CuATSM Compared With Placebo for Treatment of ALS/MND

Collaborative Medicinal Development

Status and phase: Unknown; Phase 3; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Cu(II)ATSM; Drug: Placebos

Study type: Interventional

Funder types: Industry

Identifiers

NCT04082832

CMD-2019-001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 80 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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80 participants in 2 patient groups, including a placebo group

Cu(II)ATSM

Active Comparator group

Description:

Cu(II)ATSM Powder for Oral Suspension, 36 mg, to be reconstituted with Diluent (15 mL of sugar-free flavored pharmaceutical syrup) to provide an oral suspension for immediate consumption. Specified dose is 72 mg (2 bottles) taken fasting, before breakfast each day.

Treatment:

Drug: Cu(II)ATSM

Placebo Powder for Oral Suspension

Placebo Comparator group

Description:

Placebo Powder for Oral Suspension, to be reconstituted with Diluent (15 mL of sugar-free flavored pharmaceutical syrup) to provide an oral suspension for immediate consumption. Specified dose is 72 mg (2 bottles) taken fasting, before breakfast each day.

Treatment:

Drug: Placebos