Clinical trial NCT04082832: CuATSM Compared With Placebo for Treatment of ALS/MND
Official title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study of Cu(II)ATSM in Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Collaborative Medicinal Development Pty Limited
Sponsor class: INDUSTRY
First posted: 2019-09-09
Enrollment: 80
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: ANCOVA will be used to compare efficacy endpoints between CuATSM and placebo groups
Primary purpose: TREATMENT

Brief summary: Multicenter, randomized, double-blind, placebo controlled study to assess the tolerabilty and efficacy of CuATSM in patients with ALS/MND. Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment.

Detailed description: Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment. Study drug is administered orally, once a day in fasted state (before breakfast). Assessments for safety (physical examination, vital signs, hematology, serum chemistry adverse events) will be conducted at baseline and following each cycle of treatment. Assessments for efficacy (Revised ALS Functional Rating Scale \[ASLFRS-R\] score, and Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen \[ECAS\] score, and seated slow vital capacity \[SVC\]) will be conducted at baseline and following 2, 4 and 6 cycles of treatment. Analysis of covariance (ANCOVA) will be used to compare efficacy endpoints between CuATSM and placebo groups.

Eligibility criteria: Inclusion Criteria:

* signed informed consent
* familial or sporadic ALS/MNS by Awaji-shima Consensus Recommendations
* not taking riluzole or on stable dose of riluzole for 4 weeks prior to screening visit
* no prior exposure to agents other than riluzole for treatment of ALS
* adequate bone marrow reserve, renal and liver function
* women of childbearing potential must have a negative pregnancy test and be non-lactating
* women and men with partners of childbearing potential must take effective contraception while on treatment

Exclusion Criteria:

* presence of a gastrointestinal disorder (eg, malabsorption) that might jeopardize intestinal absorption of study drug
* inability to perform seated SVC
* known immune compromising illness or treatment
* drug abuse or alcoholism
* clinically significant or active cardiovascular disease
* acute or chronic infection
* diagnosis of malignancy within 2 years prior to screening
* dementia that may affect patient understanding and/or compliance with study requirements and procedures
* current use of strong inducers or inhibitors of CYPs 2C19 and 2D6
* current us of medications (other than riluzole) that are metabolized predominantly by CYP 1A2

Primary outcomes:
measure: Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst])
timeFrame: 24 weeks
measure: Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst])
timeFrame: 24 weeks
Secondary outcomes:
measure: seated slow vital capacity (SVC)
timeFrame: 24 weeks
measure: rate of adverse events
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Macquarie
country: Australia
facility: Macquarie University
state: New South Wales
Study officials:
affiliation: Macquarie University
name: Dominic Rowe, MD
role: PRINCIPAL_INVESTIGATOR
References: