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

Part of paid clinical trials in Aurora, Colorado.

Sponsor
Arkansas Children's Hospital Research Institute
Study ID
NCT07252050
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Graft Failure
  • Haploidentical Hematopoietic Stem Cell Transplant
  • Haploidentical Stem Cell Transplantation
  • Hematopoetic Stem Cell Transplant
  • Sickle Cell Disease

Eligibility Criteria

Sex
ALL
Age
12 Years - 45 Years
Healthy Volunteers
Not accepted

Interventions

  • Ruxolitinib — DRUG
    All participants will receive ruxolitinib beginning during conditioning in addition to conventional RIC and GVHD prophylaxis.

Study Details

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.

Key Dates

Start date
Jun 8, 2026
Status verified
Jun 2026
Primary completion
Nov 19, 2028
Completion
Nov 19, 2029

Study Design

Enrollment
24 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Ruxolitinib-Enhanced RIC
    Pediatric and young adult participants who are undergoing haplo HCT for SCD will receive RIC with fludarabine, cyclophosphamide, thiotepa, ATG and low-dose TBI along with ruxolitinib. Ruxolitinib will continue post-HCT in addition to post-transplant cyclophosphamide and sirolimus or a calcineurin inhibitor for GVHD prophylaxis.

Primary Outcome Measure

Event Free Survival [ Time Frame: 1 year post-HCT ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
Children's Hospital of ColoradoAuroraColorado80045
Chris McKinney, MD (PRINCIPAL_INVESTIGATOR)
Children's Healthcare of AtlantaAtlantaGeorgia30329
Julia Gliwinski
[email protected]
(404) 785-5532
Elizabeth Stenger, MD (PRINCIPAL_INVESTIGATOR)
Manning Family Children'sNew OrleansLouisiana70118
Wyvonnia Walker
[email protected]
504-988-9378
Maria Pereda Ginocchio, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Nykirah Turner
[email protected]
Timothy Olson, MD, PhD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Aurora, CO

By condition
Sickle cell disease
By research site
Children's Hospital of Colorado· Aurora, COChildren's Healthcare of Atlanta· Atlanta, GAManning Family Children's· New Orleans, LAChildren's Hospital of Philadelphia· Philadelphia, PA

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