ALS Treatment Extension Study

NCT ID: NCT03136809

Last Updated: 2022-02-17

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 28 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-01-18
• Study Completion Date: 2022-12-31

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.

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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cu(II)ATSM

Cu(II)ATSM administered once daily

Group Type: EXPERIMENTAL

Cu(II)ATSM

Intervention Type: DRUG

copper-containing synthetic small molecule

Interventions

Cu(II)ATSM

copper-containing synthetic small molecule

Intervention Type: DRUG

Other Intervention Names

diacetylbis(N(4)-methylthiosemicarbazonato) copper(II)

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Collaborative Medicinal Development Pty Limited

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Dominic Rowe, MD

Role: PRINCIPAL_INVESTIGATOR

Macquarie University

Locations

Macquarie University

Sydney, New South Wales, Australia

Countries

Australia

Other Identifiers

CMD-2017-001

Identifier Type: -

Identifier Source: org_study_id