Oxbryta™ to enter post-approval confirmatory trial for reducing stroke risk in children with sickle cell disease

Oxbryta™ is an oral medication for the treatment of sickle cell disease (SCD) by directly inhibiting sickle haemoglobin polymerisation. As a condition for having received accelerated approval from the FDA, Oxbryta™ will be assessed in a post-approval confirmatory study, called HOPE-KIDS 2, which is set to launch by the end of the year. The trial will test Oxbryta™ tablets at a daily dose of 1500 mg, its approved dose, in pediatric patients (age 2 to 15) with SCD, and its primary goal will be to demonstrate that treatment with Oxbryta™ can reduce the risk of stroke in SCD children at 24 weeks of treatment. The trial will use transcranial doppler (TCD) to measure flow velocity. The trial will run at 50 sites across the U.S., Europe, and Africa. For more info see here.