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Clinical Trial NCT07252050 (RUX-HAPLO) for Sickle Cell Disease, Hematopoetic Stem Cell Transplant, Haploidentical Hematopoietic Stem Cell Transplant, Haploidentical Stem Cell Transplantation, Graft Failure is not yet recruiting. See the Trial Radar Card View and AI discovery tools for all the details. Or ask anything here.
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Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease (RUX-HAPLO) Phase 1, Phase 2 24

Not yet recruiting
Clinical Trial NCT07252050 (RUX-HAPLO) is designed to study Treatment for Sickle Cell Disease, Hematopoetic Stem Cell Transplant, Haploidentical Hematopoietic Stem Cell Transplant, Haploidentical Stem Cell Transplantation, Graft Failure. This Phase 1 Phase 2 interventional study is not yet recruiting. Enrollment is planned to begin on January 1, 2026 until the study accrues 24 participants. Led by University of Colorado, Denver, this study is expected to complete by November 19, 2029. The latest data from ClinicalTrials.gov was last updated on November 26, 2025.
Brief 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 addi...

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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...Show More
Official Title

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

Conditions
Sickle Cell DiseaseHematopoetic Stem Cell TransplantHaploidentical Hematopoietic Stem Cell TransplantHaploidentical Stem Cell TransplantationGraft Failure
Other Study IDs
  • RUX-HAPLO
  • 24-0735
NCT ID Number
NCT07252050
Start Date (Actual)
2026-01-01
Last Update Posted
2025-11-26
Completion Date (Estimated)
2029-11-19
Enrollment (Estimated)
24
Study Type
Interventional
PHASE
Phase 1
Phase 2
Status
Not yet recruiting
Keywords
Sickle cell disease
Hematopoietic cell transplant
Ruxolitinib
Pediatric
Young Adult
Haplo
Primary Purpose
Treatment
Design Allocation
N/A
Interventional Model
Single Group
Masking
None (Open Label)
Arms / Interventions
Participant Group/ArmIntervention/Treatment
ExperimentalRuxolitinib-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.
Ruxolitinib
All participants will receive ruxolitinib beginning during conditioning in addition to conventional RIC and GVHD prophylaxis.
Primary Outcome Measures
Outcome MeasureMeasure DescriptionTime Frame
Event Free Survival
Event Free Survival (EFS) is defined as survival without a qualifying event (primary or secondary GF, second HCT or death).
1 year post-HCT
Secondary Outcome Measures
Outcome MeasureMeasure DescriptionTime Frame
Overall Survival
Overall survival will be described at 1 and 2 years post-HCT including death from any cause after HCT.
1 and 2 years post-HCT
Event Free Survival
Event Free Survival (EFS) is defined as survival without a qualifying event (primary or secondary GF, second transplant or death).
2 years post-HCT
Neutrophil Recovery
The time to neutrophil recovery, in days, will be reported. Neutrophil recovery is defined as the first of 3 measurements on different days when the absolute neutrophil count is ≥500/μL after nadir.
Up to Day 60 post-HCT
Platelet Recovery
The time to platelet recovery, in days, will be reported. Platelet recovery is defined as the first day the platelet count is ≥50,000/μL of blood, without a transfusion in the preceding 7 days with the exception of a platelet transfusion specifically to achieve a platelet threshold to allow an elective invasive procedure.
Up to Day 180 post-HCT
Acute GVHD
Incidence of overall and severe (Grade 3-4) acute GVHD (based on MAGIC criteria) will be estimated at until Day 100 post-HCT.
Up to Day +100 post-HCT
Chronic GVHD
Incidence of overall and severe chronic GVHD (according to the NIH consensus criteria) will be estimated at 6 months, 1 year, 18 months and 2 years post-HCT.
6 months to 2 years post-HCT
Donor hematopoietic chimerism
Characterization of donor chimerism in peripheral blood for lymphoid and myeloid fractions will be performed at day 28, 60, 100, and 180 and 1 and 2 years post-HCT.
Day 28 to 2 years post-HCT
Primary Graft Failure
The incidence of primary graft failure (GF) by day 42 post-HCT will be estimated. Primary GF is defined as never achieving ≥ 5% donor whole blood or myeloid chimerism. Second infusion of stem cells is also considered indicative of primary GF.
Day 42 post-HCT
Secondary Graft Failure
The incidence of secondary graft failure (GF) by 2 years post-HCT will be estimated. Secondary GF is defined as \< 5% donor whole blood or myeloid chimerism beyond day +42 post-HCT in participants with prior documentation of hematopoietic recovery with \> 5% donor cells by day +42 post-HCT. Second infusion of stem cells beyond Day +42 is also considered indicative of secondary GF.
Up to 2 years post-HCT
Hepatic VOD/SOS
The incidence of hepatic veno-occlusive disease (VOD)/sinusoidal obstruction by 2 years post-HCT will be estimated.
Up to 2 years post-HCT
IPS
The incidence of idiopathic pneumonia syndrome (IPS) by 2 years post-HCT will be estimated.
Up to 2 years post-HCT
CNS Toxicity
The incidence of CNS toxicity, defined as seizures, intracranial hemorrhage (ICH), posterior reversible encephalopathy syndrome (PRES) or reversible posterior leukoencephalopathy syndrome (RPLS) will be estimated.
Up to 2 years post-HCT
Significant infections
The incidence of cytomegalovirus (CMV) infection, adenovirus infection, Epstein-Barr Virus (EBV) post-transplant lymphoproliferative disease (PTLD), or other clinically significant viral reactivations, invasive fungal infections and bacterial sepsis will be estimated.
Up to 2 years post-HCT
Prolonged Immunosuppressive Therapy
The proportion of participants receiving immunosuppressive therapy beyond 1 year post-HCT because of GVHD or concerns about graft rejection will be determined.
Up to 2 years post-HCT
SCD-related Complications
SCD-related complications at 6 months, 1 and 2 years post-HCT will be described.
Up to 2 years post-HCT
Participation Assistant
Eligibility Criteria

Eligible Ages
Child, Adult
Minimum Age
12 Years
Eligible Sexes
All

  1. Participants with any genotypic form of SCD aged 12 - 45 years at enrollment with ≥1 of the following:

    1. History of stroke and/or vasculopathy, including evidence of asymptomatic cerebrovascular disease for pediatric patients.
    2. Recurrent moderate-severe acute chest syndrome (ACS)
    3. Recurrent vaso-occlusive pain episodes requiring parenteral analgesia despite the institution of supportive care.
    4. Need for chronic transfusion therapy to prevent vaso-occlusive complications (i.e. pain, stroke, and ACS).
    5. For adult patients, an echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) ≥ 2.7 m/sec.

  2. Participants must have an HLA haploidentical first degree relative (parent, sibling, or half sibling) who is willing and able to donate bone marrow.

  3. Participants must meet institutional eligibility criteria for HCT.

  1. Presence of an HLA-matched sibling who is willing and able to donate bone marrow.
  2. Uncontrolled infection, evidence of active TB, Hepatitis B or C infection, or HIV seropositivity or infection.
  3. Previous HCT or solid organ transplant.
  4. CNS revascularization procedure, myocardial infarction, pulmonary embolus or deep vein thrombosis in the past 6 months.
  5. Use of medications which significantly interfere with ruxolitinib metabolism.
  6. Known hypersensitivity or severe reaction to ruxolitinib or any component of the conditioning regimen or its excipients.
  7. Inability to swallow and retain oral medication (use of nasogastric or gastrostomy tube permitted).
  8. History of malignancy except resected basal cell carcinoma or treated carcinoma in-situ.
  9. Participation in another clinical trial involving an investigational or off-label use of a drug or device in the past 3 months.
  10. Currently pregnant or breast feeding.
  11. Clinically significant, uncontrolled autoimmune disease.
  12. High-titer anti-donor specific HLA antibodies (without review and approval by Study Chair).
  13. Participant (or guardian) inability or unwillingness to comply with the dose schedule and study evaluations, comprehend or sign informed consent and utilize a highly effective method of contraception (for participants of child-bearing potential).
  14. Any condition that would, in the investigator's judgment, interfere with full participation in the study, pose a significant risk to the subject, or interfere with interpretation of study data.
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Study Responsible Party
Laura McLaughlin, Principal Investigator, Lead Principal Investigator/Study Chair, University of Colorado, Denver
Study Central Contact
Contact: Laura McLaughlin, MD, 720-777-7008, [email protected]
Contact: Kayla Ortiz, 720-777-4151, [email protected]
4 Study Locations in 1 Countries

Colorado

Children's Hospital of Colorado, Aurora, Colorado, 80045, United States
Laura McLaughlin, MD, Principal Investigator

Georgia

Children's Healthcare of Atlanta, Atlanta, Georgia, 30329, United States
Maria Frazer, Contact, (404) 785-6162, [email protected]
Elizabeth Stenger, MD, Principal Investigator

Louisiana

Manning Family Children's, New Orleans, Louisiana, 70118, United States
Wyvonnia Walker, Contact, 504-988-9378, [email protected]
Maria Pereda Ginocchio, MD, Principal Investigator

Pennsylvania

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States
Timothy Olson, MD, PhD, Principal Investigator