We know that babies with hypoplastic left heart syndrome (HLHS) commonly struggle to grow and gain weight. We also know that growth failure is related to worse outcomes. The main reason for this research study was to learn if a medicine called oxandrolone is safe and well tolerated in babies with HLHS or other related single right ventricular (RV) anomalies who have undergone a Norwood procedure. We also wanted to see if it also helped babies with HLHS to grow better.
In Oxandrolone Perceptions, participants and their families were invited to complete a questionnaire about their experiences being in the study.
The trial randomized 34 infants up to 14 days old with HLHS or other single right ventricular anomalies who underwent a Norwood procedure and did not meet any of the study exclusion criteria. The trial stopped because the FDA withdrew new drug applications and abbreviated new drug applications for oxandrolone tablets effective June 28, 2023, and the manufacturer subsequently decided to discontinue production of the medication.
Infants were randomly assigned to either 28-days of oxandrolone or no oxandrolone (standard of care). The trial planned a cautious dose-escalation over 3 consecutive cohorts (25 infants each). The growth and feeding history of the infants were monitored until they were discharged from the hospital following their second stage surgery (approximately 4-6 months).
The trial ended too soon (in the middle of the 2nd dosing cohort) to draw any conclusions, but no harmful effects of oxandrolone were observed. Check back soon for a summary of other findings from the study.