Trial Profile

A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Status: Recruiting

Phase of Trial: Phase II

Latest Information Update: 06 Mar 2025

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At a glance

Drugs Risdiplam (Primary)
Indications Spinal muscular atrophy
Focus Adverse reactions; Pharmacokinetics
Acronyms PUPFISH
Sponsors Roche

Most Recent Events

28 Feb 2025 Planned End Date changed from 31 Aug 2025 to 30 Oct 2025.
28 Feb 2025 Planned primary completion date changed from 31 Aug 2025 to 30 Oct 2025.
10 May 2024 Status changed from not yet recruiting to recruiting.

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