CuATSM Compared With Placebo for Treatment of ALS/MND

• Sponsor: Collaborative Medicinal Development Pty Limited (Industry)
• Overall Status: Unknown status
• CT.gov ID: NCT04082832
• Collaborator: (none)
• Enrollment: 80
• Location: 1
• Arms: 2
• Anticipated Duration (Months): 15
• Patients Per Site Per Month: 5.3

Study Details

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.

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

• Study Type: Interventional
• Anticipated Enrollment: 80 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Intervention Model Description: ANCOVA will be used to compare efficacy endpoints between CuATSM and placebo groupsANCOVA 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: Sep 30, 2019
• Anticipated Primary Completion Date: Dec 30, 2020
• Anticipated Study Completion Date: Dec 30, 2020

Arms and Interventions

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

Primary Outcome Measures

1. Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst]) [24 weeks]

   assessment of disease severity

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

   assessment of cognitive function

Secondary Outcome Measures

1. seated slow vital capacity (SVC) [24 weeks]

   assessment of respiratory function

2. rate of adverse events [24 weeks]

   tolerability assessment

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 75 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Locations

	Site	City	State	Country	Postal Code
1	Macquarie University	Macquarie	New South Wales	Australia

Sponsors and Collaborators

• Collaborative Medicinal Development Pty Limited

Investigators

• Principal Investigator: Dominic Rowe, MD, Macquarie University

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Collaborative Medicinal Development Pty Limited
• ClinicalTrials.gov Identifier: NCT04082832
• Other Study ID Numbers: CMD-2019-001
• First Posted: Sep 9, 2019
• Last Update Posted: Nov 6, 2019
• Last Verified: Nov 1, 2019

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis

Study Results

No Results Posted as of Nov 6, 2019