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

Launched by HOFFMANN-LA ROCHE · Mar 30, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a medication called risdiplam to see how it works and how safe it is for newborns with spinal muscular atrophy (SMA). SMA is a rare genetic condition that affects muscle strength and movement. The trial is open to infants who are less than 20 days old and have been diagnosed with SMA. To participate, babies need to be born after 37 weeks of pregnancy and be healthy enough to join the study without any serious medical issues. Parents should also be open to possible feeding tube placement if needed for the baby's nutrition and hydration during the study.

If your baby is eligible and joins the trial, they will receive risdiplam and be closely monitored by healthcare professionals for any side effects. The goal is to gather important information about how the medication is processed in the body and to ensure it is safe for infants with SMA. This study is currently recruiting participants, so if you think your baby might qualify, you can talk to your doctor for more information.

Gender

ALL

Eligibility 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