NCT03779334View on ClinicalTrials.gov

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

PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

26

Participants

Timeline

Start Date

August 7, 2019

Primary Completion Date

February 20, 2023

Study Completion Date

March 31, 2027

Conditions
Muscular Atrophy, Spinal
Interventions
DRUG

Risdiplam

Risdiplam will be administered orally.

Trial Locations (7)

807

Kaohsiung Medical University Chung-Ho Hospital, Kaohsiung City

2031

Sydney Children's Hospital, Randwick

4000

Chr de La Citadelle, Liège

32837

Nemours Children's Hospital, Orlando

125412

Russian Children Neuromuscular Center of Veltischev, Moscow

05403-000

Hospital das Clinicas - FMUSP_X, São Paulo

80-952

Szpital Gdanskiego Uniwersytetu Medycznego, Gda?sk

All Listed Sponsors
lead

Hoffmann-La Roche

INDUSTRY

NCT03779334 - A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | Biotech Hunter | Biotech Hunter