Haploidentical Hematopoietic Stem Cell Transplantation

Phase 1 Completed 7 enrolled Catherine Bollard Children's National Research Institute · collab

NaSingle-groupOpen-labelTreatment

Summary

The study is designed as a Pilot/Phase 1 trial of reduced intensity Haploidentical HSCT in patients with sickle cell disease and thalassemia. The purpose of the study is to assess the safety and toxicity of reduced intensity conditioning haploidentical hematopoietic stem cell transplantation.

Timeline

Start: 2015-01
Primary completion: 2024-01
Completion: 2024-10

Publications

Leonard A, Furstenau D, Abraham A, Darbari DS, Nickel RS, Limerick E, Fitzhugh C, Hsieh M, Tisdale JF. Reduction in vaso-occlusive events following stem cell transplantation in patients with sickle cell disease. Blood Adv. 2023 Jan 24;7(2):227-234. doi: 10.1182/bloodadvances.2022008137.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	peripheral blood stem cell graft that are CD34+ selected	Unknown	—	—
Background	Alemtuzumab	Monoclonal antibody	—	—
Background	Fludarabine	Small molecule	—	—
Background	Hydroxyurea	Other / unclassified	—	—
Background	Melphalan	Small molecule	—	—
Background	Sirolimus	Small molecule	—	—
Background	Thiotepa	Other / unclassified	—	—
Background	Thymoglobulin	Protein / enzyme biologic	—	—

Haploidentical Hematopoietic Stem Cell Transplantation

Summary

Timeline

Publications

Drugs

Indications