Stem Cell Transplantation for Sickle Cell Anemia

Sickle Cell Disease Clinical Trial

Official title: Reduced Intensity Matched Sibling Bone Marrow Transplantation for Sickle Cell Anemia in Patients 2-30 Years Old

Status Completed

Clinical Trial Summary

This protocol will be investigating the use of stem cell transplantation, in related donors, to cure sickle cell disease. Sickle cell disease is a recessive disorder caused by a point mutation that results in the substitution of valine for glutamic acid at the sixth position in the B-chain of hemoglobin. This leads to sickling of the red blood cells under many conditions, such as hypoxia, dehydration, and hyperthermia. The sickling leads to vaso-occlusion, which causes irreversible damage in almost all systems in the body, including the central nervous system (CNS), lungs, heart, bones, eyes, liver, and kidneys.

Clinical Trial Description

Primary objective:

1) To determine disease free survival (DFS) at two years after matched sibling transplant using bone marrow (BM) after a conditioning regimen consisting of distal timed Alemtuzumab, Fludarabine, and Melphalan for patients 2-30 y/o

Secondary objectives:

1. Overall survival

2. Rate of neutrophil and platelet engraftment for BM

3. Incidence of graft failure

4. Incidence of grade II-IV and grade III-IV acute graft vs host disease (GVHD)

5. Incidence of chronic GVHD

6. Incidence of other transplant complications, such as veno-occlusive disease, central nervous system (CNS) toxicity, and idiopathic pneumonia syndrome (IPS)

7. Incidence of reactivation of CMV, EBV, adenovirus, BK/JC virus

8. Incidence of invasive fungal disease

9. Time to immune reconstitution via monitoring of lymphocyte subpopulations and immunoglobulin levels ;

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

• NCT number: NCT01877837
• Study type: Interventional
• Source: Hackensack Meridian Health
• Status: Completed
• Phase: Phase 3
• Start date: June 2011
• Completion date: September 2021