Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease (NCT07252050) | Clinical Trial Compass
RecruitingPhase 1/2
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
United States24 participantsStarted 2026-04-10
Plain-language summary
This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD).
This study will enroll and treat up to 24 participants. Recruitment is expected to last for about 2 years and participants will be followed for an additional 2 years post-HCT.
Who can participate
Age range12 Years – 45 Years
SexALL
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Inclusion criteria
✓. Participants with any genotypic form of SCD aged 12 - 45 years at enrollment with ≥1 of the following:
✓. History of stroke and/or vasculopathy, including evidence of asymptomatic cerebrovascular disease for pediatric patients.
✓. Recurrent vaso-occlusive pain episodes requiring parenteral analgesia despite the institution of supportive care.
✓. Need for chronic transfusion therapy to prevent vaso-occlusive complications (i.e. pain, stroke, and ACS).
✓. For adult patients, an echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) ≥ 2.7 m/sec.
✓. Participants must have an HLA haploidentical first degree relative (parent, sibling, or half sibling) who is willing and able to donate bone marrow.
✓. Participants must meet institutional eligibility criteria for HCT.
Exclusion criteria
✕. Presence of an HLA-matched sibling who is willing and able to donate bone marrow.
✕. Uncontrolled infection, evidence of active TB, Hepatitis B or C infection, or HIV seropositivity or infection.
✕. Previous HCT or solid organ transplant.
✕. CNS revascularization procedure, myocardial infarction, pulmonary embolus or deep vein thrombosis in the past 6 months.