CuATSM Compared With Placebo for Treatment of ALS/MND

Amyotrophic Lateral Sclerosis Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04082832
• Other study ID #: CMD-2019-001
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: September 30, 2019
• Est. completion date: December 30, 2020

Study information

• Verified date: November 2019
• Source: Collaborative Medicinal Development Pty Limited
• Contact: Kay Noel, PhD
• Phone: (415) 444 9600
• Email: Kay.Noel[@]colMedDev.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 80
• Est. completion date: December 30, 2020
• Est. primary completion date: December 30, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• Cu(II)ATSM
oral suspension
• Placebos
oral suspension

Locations

Country	Name	City	State
Australia	Macquarie University	Macquarie	New South Wales

Sponsors (1)

Lead Sponsor	Collaborator
Collaborative Medicinal Development Pty Limited

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst])	assessment of disease severity	24 weeks
Primary	Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst])	assessment of cognitive function	24 weeks
Secondary	seated slow vital capacity (SVC)	assessment of respiratory function	24 weeks
Secondary	rate of adverse events	tolerability assessment	24 weeks