Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
About this study
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.
- Condition
- Sickle Cell Disease, Hematopoetic Stem Cell Transplant, Haploidentical Hematopoietic Stem Cell Transplant, Haploidentical Stem Cell Transplantation, Graft Failure
- Tested
- Ruxolitinib
- Sponsor
- Arkansas Children's Hospital Research Institute
Who can join
- Age
- 12 to 45 years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
Inclusion 11
- Minimum age: 12 years
- Maximum age: 45 years
- Study condition: Sickle Cell Disease, Hematopoetic Stem Cell Transplant, Haploidentical Hematopoietic Stem Cell Transplant, Haploidentical Stem Cell Transplantation, Graft Failure
- 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 14
- 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
4 sites, 4 recruiting
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Children's Hospital of Colorado
Aurora, Colorado, United States
Manning Family Children's
New Orleans, Louisiana, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Contact
-
Laura McLaughlin, MD
501-364-3908 LMcLaughlin@uams.edu
-
Kayla Ortiz
720-777-4151 Kayla.Ortiz@childrenscolorado.org
Potential match only. Final eligibility is determined by the study team.