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

No results posted yet for this study

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

Ruxolitinib

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
  • Incyte Corporation

    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

More Related Trials

Entities

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07252050 on ClinicalTrials.gov