CuATSM Compared With Placebo for Treatment of ALS/MND

NCT04082832 | Unknown Phase 2 DMC

• 1 other identifier: CMD-2019-001
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (2)

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

  assessment of disease severity

  24 weeks

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

  assessment of cognitive function

  24 weeks

Secondary Outcomes (2)

• seated slow vital capacity (SVC)

  24 weeks

• rate of adverse events

  24 weeks

Study Arms (2)

Cu(II)ATSM

ACTIVE COMPARATOR

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

Placebo Powder for Oral Suspension

PLACEBO COMPARATOR

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

Interventions

Cu(II)ATSM DRUG

oral suspension

Cu(II)ATSM

Placebos DRUG

oral suspension

Placebo Powder for Oral Suspension

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Collaborative Medicinal Development Pty Limited: lead

Study Sites (1)

Macquarie University

Macquarie, New South Wales, Australia

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

copper (II) diacetyl-di(N(4)-methylthiosemicarbazone)

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Dominic Rowe, MD

  Macquarie University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kay Noel, PhD

CONTACT

(415) 444 9600; Kay.Noel@colMedDev.com

Craig Rosenfeld, MD

CONTACT

(415) 444 9600; CraigR@ColMedDev.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: placebo controlled
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: ANCOVA will be used to compare efficacy endpoints between CuATSM and placebo groups

Study Record Dates

• First Submitted: September 2, 2019
• First Posted: September 9, 2019
• Study Start: September 30, 2019
• Primary Completion: December 30, 2020
• Study Completion: December 30, 2020
• Last Updated: November 6, 2019

Record last verified: 2019-11

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)