A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

PHASE2/PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

259

Participants

Timeline

Start Date

June 2, 2022

Primary Completion Date

February 27, 2029

Study Completion Date

February 27, 2029

Conditions
Spinal Muscular Atrophy (SMA)
Interventions
DRUG

RO7204239

"RO7204239 will administered every 4 weeks (Q4W) by subcutaneous (SC) injection into the abdomen.~RO7204239 will be investigated at low- and high-dose in Part 1."

DRUG

Placebo

Placebo will be administered Q4W by SC injection into the abdomen.

DRUG

Risdiplam

Risdiplam will be administered orally once daily (QD) for the duration of the study.

Trial Locations (35)

2031

Sydney Children's Hospital, Randwick

4000

Chr de La Citadelle, Liège

9000

UZ Gent, Ghent

10000

Clinical Hospital Centre Zagreb, Zagreb

10032

Columbia University Medical Center, New York

16147

IRCCS Istituto Giannina Gaslini, Genoa

20133

Fondazione IRCCS Istituto Neurologico ?Carlo Besta?, Milan

20162

Asst Grande Ospedale Metropolitano Niguarda, Milan

28046

Hospital Universitario La Paz, Madrid

32827

Nemours Children's Hospital, Orlando

46026

Hospital Universitario la Fe, Valencia

60126

Ospedali Riuniti Torrette di Ancona, Ancona

76208

Neurology & Neuromuscular Care Center, Denton

02115

Boston Childrens Hospital, Boston

V6H 3N1

British Columbia Children's Hospital, Vancouver

M5G 1X8

The Hospital for Sick Children, Toronto

H4A 3J1

McGill University Health Centre - Glen Site, Montreal

00165

Ospedale Pediatrico Bambino Gesù, Rome

00168

Policlinico Agostino Gemelli, Rome

650-0017

Kobe University Hospital, Hyōgo

890-8520

Kagoshima University Hospital, Kagoshima

162-0052

National Center for Global Health and Medicine, Tokyo

3584 CX

Universitair Medisch Centrum Utrecht, Utrecht

93-338

Instytut Centrum Zdrowia Matki Polki, ?ód?

80-952

Uniwersyteckie Centrum Kliniczne, Gda?sk

60-355

Uniwersytecki Szpital Kliniczny w Poznaniu, Późna

02-097

Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie, Warsaw

04-730

Instytut Pomnik Centrum Zdrowia Dziecka, Warsaw

1169-045

CHULC, E.P.E. - Hospital Dona Estefania, Lisbon

1649-035

Hospital de Santa Maria, Lisbon

08950

Hospital Sant Joan De Deu, Esplugues de Llobregas

08035

Hospital Vall d'Hebron, Barcelona

B9 5SS

Birmingham Heartlands Hospital, Birmingham

WC1N 3JH

Great Ormond Street Hospital For Children, London

OX3 9DU

John Radcliffe Hospital, Oxford

All Listed Sponsors
lead

Hoffmann-La Roche

INDUSTRY