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

Completed · Phase 2

Conditions studied: Muscular Atrophy, Spinal

In brief

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.

Key facts

Study ID

NCT05808764

Run by

Hoffmann-La Roche

People needed

11

Starts

2024-04-26

Expected to finish

2026-05-04

Last updated by the study team

2026-06-22

Who can join

Age: any, up to 0. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female newborn infant aged <20 days at first dose
• Newborn infants with genetic diagnosis of 5q-autosomal recessive SMA or newborn infants identified as positive for SMA via newborn screening or via prenatal testing.
• Gestational age equal to or greater than 37 weeks
• Receiving adequate nutrition and hydration at the time of screening
• Adequately recovered from any acute illness at baseline and considered well enough to participate in the study
• Parent/caregiver is willing to consider nasogastric, nasojejunal, or gastrostomy tube placement during the study to maintain safe hydration, nutrition, and treatment delivery, if recommended by the investigator.

You may not qualify if…

• Presence of clinical symptoms or signs consistent with SMA Type 0
• In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures
• Systolic blood pressure or diastolic blood pressure or heart rate abnormalities
• Presence of clinically relevant electrocardiogram (ECG) abnormalities
• The infant (or the person breastfeeding the infant) taking any of the following: any inhibitor of CYP3A4 taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing, any inducer of CYP3A4 taken within 4 weeks (or within 5 times the elimination half-life, whichever is longer prior to dosing, and/or use of any multidrug and toxin extrusion (MATE) substrates taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing
• Concurrent or previous administration of nusinersen or onasemnogene abeparvovec
• Clinically significant abnormalities in laboratory test

Where it is running

• Ann and Robert H. Lurie Children Hospital of Chicago — Chicago, Illinois, United States
• University Of Michigan — Ann Arbor, Michigan, United States
• Clinic for Special Children. — Gordonville, Pennsylvania, United States
• Hopital Universitaire des Enfants Reine Fabiola — Brussels, Belgium
• CHR Citadelle — Liège, Belgium
• Children'S Hospital of Eastern Ontario — Ottawa, Ontario, Canada
• Universitatsklinikum Essen — Essen, Germany
• Fondazione Serena Onlus - CENTRO CLINICO NEMO — Milano, Emilia-Romagna, Italy
• Fondazione Policlinico Univeristario A. Gemelli — ROMA, Emilia-Romagna, Italy
• UMC Utrecht — Utrecht, Netherlands
• OUS (Oslo University Hospital), Rikshospitalet — Oslo, Norway
• Uniwersyteckie Centrum Kliniczne — Gdansk, Poland
• Instytut Pomnik - Centrum Zdrowia Dziecka — Warsaw, Poland

Full record on ClinicalTrials.gov

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