Actively Recruiting
Ruxolitinib-Enhanced Conditioning for Pediatric and Young Adult Patients With Symptomatic Sickle Cell Disease Undergoing Haploidentical Hematopoietic Cell Transplantation
Led by Arkansas Children's Hospital Research Institute · Updated on 2026-06-04
24
Participants Needed
4
Research Sites
52 weeks
Total Duration
On this page
Sponsors
A
Arkansas Children's Hospital Research Institute
Lead Sponsor
C
Children's Healthcare of Atlanta
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating whether adding the drug ruxolitinib to a reduced intensity conditioning (RIC) regimen can lower the chance of graft failure after haploidentical hematopoietic cell transplant (HCT) in children and young adults with sickle cell disease (SCD). Graft failure is a serious problem especially in pediatric patients and can lead to the return of SCD symptoms. This Phase 1/2 open-label trial aims to improve transplant success while keeping treatment side effects manageable. All participants will first receive hydroxyurea for at least 60 days before starting the conditioning regimen. Then, they will undergo RIC with cyclophosphamide, fludarabine, thiotepa, ATG, and low-dose total body irradiation beginning on Day -9. Ruxolitinib treatment will start during conditioning and continue after transplant. To help prevent graft versus host disease, participants will receive post-transplant cyclophosphamide plus either sirolimus or a calcineurin inhibitor. Participants will be followed for up to two years after transplant to monitor their health and treatment effects. The study team will assess event-free survival at one year, considering graft failure or death as events. Additional outcomes include overall survival, blood cell recovery, graft rejection, graft versus host disease, organ toxicities, infections, and sickle cell-related complications. Up to 24 participants aged 12 to 45 years will be enrolled and closely monitored throughout the study.
CONDITIONS
Brief Title
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ages 12 to 45 years with any form of sickle cell disease
- History of stroke, cerebrovascular disease, recurrent acute chest syndrome, frequent pain episodes, chronic transfusion needs, or elevated echocardiographic tricuspid valve regurgitant jet velocity
- Have a haploidentical first-degree relative willing and able to donate bone marrow
- Meet institutional requirements for hematopoietic cell transplant eligibility
- Willing and able to comply with study procedures and informed consent
- Use highly effective contraception if of child-bearing potential
You will not qualify if you...
- Have an HLA-matched sibling donor available for bone marrow donation
- Active uncontrolled infection, including tuberculosis, hepatitis B or C, or HIV
- Previous hematopoietic cell or solid organ transplant
- Recent major cardiovascular or thrombotic events within 6 months
- Use medications that interfere with ruxolitinib metabolism
- Known severe allergy to ruxolitinib or conditioning regimen components
- Unable to swallow and retain oral medication (except via feeding tubes)
- History of malignancy except specific treated skin cancers
- Participated in another investigational drug or device trial within past 3 months
- Currently pregnant or breastfeeding
- Significant uncontrolled autoimmune disease
- High levels of anti-donor specific HLA antibodies without approval
- Unable or unwilling to comply with study dosing and evaluations
- Any condition posing significant risk or interfering with study participation as judged by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - At least 60 days
Participants receive cytoreduction with hydroxyurea for at least 60 days before the start of conditioning.
Visits as per routine care for hydroxyurea management
Duration - Conditioning starting Day -9 through post-transplant period up to 1 year
Participants receive reduced intensity conditioning with cyclophosphamide, fludarabine, thiotepa, ATG, and low-dose TBI along with ruxolitinib starting during conditioning and continuing post-transplant. GVHD prophylaxis with post-transplant cyclophosphamide and sirolimus or a calcineurin inhibitor is also administered.
Multiple visits during conditioning and frequent visits post-transplant for monitoring and treatment
Duration - Up to 2 years post-transplant
Participants are monitored for event-free survival, graft failure, GVHD, recovery, and other complications up to 2 years post-transplant.
Regular follow-up visits throughout the 2-year period
Trial Site Locations
Total: 4 locations
1
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
Not Yet Recruiting
2
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
Actively Recruiting
3
Manning Family Children's
New Orleans, Louisiana, United States, 70118
Not Yet Recruiting
4
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
Research Team
L
Laura McLaughlin, MD
K
Kayla Ortiz
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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