pty Limited

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Model Description
ANCOVA will be used to compare efficacy endpoints between CuATSM and placebo groups
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
placebo controlled
Allocation
Randomized
Enrollment
80 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cu(II)ATSM
Arm Type
Active Comparator
Arm Description
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.
Arm Title
Placebo Powder for Oral Suspension
Arm Type
Placebo Comparator
Arm Description
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 Type
Drug
Intervention Name(s)
Cu(II)ATSM
Intervention Description
oral suspension
Intervention Type
Drug
Intervention Name(s)
Placebos
Intervention Description
oral suspension
Primary Outcome Measure Information:
Title
Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst])
Description
assessment of disease severity
Time Frame
24 weeks
Title
Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst])
Description
assessment of cognitive function
Time Frame
24 weeks
Secondary Outcome Measure Information:
Title
seated slow vital capacity (SVC)
Description
assessment of respiratory function
Time Frame
24 weeks
Title
rate of adverse events
Description
tolerability assessment
Time Frame
24 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Kay Noel, PhD
Phone
(415) 444 9600
Email
Kay.Noel@colMedDev.com
First Name & Middle Initial & Last Name or Official Title & Degree
Craig Rosenfeld, MD
Phone
(415) 444 9600
Email
CraigR@ColMedDev.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dominic Rowe, MD
Organizational Affiliation
Macquarie University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Macquarie University
City
Macquarie
State/Province
New South Wales
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dominic Rowe, MD

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

CuATSM Compared With Placebo for Treatment of ALS/MND

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