Ignite Creation Date:
2025-12-26 @ 12:18 PM
Ignite Modification Date:
2026-03-01 @ 11:28 AM
Study NCT ID:
NCT02867800
Status:
COMPLETED
Last Update Posted:
2024-06-13
First Post:
2016-08-11
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease
Sponsor:
Monica Bhatia
Organization:
Study Overview
Official Title:
Abatacept for Graft Versus Host Disease Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease: a Sickle Transplant Alliance for Research Trial
Status:
COMPLETED
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
To assess the tolerability of the costimulation blocking agent abatacept (CTLA4-Ig) when added to the standard graft versus host disease (GVHD) prophylaxis regimen of a calcineurin inhibitor and methotrexate in patients receiving early alemtuzumab followed by fludarabine, thiotepa, melphalan, and alemtuzumab for conditioning.
Detailed Description:
Outcomes of hematopoietic stem cell transplantation (HSCT) for children and adolescents with sickle cell disease (SCD) have improved. Graft versus host disease (GVHD), however, remains a barrier to success. GVHD accounts for most of the transplant related mortality and much of the morbidity in this setting-in part through the injury it directly causes and in part through the deleterious effects of steroids and the other immunosuppressive agents used to prevent and treat it. The results of pre-clinical studies and a phase I clinical study in patients with hematologic malignancies suggest that the costimulation blocking agent CTLA4-Ig may hold promise an agent for GVHD prophylaxis. In the present trial, the investigators are assessing the tolerability of adding abatacept to standard GVHD prophylaxis-a calcineurin inhibitor and methotrexate-in pediatric SCD patients receiving early alemtuzumab (completed by day -18) followed by fludarabine, thiotepa, and melphalan for conditioning. This trial will provide the foundation for subsequent trials designed to test the long-term hypothesis that abatacept is a safe, steroid-sparing effective adjunct to standard GVHD prophylaxis in this setting.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: