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Clinical Trial 19203
Interventions:Cellcept (Mycophenolate Mofetil); Hydroxyurea (Droxia); MESNA; Mycophenolate Mofetil; Radiotherapy; Rapamune (Sirolimus); Rapamycin (Sirolimus); Sirolimus; Thiotepa (Thioplex); Thymoglobulin; cyclophosphamide; cytoxan (cyclophosphamide); fludarabine (Fludarabine phosphate)
Study Type: Treatment
Phase of Study: Phase II
- Michael Nieder
Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease
The purpose of this study is to learn if a haploidentical transplant is safe and effective to treat Sickle Cell Disease (SCD). A haploidentical transplant uses a half or partially-matched donor. ("Haplo" means half.) This study will use a parent or other family member who is a half tissue match. This site will not enroll children.
The primary objective is to estimate event-free survival (EFS) at 2 years after a reduced intensity conditioning regimen and human leukocyte antigen (HLA)-haploidentical bone marrow transplantation (haploBMT) in children with SCD and adults with severe SCD. Secondary Objectives: Within each stratum: 1. Estimate overall survival at one year and two years after enrollment and after haploBMT. 2. Estimate EFS at one year after haploBMT. 3. Estimate incidence of primary and secondary graft rejection at one and two years after haploBMT. 4. Estimate incidence and severity of acute GVHD until Day 100 then chronic GVHD at six months, one year, 18 months, and two years post-transplant. 5. Characterize donor hematopoietic chimerism in peripheral blood at days 28, 100, and 180 and at 1 and 2 years after haploBMT. 6. Characterize hematologic and non-hematologic toxicities of haploBMT, including the incidence and severity of acute and chronic graft-versus-host disease; time to and probability of red blood cell, neutrophil, and platelet recovery; hepatic veno-occlusive disease (VOD); idiopathic pneumonia syndrome (IPS); central nervous system (CNS) toxicity (reversible posterior leukoencephalopathy syndrome [RPLS], hemorrhage, and seizures); cytomegalovirus (CMV) infection; adenovirus infection; Epstein Barr virus post-transplant lymphoproliferative disease (EBV PTLD); invasive fungal infection. 7. Evaluate if sickle vasculopathy is halted by successful transplantation as determined by comparing brain MRI pre- and 2 years post-haploBMT. Cerebral MRI/MRA is required for all pediatric patients to assess progression of CNS disease. For adults with where the indication for the transplant is a stroke, cerebral MRI/MRA is required after transplant to assess progression of CNS disease. 8. Evaluate sickle-related events and end organ function in all recipients after haploBMT to determine if severe and debilitating vaso-occlusive pain and cerebral infarction are stabilized after transplantation. 9. Evaluate patient-reported quality of life (pain and fatigue domains) pre- and 1 and 2 years post-haploBMT in the adult stratum. 10. Lung function pre- and 2 years post-haploBMT. 11. TRJV pre- and 1 and 2 years post-haploBMT. 12. 6 min walk distance pre- and 1 and 2 years post-haploBMT. 13. Pain intensity assessed by an electronic pain diary at baseline, 1 and 2 years post-haploBMT for patient >/= 13.00 years of age at time of enrollment. 14. Hematological outcomes at 2 years (hgb, retic, %HbS, LDH, bili, last date of red blood cell transfusion). 15. Viral mold infections/bacterial or fungal sepsis at anytime up to 2 years post transplant. 16. Proportion on immunosuppression at 2 years post-haploBMT.
Adequate physical function as measured by all of the following: