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ALS Treatment Extension Study

Sponsor
Collaborative Medicinal Development Pty Limited (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03136809
Collaborator
(none)
28
Enrollment
1
Location
1
Arm
59.4
Anticipated Duration (Months)
0.5
Patients Per Site Per Month

Study Details

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
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
Actual Enrollment :
28 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Intervention Model Description:
Multicenter, open-label treatment extension studyMulticenter, 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 :
Jan 18, 2018
Anticipated Primary Completion Date :
Dec 31, 2022
Anticipated Study Completion Date :
Dec 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 Names:
  • diacetylbis(N(4)-methylthiosemicarbazonato) copper(II)

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Tolerance of extended treatment [24 months]

      safety

    Secondary Outcome Measures

    1. Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R) [24 months]

      efficacy

    2. Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score [24 months]

      efficacy

    3. Treatment-related change in respiratory function by seated forced vital capacity (FVC) [24 months]

      efficacy

    4. Treatment-related change in quality of life by ALSSQOL-R score [24 months]

      efficacy

    5. Treatment-related change in respiratory function by sniff nasal pressure (SNP) test [24 months]

      efficacy

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 75 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Site City State Country Postal Code
    1 Macquarie University Sydney New South Wales Australia 2109

    Sponsors and Collaborators

    • Collaborative Medicinal Development Pty Limited

    Investigators

    • Principal Investigator: Dominic Rowe, MD, Macquarie University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    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:
    Feb 17, 2022
    Last Verified:
    Feb 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    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
    Copper

    Study Results

    No Results Posted as of Feb 17, 2022