Stem Cell Transplantation for Sickle Cell Anemia
Last updated on July 2021Recruitment
- Recruitment Status
- Active, not recruiting
- Estimated Enrollment
- 40
Summary
- Conditions
- Sickle Cell Disease
- Type
- Interventional
- Phase
- Phase 3
- Design
- Allocation: N/AIntervention Model: Single Group AssignmentMasking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Between 2 years and 30 years
- Gender
- Both males and females
Description
Primary objective: 1) To determine disease free survival (DFS) at one year after matched sibling transplant using either bone marrow (BM), peripheral blood stem cells (PBSC), or umbilical cord blood (UCB) after a conditioning regimen consisting of Alemtuzumab, Fludarabine, and Melphalan in patients ...
Primary objective: 1) To determine disease free survival (DFS) at one year after matched sibling transplant using either bone marrow (BM), peripheral blood stem cells (PBSC), or umbilical cord blood (UCB) after a conditioning regimen consisting of Alemtuzumab, Fludarabine, and Melphalan in patients 2-30 y/o Secondary objectives: Overall survival Rate of neutrophil and platelet engraftment for BM vs. UCB Incidence of graft failure Incidence of grade II-IV and grade III-IV acute graft vs host disease (GVHD) Incidence of chronic GVHD Incidence of other transplant complications, such as veno-occlusive disease, central nervous system (CNS) toxicity, and idiopathic pneumonia syndrome (IPS) Incidence of reactivation of CMV, EBV, adenovirus, BK/JC virus Incidence of invasive fungal disease Time to immune reconstitution via monitoring of lymphocyte subpopulations and immunoglobulin levels
Tracking Information
- NCT #
- NCT01877837
- Collaborators
- Not Provided
- Investigators
- Principal Investigator: Jennifer Krajewski, MD Hackensack Meridian Health
Get Well Soon