A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
NCT ID: NCT05808764
Last Updated: 2026-01-09
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
10 participants
INTERVENTIONAL
2024-04-26
2026-11-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Risdiplam
Participants will receive risdiplam once daily for 28 days.
Risdiplam
Participants will receive 0.15 mg/kg risdiplam orally once daily for 28 days.
Interventions
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Risdiplam
Participants will receive 0.15 mg/kg risdiplam orally once daily for 28 days.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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
19 Days
ALL
No
Sponsors
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Hoffmann-La Roche
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trials
Role: STUDY_DIRECTOR
Hoffmann-La Roche
Locations
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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
Countries
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Central Contacts
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Reference Study ID Number: BN44619 https://forpatients.roche.com/
Role: CONTACT
Other Identifiers
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2023-505602-42-00
Identifier Type: CTIS
Identifier Source: secondary_id
BN44619
Identifier Type: -
Identifier Source: org_study_id
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