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Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
Sponsor: University of Colorado, Denver
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.
Official title: Ruxolitinib-Enhanced Conditioning for Pediatric and Young Adult Patients With Symptomatic Sickle Cell Disease Undergoing Haploidentical Hematopoietic Cell Transplantation
Key Details
Gender
All
Age Range
12 Years - 45 Years
Study Type
INTERVENTIONAL
Enrollment
24
Start Date
2026-01-01
Completion Date
2029-11-19
Last Updated
2025-11-26
Healthy Volunteers
No
Conditions
Interventions
Ruxolitinib
All participants will receive ruxolitinib beginning during conditioning in addition to conventional RIC and GVHD prophylaxis.
Locations (4)
Children's Hospital of Colorado
Aurora, Colorado, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Manning Family Children's
New Orleans, Louisiana, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States