NCT02913482

Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants With Type 1 Spinal Muscular Atrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 62 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 1 Month – 7 Months
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Muscular Atrophy, Spinal

Interventions

Type	Name	Description
DRUG	Risdiplam	Risdiplam will be administered orally.

Timeline

• Start date: 2016-12-23
• Primary completion: 2019-11-14
• Completion: 2023-12-22
• First posted: 2016-09-23
• Last updated: 2024-08-07
• Results posted: 2021-01-08

Locations

32 sites across 16 countries: United States, Belgium, Brazil, China, Croatia, France, Italy, Japan, Poland, Russia, Saudi Arabia, Serbia, Spain, Switzerland, Turkey (Türkiye), Ukraine

Regulatory

• FDA-regulated drug study