clinical trial · NCT05808764
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Hoffmann-La Roche·phase2·recruiting·n = 10
Muscular Atrophy, SpinalRisdiplam
brief summary
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
started
Apr 26, 2024
primary completion
Nov 30, 2026
completion
Nov 30, 2026
last updated
Apr 3, 2026
official title
A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol