Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
About the study
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate Risdiplam (RO7034067) for 24-months at the dose selected in Part 1.
Study Locations
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How to Apply
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Study’s details
Contition
Muscular Atrophy, Spinal
Age (in years)
1 - 7
Phase
Phase 2
Participants needed
62
Est. Completion Date
Dec 22, 2023
Treatment type
Interventional
Sponsor
Hoffmann-La Roche
ClinicalTrials.gov identifier
NCT02913482
Study number
BP39056
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