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Clinical trial

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

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Last updated:24th Dec 2016
Identifier: NCT02913482

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 Type: Interventional  (Clinical Trial)
Estimated Enrollment: 48 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy
Actual Study Start Date: December 24, 2016
Estimated Primary Completion Date: November 17, 2023
Estimated Study Completion Date: November 17, 2023

- Experimental:
Part 1 (Dose Finding): Risdiplam (RO7034067)
- Experimental: Part 2 (Confirmatory): Risdiplam (RO7034067)

Category Value
Date last updated at source 2019-09-04
Study type(s) Interventional
Expected enrolment 48
Study start date 2016-12-24
Estimated primary completion date 2023-11-17

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