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A Phase II, Open-label 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
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the pharmacokinetics and safety of risdiplam in infants with spinal muscular atrophy (SMA) who are less than 20 days old at their first dose. This Phase II, open-label study focuses on how the drug behaves in the body and monitors any safety concerns in this very young population. The study is sponsored by Hoffmann-La Roche and targets newborn infants diagnosed genetically with 5q-autosomal recessive SMA or identified through newborn screening or prenatal testing. Participants will receive risdiplam orally once daily at a dose of 0.15 mg/kg for 28 days. The treatment period is straightforward, with all infants receiving the same study drug without placebo or comparator groups. Caregivers may be asked to consider feeding tube placement if necessary to ensure safe hydration, nutrition, and treatment delivery during the study. During the study, researchers will collect blood samples to measure plasma concentrations of risdiplam and analyze key pharmacokinetic parameters such as area under the curve (AUC), steady-state concentration, and free fraction of the drug. Safety will be closely monitored through assessing adverse events up to 30 days after the last dose, including serious events and treatment discontinuations due to side effects. The total observation period may last up to 58 days. This detailed monitoring aims to provide important information on how risdiplam is processed and tolerated in newborns with SMA.
CONDITIONS
Brief 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...
- Male or female newborn infant aged <20 days at first dose
- Genetic diagnosis of 5q-autosomal recessive SMA or positive for SMA via newborn screening or prenatal testing
- Gestational age equal to or greater than 37 weeks
- Receiving adequate nutrition and hydration at screening
- Adequately recovered from any acute illness and well enough to participate
- Parent/caregiver willing to consider feeding tube placement if recommended
You will not qualify if you...
- Clinical symptoms or signs consistent with SMA Type 0
- Inadequate venous or capillary blood access for study procedures
- Abnormal systolic or diastolic blood pressure or heart rate
- Clinically relevant electrocardiogram (ECG) abnormalities
- Current or recent use of CYP3A4 inhibitors, inducers, or MATE substrates as specified
- Previous or concurrent treatment with nusinersen or onasemnogene abeparvovec
- Clinically significant laboratory abnormalities
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 28 days
Participants receive risdiplam orally once daily for 28 days.
Daily dosing with assessments during the treatment period
Duration - Up to 30 days after treatment
Participants are monitored for safety and adverse events for up to 30 days after the final dose of study treatment.
Approximately 1 to 2 follow-up visits
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
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4
Hopital Universitaire des Enfants Reine Fabiola
Brussels, Belgium, 1020
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5
CHR Citadelle
Liège, Belgium, 4000
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6
Children'S Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
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7
Universitatsklinikum Essen
Essen, Germany, 45147
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8
Fondazione Serena Onlus - CENTRO CLINICO NEMO
Milano, Emilia-Romagna, Italy, 20162
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9
Fondazione Policlinico Univeristario A. Gemelli
ROMA, Emilia-Romagna, Italy, 00168
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10
UMC Utrecht
Utrecht, Netherlands, 3508
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11
OUS (Oslo University Hospital), Rikshospitalet
Oslo, Norway, 0372
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12
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland, 80-952
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13
Instytut Pomnik - Centrum Zdrowia Dziecka
Warsaw, Poland, 04-730
Actively Recruiting
Research Team
R
Reference Study ID Number: BN44619 https://forpatients.roche.com/
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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