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

Study Identifier:
BN44619
ClinicalTrials.gov Identifier:
NCT05808764
EudraCT Identifier:
N/A
EU CT Number:
2023-505602-42-00
Sponsor:
Hoffmann-La Roche
Recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Risdiplam
Date
Apr 2024 - Nov 2026
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: N/A - 19 Days
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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.
Exclusion Criteria
  • 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

Protocol Summary

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.

Trial Locations

Location
Status
Location
Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois, United States, 60611
Status
Recruiting
Location
University Of Michigan
Ann Arbor, Michigan, United States, 48109
Status
Recruiting
Location
Clinic for Special Children.
Gordonville, Pennsylvania, United States, 17529
Status
Recruiting
Location
Hopital Universitaire des Enfants Reine Fabiola
Brussels, Belgium, 1020
Status
Recruiting
Location
CHR Citadelle
Liège, Belgium, 4000
Status
Recruiting
Location
Children'S Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Status
Recruiting
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