Clinical trial NCT03136809: ALS Treatment Extension Study
Official title: A Treatment Continuation Study for Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease Who Have Successfully Completed Study CMD-2016-001
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Collaborative Medicinal Development Pty Limited
Sponsor class: INDUSTRY
First posted: 2017-05-02
Enrollment: 28
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Multicenter, open-label treatment extension study
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Tolerance of extended treatment
timeFrame: 24 months
Secondary outcomes:
measure: Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)
timeFrame: 24 months
measure: Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score
timeFrame: 24 months
measure: Treatment-related change in respiratory function by seated forced vital capacity (FVC)
timeFrame: 24 months
measure: Treatment-related change in quality of life by ALSSQOL-R score
timeFrame: 24 months
measure: Treatment-related change in respiratory function by sniff nasal pressure (SNP) test
timeFrame: 24 months
Other outcomes:
Locations:
city: Sydney
country: Australia
facility: Macquarie University
state: New South Wales
Study officials:
affiliation: Macquarie University
name: Dominic Rowe, MD
role: PRINCIPAL_INVESTIGATOR
References: