A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Phase 2 Completed 11 enrolled Hoffmann-La Roche

NaSingle-groupOpen-labelTreatment

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.

Timeline

Start: 2024-04-26
Primary completion: 2026-05-04
Completion: 2026-05-04

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Risdiplam	Small molecule	0.15 mg/kg	Oral

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Summary

Timeline

Drugs

Indications