Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease (NCT07252050)
🩺 Plain-English Summary (8th-Grade Level)
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 join: To join this study, participants generally need to meet the listed inclusion criteria (8 items) and avoid the listed exclusions (15 items).
Location: Children's Hospital of Colorado — Aurora, Colorado
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- 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.
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (4 Sites)
- Children's Hospital of Colorado — Aurora, Colorado
- Children's Healthcare of Atlanta — Atlanta, Georgia
- Manning Family Children's — New Orleans, Louisiana
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.