NCT07252050 Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
| NCT ID | NCT07252050 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Arkansas Children's Hospital Research Institute |
| Condition | Sickle Cell Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 24 participants |
| Start Date | 2026-06-08 |
| Primary Completion | 2028-11-19 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 24 participants in total. It began in 2026-06-08 with a primary completion date of 2028-11-19.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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 additional 2 years post-HCT.
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Contact & Investigator
Laura McLaughlin, MD
PRINCIPAL INVESTIGATOR
Arkansas Children's Hospital Research Institute
Frequently Asked Questions
Who can join the NCT07252050 clinical trial?
This trial is open to participants of all sexes, aged 12 Years or older, up to 45 Years, studying Sickle Cell Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07252050 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07252050 currently recruiting?
Yes, NCT07252050 is actively recruiting participants. Contact the research team at LMcLaughlin@uams.edu for enrollment information.
Where is the NCT07252050 trial being conducted?
This trial is being conducted at Aurora, United States, Atlanta, United States, New Orleans, United States, Philadelphia, United States.
Who is sponsoring the NCT07252050 clinical trial?
NCT07252050 is sponsored by Arkansas Children's Hospital Research Institute. The principal investigator is Laura McLaughlin, MD at Arkansas Children's Hospital Research Institute. The trial plans to enroll 24 participants.
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