NCT07252050 · Arkansas Children's Hospital Research Institute
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
(RUX-HAPLO)
What this study is about
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.
View original scientific description
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Participants with any genotypic form of SCD aged 12 - 45 years at enrollment with ≥1 of the following:
- History of stroke and/or vasculopathy, including evidence of asymptomatic cerebrovascular disease for pediatric patients.
- Recurrent moderate-severe acute chest syndrome (ACS)
- Recurrent vaso-occlusive pain episodes requiring parenteral analgesia despite the institution of supportive care.
- Need for chronic transfusion therapy to prevent vaso-occlusive complications (i.e. pain, stroke, and ACS).
- For adult patients, an echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) ≥ 2.7 m/sec.
- Participants must have an HLA haploidentical first degree relative (parent, sibling, or half sibling) who is willing and able to donate bone marrow.
- Participants must meet institutional eligibility criteria for HCT.
Exclusion criteria
- Presence of an HLA-matched sibling who is willing and able to donate bone marrow.
- Uncontrolled infection, evidence of active TB, Hepatitis B or C infection, or HIV seropositivity or infection.
- Previous HCT or solid organ transplant.
- CNS revascularization procedure, myocardial infarction, pulmonary embolus or deep vein thrombosis in the past 6 months.
- Use of medications which significantly interfere with ruxolitinib metabolism.
- Known hypersensitivity or severe reaction to ruxolitinib or any component of the conditioning regimen or its excipients.
- Inability to swallow and retain oral medication (use of nasogastric or gastrostomy tube permitted).
- History of malignancy except resected basal cell carcinoma or treated carcinoma in-situ.
- Participation in another clinical trial involving an investigational or off-label use of a drug or device in the past 3 months.
- Currently pregnant or breast feeding.
- Clinically significant, uncontrolled autoimmune disease.
- High-titer anti-donor specific HLA antibodies (without review and approval by Study Chair).
- 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).
- 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.
Where
- Aurora, Colorado
- Atlanta, Georgia
- New Orleans, Louisiana
- Philadelphia, Pennsylvania
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 4, 2026 · Source of record for eligibility and locations