Adding Ruxolitinib to Reduced Intensity Conditioning in Haploidentical Stem Cell Transplant for Children and Young Adults with Sickle Cell Disease

What this Trial Is About

Researchers are evaluating whether adding the drug ruxolitinib to a reduced intensity conditioning RIC regimen can lower the chance of graft failure after haploidentical hematopoietic cell transplant HCT in children and young adults with sickle cell disease SCD. Graft failure is a serious problem especially in pediatric patients and can lead to the return of SCD symptoms. This Phase 12 open-label trial aims to improve transplant success while keeping treatment side effects manageable. All participants will first receive hydroxyurea for at least 60 days before starting the conditioning regimen. Then, they will undergo RIC with cyclophosphamide, fludarabine, thiotepa, ATG, and low-dose total body irradiation beginning on Day -9. Ruxolitinib treatment will start during conditioning and continue after transplant. To help prevent graft versus host disease, participants will receive post-transplant cyclophosphamide plus either sirolimus or a calcineurin inhibitor. Participants will be followed for up to two years after transplant to monitor their health and treatment effects. The study team will assess event-free survival at one year, considering graft failure or death as events. Additional outcomes include overall survival, blood cell recovery, graft rejection, graft versus host disease, organ toxicities, infections, and sickle cell-related complications. Up to 24 participants aged 12 to 45 years will be enrolled and closely monitored throughout the study.

Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease