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Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease — SUN  

Sickle Cell Disease Clinical Trial

Official title:
Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease

Clinical Trial Summary

This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).

Clinical Trial Description

This is a prospective, multicenter phase II study of HLA-identical sibling donor HSCT in 30 pediatric patients with SCD using nonmyeloablative conditioning with alemtuzumab, total-body irradiation, and sirolimus. The primary Objective of this study is to determine if the SUN regimen can decrease the incidence of grade II-IV acute graft-versus host disease (GVHD) by day +100 while maintaining similar disease-free survival compared to establish HLA-identical donor hematopoietic stem cell transplant (HSCT) regimens in children with SCD. The secondary Objective is to determine if health-related quality of life (HRQoL) for children undergoing SUN HSCT is preserved during the early post-transplant time period. To determine if the SUN regimen can decrease the number of platelet transfusions compared to established HLA-identical HSCT regimens in children with SCD. The tertiary/Exploratory Objectives: To describe other markers of toxicity (duration of neutropenia, mucositis, length of hospitalization) and indicators of a successful HSCT (HRQoL at 1 year, proportion needing additional immunosuppression during the first year, proportion able to wean sirolimus at 1 year). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03587272
Study type Interventional
Source Children's National Research Institute
Contact Robert Nickel, MD
Phone 202-476-5000
Email RNickel@childrensnational.org
Status Recruiting
Phase Phase 2
Start date April 17, 2018
Completion date November 2025
View Details
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