A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

PHASE2CompletedINTERVENTIONAL
Enrollment

174

Participants

Timeline

Start Date

March 3, 2017

Primary Completion Date

February 7, 2025

Study Completion Date

February 7, 2025

Conditions
Spinal Muscular Atrophy
Interventions
DRUG

Risdiplam

Risdiplam will be administered orally once daily.

Trial Locations (24)

3000

UZ Leuven Gasthuisberg, Leuven

4005

Universitäts-Kinderspital (UKBB) Neuropädiatrie, Basel

9000

UZ Gent, Ghent

10032

Columbia University Medical Center, New York

16147

IRCCS Istituto Giannina Gaslini, Genoa

20122

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan

31059

Hopital des Enfants, Toulouse

32827

Nemours Children's Hospital, Orlando

34295

CHRU de Montpellier, Hopital Gui de Chauliac, Montpellier

45147

Universitätsklinikum Essen, Essen

59037

Hopital Roger Salengro, Lille

69677

Hopital Femme Mere Enfant, Bron

75015

Hôpital Necker-Enfants Malades, Paris

79106

Universitätsklinikum Freiburg, Freiburg im Breisgau

94304

Stanford University Medical Center, Palo Alto

98125

UOSD Malattie Neurodegenerative, Messina

02115

Boston Childrens Hospital, Boston

00165

IRCCS Ospedale Pediatrico Bambino Gesù, Rome

00168

Policlinico Agostino Gemelli, Rome

3584 CX

UMC Utrecht, Utrecht

02-097

Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie, Warsaw

B9 5SS

Birmingham Heartlands Hospital, Birmingham

WC1N 1EH

UCL Institute of Child Health & Great Ormond Street Hospital for Children, London

NE1 4LP

The Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne

All Listed Sponsors
lead

Hoffmann-La Roche

INDUSTRY

NCT03032172 - A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy | Biotech Hunter | Biotech Hunter