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ACTIVE NOT RECRUITING
NCT03779334
PHASE2

A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Sponsor: Hoffmann-La Roche

View on ClinicalTrials.gov

Summary

A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).

Official title: An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Key Details

Gender

All

Age Range

1 Day - 6 Weeks

Study Type

INTERVENTIONAL

Enrollment

26

Start Date

2019-08-07

Completion Date

2027-02-28

Last Updated

2026-04-08

Healthy Volunteers

No

Interventions

DRUG

Risdiplam

Risdiplam will be administered orally.

Locations (7)

Nemours Children's Hospital

Orlando, Florida, United States

Sydney Children's Hospital

Randwick, New South Wales, Australia

Chr de La Citadelle

Liège, Belgium

Hospital das Clinicas - FMUSP_X

São Paulo, São Paulo, Brazil

Szpital Gdanskiego Uniwersytetu Medycznego

Gda?sk, Poland

Russian Children Neuromuscular Center of Veltischev

Moscow, Moscow Oblast, Russia

Kaohsiung Medical University Chung-Ho Hospital

Kaohsiung City, Taiwan