A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

PHASE2RecruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

April 26, 2024

Primary Completion Date

April 17, 2026

Study Completion Date

April 17, 2026

Conditions
Muscular Atrophy, Spinal
Interventions
DRUG

Risdiplam

Participants will receive 0.15 mg/kg risdiplam orally once daily for 28 days.

Trial Locations (13)

1020

RECRUITING

Hopital Universitaire des Enfants Reine Fabiola, Brussels

3508

RECRUITING

UMC Utrecht, Utrecht

4000

RECRUITING

CHR Citadelle, Liège

17529

RECRUITING

Clinic for Special Children., Gordonville

20162

RECRUITING

Fondazione Serena Onlus - CENTRO CLINICO NEMO, Milano

45147

RECRUITING

Universitatsklinikum Essen, Essen

48109

RECRUITING

University Of Michigan, Ann Arbor

60611

RECRUITING

Ann and Robert H. Lurie Children Hospital of Chicago, Chicago

K1H 8L1

RECRUITING

Children'S Hospital of Eastern Ontario, Ottawa

00168

RECRUITING

Fondazione Policlinico Univeristario A. Gemelli, ROMA

0372

RECRUITING

OUS (Oslo University Hospital), Rikshospitalet, Oslo

80-952

RECRUITING

Uniwersyteckie Centrum Kliniczne, Gda?sk

04-730

RECRUITING

Instytut Pomnik - Centrum Zdrowia Dziecka, Warsaw

All Listed Sponsors
lead

Hoffmann-La Roche

INDUSTRY