Evaluating Crizanlizumab Versus Placebo in Adolescents and Adults with Sickle Cell Disease and Frequent Vaso-Occlusive Crises

What this Trial Is About

Researchers are investigating the effects of crizanlizumab compared to a placebo in adolescents and adults with Sickle Cell Disease who experience frequent vaso-occlusive crises VOCs. This Phase III, randomized, double-blind study involves patients aged 12 years and older who have had 4 to 12 VOCs managed by healthcare professionals in the past year. The study evaluates the safety and effectiveness of crizanlizumab with or without standard hydroxyureahydroxycarbamide therapy. Participants are randomly assigned in a 21 ratio to receive either crizanlizumab at a dose of 5 mgkg or a placebo, both given alongside standard care. The treatment is administered intravenously as a concentrate for infusion. The study is stratified by hydroxyurea use and geographical region to ensure balanced groups. The main treatment period lasts for one year. During the study, participants will attend regular visits for treatment and monitoring. Researchers will assess the number of healthcare-managed VOCs, including those treated in person or via remote consultation, and measure various other outcomes such as time to first VOC, VOC duration, antibody development to crizanlizumab, adverse events, and changes in hemoglobin levels. Safety and efficacy will be observed over two years, with detailed documentation of VOC events and other health assessments throughout participation.

A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)