NCT02644668

A Study of CK-2127107 in Patients With Spinal Muscular Atrophy

A Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients With Spinal Muscular Atrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 70 (actual)
• Sponsor: Cytokinetics · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the pharmacodynamic (PD) effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on measures of skeletal muscle function or fatigability in patients with Type II, III, or IV spinal muscular atrophy (SMA).

Detailed description

CY 5021 is a Phase 2, double-blind, randomized, placebo-controlled, multiple dose study of reldesemtiv in 2 sequential ascending dose cohorts of patients with SMA. Patients will be randomized 2:1 to receive reldesemtiv or placebo twice daily for 8 weeks. Patients randomized to reldesemtiv in Cohort 1 will receive a dose of 150 mg twice daily and patients randomized to reldesemtiv in Cohort 2 will receive 450 mg twice daily. Within each cohort, randomization will be stratified by ambulatory status (ambulatory versus non ambulatory). The primary objective of the study is to determine the PD effects of reldesemtiv on measures of pulmonary function, respiratory function, muscle strength, and motor function. Other PD measures include changes in the timed up and go (TUG) test, a 6-minute walk test (6MWT), and patient and investigator global assessments. Secondary objectives include the safety of multiple doses of reldesemtiv and an evaluation of the pharmacokinetics of reldesemtiv.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	Placebo	Granules for oral suspension (placebo)
DRUG	Reldesemtiv 150 mg	Granules for oral suspension, 18.7% reldesemtiv
DRUG	Reldesemtiv 450 mg	Granules for oral suspension, 56.0% reldesemtiv

Timeline

• Start date: 2016-01-14
• Primary completion: 2018-05-31
• Completion: 2018-05-31
• First posted: 2016-01-01
• Last updated: 2020-08-31
• Results posted: 2020-08-31

Locations

19 sites across 2 countries: United States, Canada