ALS Treatment Extension Study

• ClinicalTrials.gov Identifier: NCT03136809
• Recruitment Status: Active, not recruiting
• First Posted: May 2, 2017
• Last Update Posted: February 17, 2022
• Sponsor: Collaborative Medicinal Development Pty Limited
• Information provided by (Responsible Party): Collaborative Medicinal Development Pty Limited

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Amyotrophic Lateral Sclerosis	Drug: Cu(II)ATSM	Phase 1; Phase 2

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 28 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Intervention Model Description: Multicenter, open-label treatment extension study
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Treatment Continuation Study for Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease Who Have Successfully Completed Study CMD-2016-001
• Actual Study Start Date: January 18, 2018
• Estimated Primary Completion Date: December 31, 2022
• Estimated Study Completion Date: December 31, 2022

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. Tolerance of extended treatment [ Time Frame: 24 months ]
   safety

Secondary Outcome Measures :

1. Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R) [ Time Frame: 24 months ]
   efficacy
2. Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score [ Time Frame: 24 months ]
   efficacy
3. Treatment-related change in respiratory function by seated forced vital capacity (FVC) [ Time Frame: 24 months ]
   efficacy
4. Treatment-related change in quality of life by ALSSQOL-R score [ Time Frame: 24 months ]
   efficacy
5. Treatment-related change in respiratory function by sniff nasal pressure (SNP) test [ Time Frame: 24 months ]
   efficacy

Eligibility Criteria

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

Criteria

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

Contacts and Locations

Locations

Australia, New South Wales

Macquarie University

Sydney, New South Wales, Australia, 2109

Sponsors and Collaborators

Collaborative Medicinal Development Pty Limited

Investigators

• Principal Investigator: Dominic Rowe, MD; Macquarie University

More Information

• Responsible Party: Collaborative Medicinal Development Pty Limited
• ClinicalTrials.gov Identifier: NCT03136809
• Other Study ID Numbers: CMD-2017-001
• First Posted: May 2, 2017
• Last Update Posted: February 17, 2022
• Last Verified: February 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: Plan to publish study results in a peer-reviewed journal

• 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

Additional relevant MeSH terms:

Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis; Pathologic Processes; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Copper; Trace Elements; Micronutrients; Physiological Effects of Drugs