NCT07252050

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

Ruxolitinib-Enhanced Conditioning for Pediatric and Young Adult Patients With Symptomatic Sickle Cell Disease Undergoing Haploidentical Hematopoietic Cell Transplantation

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: University of Colorado, Denver · Academic / Other
• Sex: All
• Age: 12 Years – 45 Years
• Healthy volunteers: Not accepted

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.

Detailed description

While haplo HCT following a RIC regimen cures most patients with SCD, graft failure (GF) can occur and result in return of SCD. GF occurs more often in pediatric SCD patients and can be associated with significant morbidity and/or mortality. Development of strategies which reduce the risk of GF is needed to further improve haplo HCT outcomes for SCD, particularly in pediatric patients. This trial hopes to demonstrate that addition of ruxolitinib to a RIC regimen will reduce the incidence of GF without increasing conditioning-related toxicities. The RUX-HAPLO study is a Phase 1/2 single-arm, multi-center, open-label trial for pediatric and young adult patients undergoing haplo HCT for SCD. The study will enroll up to 24 participants over approximately 2 years. All participants will receive cytoreduction with hydroxyurea (HU) for at least 60 days (Day -70 to Day -10) prior to the start of conditioning. All participants will then receive a RIC regimen consisting of cyclophosphamide, fludarabine, thiotepa, ATG and TBI beginning on Day -9. Ruxolitinib will begin during conditioning and will continue post-HCT. Participants will also receive GVHD prophylaxis with post-transplant cyclophosphamide, in addition to sirolimus or a calcineurin inhibitor. The primary objective is to estimate 1-year event-free survival (EFS) with primary or secondary GF or death counting as events for this endpoint.

Conditions

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

Interventions

Type	Name	Description
DRUG	Ruxolitinib	All participants will receive ruxolitinib beginning during conditioning in addition to conventional RIC and GVHD prophylaxis.

Timeline

• Start date: 2026-01-01
• Primary completion: 2028-11-19
• Completion: 2029-11-19
• First posted: 2025-11-26
• Last updated: 2025-11-26

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study