ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 2 of 5 for:    collaborative medicinal

ALS Treatment Extension Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03136809
Recruitment Status : Recruiting
First Posted : May 2, 2017
Last Update Posted : July 21, 2017
Sponsor:
Information provided by (Responsible Party):
Collaborative Medicinal Development Pty Limited

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
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 : May 8, 2017
Estimated Primary Completion Date : May 31, 2019
Estimated Study Completion Date : May 31, 2019


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)



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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 75 Years   (Adult, Senior)
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03136809


Contacts
Contact: Kay Noel, PhD 415 444 9602 Kay.Noel@ColMedDev.com
Contact: Craig Rosenfeld, MD 415 444 9602 Craig@ColMedDev.com

Locations
Australia, New South Wales
Macquarie University Recruiting
Sydney, New South Wales, Australia, 2109
Contact: Dominic Rowe, MD       dominic.rowe@mq.edu.au   
Sponsors and Collaborators
Collaborative Medicinal Development Pty Limited
Investigators
Principal Investigator: Dominic Rowe, MD Macquarie University

Responsible Party: Collaborative Medicinal Development Pty Limited
ClinicalTrials.gov Identifier: NCT03136809     History of Changes
Other Study ID Numbers: CMD-2017-001
First Posted: May 2, 2017    Key Record Dates
Last Update Posted: July 21, 2017
Last Verified: July 2017
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:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral 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
Growth Substances
Physiological Effects of Drugs