Spinal Muscular Atrophy Clinical Trial
Official title:
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
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).
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.
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