ALS Treatment Extension Study

NCT03136809 | Unknown Phase 1 DMC FDA Drug

• 1 other identifier: CMD-2017-001
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 1

Brief Summary

Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Tolerance of extended treatment

  safety

  24 months

Secondary Outcomes (5)

• Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)

  24 months

• Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score

  24 months

• Treatment-related change in respiratory function by seated forced vital capacity (FVC)

  24 months

• Treatment-related change in quality of life by ALSSQOL-R score

  24 months

• Treatment-related change in respiratory function by sniff nasal pressure (SNP) test

  24 months

Study Arms (1)

Cu(II)ATSM

EXPERIMENTAL

Cu(II)ATSM administered once daily

Drug: Cu(II)ATSM

Interventions

Cu(II)ATSM DRUG

copper-containing synthetic small molecule

Also known as: diacetylbis(N(4)-methylthiosemicarbazonato) copper(II)

Cu(II)ATSM

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 prior to initiation of any study-specific procedures and treatment
• Documented completion of protocol-specific assessments following completion of six 28-day treatment cycles in study CMD-2016-001
• Principal Investigator considers the patient would benefit from continued treatment with Cu(II)ATSM
• Not taking riluzole or on the same (or lower) dose used during the CMD-2016-001 study
• Adequate bone marrow reserve, renal and liver function
• Women and men with partners of childbearing potential must take effective contraception while on study treatment

You may not qualify if:

• Inability to swallow oral medications or presence of a gastrointestinal disorder (e.g.,malabsorption) deemed to jeopardize intestinal absorption of study drug
• Dependence on mechanical ventilation (invasive or non-invasive) for any part of day or night, where dependence is defined as being unable to lie flat (supine) without it, unable to sleep without it, or daytime use
• Dementia that may affect either outcome measures or patient understanding and/or compliance with study requirements and procedures
• Current use of strong inducers or inhibitors of CYPs 2C19 and 2D6

Sponsors & Collaborators

• Collaborative Medicinal Development Pty Limited: lead

Study Sites (1)

Macquarie University

Sydney, New South Wales, 2109, Australia

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

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

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

Intervention Hierarchy (Ancestors)

Metals, Heavy; Elements; Inorganic Chemicals; Transition Elements; Metals

Study Officials

• Dominic Rowe, MD

  Macquarie University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Multicenter, open-label treatment extension study

Study Record Dates

• First Submitted: April 28, 2017
• First Posted: May 2, 2017
• Study Start: January 18, 2018
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2022
• Last Updated: February 17, 2022

Record last verified: 2022-02

Data Sharing

• IPD Sharing: Will share

Locations

AU (1)