ALS Treatment Extension Study

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03136809
• Other study ID #: CMD-2017-001
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: January 18, 2018
• Est. completion date: December 31, 2022

Study information

• Verified date: February 2022
• Source: Collaborative Medicinal Development Pty Limited
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 28
• Est. completion date: December 31, 2022
• Est. primary completion date: December 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• Cu(II)ATSM
copper-containing synthetic small molecule

Locations

Country	Name	City	State
Australia	Macquarie University	Sydney	New South Wales

Sponsors (1)

Lead Sponsor	Collaborator
Collaborative Medicinal Development Pty Limited

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Tolerance of extended treatment	safety	24 months
Secondary	Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)	efficacy	24 months
Secondary	Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score	efficacy	24 months
Secondary	Treatment-related change in respiratory function by seated forced vital capacity (FVC)	efficacy	24 months
Secondary	Treatment-related change in quality of life by ALSSQOL-R score	efficacy	24 months
Secondary	Treatment-related change in respiratory function by sniff nasal pressure (SNP) test	efficacy	24 months