This study is testing if adding a medication called ruxolitinib to a reduced intensity conditioning (RIC) regimen can help children and young adults with sickle cell disease (SCD) who are receiving a haploidentical (half-matched donor) stem cell transplant (HCT). The main goal is to see if ruxolitinib can reduce the chance of graft failure, which is when the transplanted cells don't take hold. This study is for people aged 12 to 45 with SCD who have had a stroke, vasculopathy, or recurrent severe acute chest syndrome. We will measure how many participants are alive and free of certain events one year after their transplant. The study plans to enroll up to 24 participants, but the current recruitment status is unclear.

Study design

This is a single-arm, multi-center, open-label study that will enroll up to 24 participants. It is a Phase 1/2 trial.

What's involved

Participants will receive hydroxyurea for at least 60 days before conditioning, followed by a RIC regimen and ruxolitinib. Ruxolitinib will continue after the transplant.

Compensation

Not stated in the trial record.

Follow-up

Participants will be followed for an additional 2 years after their transplant. Event-free survival will be measured at 1 year post-HCT.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.