Actively Recruiting
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Led by Hoffmann-La Roche · Updated on 2026-05-04
10
Participants Needed
13
Research Sites
135 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
CONDITIONS
Official Title
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Newborn infants younger than 20 days at first dose
- Genetic diagnosis of 5q-autosomal recessive SMA or positive SMA diagnosis via newborn screening or prenatal testing
- Born at gestational age of 37 weeks or more
- Receiving adequate nutrition and hydration at screening
- Recovered from any acute illness and well enough to participate
- Parent or caregiver willing to consider feeding tube placement if recommended for safe treatment delivery
You will not qualify if you...
- Presence of clinical symptoms or signs consistent with SMA Type 0
- Inadequate venous or capillary blood access for study procedures as judged by investigator
- Abnormalities in systolic blood pressure, diastolic blood pressure, or heart rate
- Clinically relevant electrocardiogram (ECG) abnormalities
- Infant or breastfeeding person taking inhibitors or inducers of CYP3A4 or multidrug and toxin extrusion (MATE) substrates within specified timeframes prior to dosing
- Prior or concurrent use of nusinersen or onasemnogene abeparvovec
- Clinically significant laboratory abnormalities
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 13 locations
1
Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
2
University Of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
3
Clinic for Special Children.
Gordonville, Pennsylvania, United States, 17529
Actively Recruiting
4
Hopital Universitaire des Enfants Reine Fabiola
Brussels, Belgium, 1020
Actively Recruiting
5
CHR Citadelle
Liège, Belgium, 4000
Actively Recruiting
6
Children'S Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
7
Universitatsklinikum Essen
Essen, Germany, 45147
Actively Recruiting
8
Fondazione Serena Onlus - CENTRO CLINICO NEMO
Milano, Emilia-Romagna, Italy, 20162
Actively Recruiting
9
Fondazione Policlinico Univeristario A. Gemelli
ROMA, Emilia-Romagna, Italy, 00168
Actively Recruiting
10
UMC Utrecht
Utrecht, Netherlands, 3508
Actively Recruiting
11
OUS (Oslo University Hospital), Rikshospitalet
Oslo, Norway, 0372
Actively Recruiting
12
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland, 80-952
Actively Recruiting
13
Instytut Pomnik - Centrum Zdrowia Dziecka
Warsaw, Poland, 04-730
Actively Recruiting
Research Team
R
Reference Study ID Number: BN44619 https://forpatients.roche.com/
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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