A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Hoffmann-La Roche
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.
Eligibility
- Age range
- Up to 0 years
- Sex
- All
- Healthy volunteers
- No
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…