ALS Treatment Extension Study

A Treatment Continuation Study for Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease Who Have Successfully Completed Study CMD-2016-001

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: Cu(II)ATSM

Detailed Description

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. The same safety assessments as in the CMD-2016-001 study will be conducted after each cycle of treatment and the same efficacy assessments as in the CMD-2016-001 study will be conducted after every 3 cycles of treatment. Treatment will continue until the first to occur of (1) Investigator considers the patient is no longer deriving benefit from CuATSM treatment, (2) patient develops dependence on mechanical ventilation where dependence is defined as being unable to lie flat (supine) without it, unable to sleep without it, or daytime use, or (3) the patient withdraws consent.

• Study Type: Interventional
• Enrollment (Actual): 28
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2109
      • Macquarie University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cu(II)ATSM; Cu(II)ATSM administered once daily	Drug: Cu(II)ATSM; copper-containing synthetic small molecule; Other Names: diacetylbis(N(4)-methylthiosemicarbazonato) copper(II)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tolerance of extended treatment	safety	24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)	efficacy	24 months
Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score	efficacy	24 months
Treatment-related change in respiratory function by seated forced vital capacity (FVC)	efficacy	24 months
Treatment-related change in quality of life by ALSSQOL-R score	efficacy	24 months
Treatment-related change in respiratory function by sniff nasal pressure (SNP) test	efficacy	24 months

Collaborators and Investigators

• Sponsor: Collaborative Medicinal Development Pty Limited

Study record dates

Study Major Dates

• Study Start (Actual): January 18, 2018
• Primary Completion (Anticipated): December 31, 2022
• Study Completion (Anticipated): December 31, 2022

Study Registration Dates

• First Submitted: April 28, 2017
• First Submitted That Met QC Criteria: April 28, 2017
• First Posted (Actual): May 2, 2017

Study Record Updates

• Last Update Posted (Actual): February 17, 2022
• Last Update Submitted That Met QC Criteria: February 15, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Physiological Effects of Drugs; Trace Elements; Micronutrients; Copper
• Other Study ID Numbers: CMD-2017-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: Plan to publish study results in a peer-reviewed journal

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes