A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers
Completed
Hoffmann-La Roche
Phase 1
2016-01-07
The objective of this study is to assess the safety and tolerability of Risdiplam (RO7034067)
in healthy people. The study will assess what the body does to Risdiplam (RO7034067) and what
Risdiplam (RO7034067) does to the body. Risdiplam (RO7034067) will be given by mouth in
gradually increasing doses. The data from this study will help to define the dose to further
explore Risdiplam (RO7034067) in patients with Spinal Muscular Atrophy.
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
Active, not recruiting
Hoffmann-La Roche
Phase 2/Phase 3
2016-10-20
Multi-center, randomized, double-blind, placebo-controlled study to assess the safety,
tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and
pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an
exploratory dose finding part (Part 1) of Risdiplam for 12 weeks and a confirmatory part
(Part 2) of Risdiplam for 24 months.
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Active, not recruiting
Hoffmann-La Roche
Phase 2/Phase 3
2016-12-24
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.
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