Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
NCT07252050 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 24
Last updated 2026-05-15
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.
Conditions
- Sickle Cell Disease
- Hematopoetic Stem Cell Transplant
- Haploidentical Hematopoietic Stem Cell Transplant
- Haploidentical Stem Cell Transplantation
- Graft Failure
Interventions
- DRUG
-
All participants will receive ruxolitinib beginning during conditioning in addition to conventional RIC and GVHD prophylaxis.
Sponsors & Collaborators
-
Children's Healthcare of Atlanta
collaborator OTHER -
Emory University
collaborator OTHER - collaborator INDUSTRY
-
University of Colorado, Denver
lead OTHER
Principal Investigators
-
Laura McLaughlin, MD · University of Colorado, Denver
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 12 Years
- Max Age
- 45 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2026-05-31
- Primary Completion
- 2028-11-19
- Completion
- 2029-11-19
- FDA Drug
- Yes
Countries
- United States
Study Locations
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