Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | Aplastic Anemia & MDS International Foundation
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Clinical Trial: NCT02179359

For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:
Purpose: 

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

Status: 
Recruiting
Study Date: 
Tue, 09/02/2014 to Mon, 08/01/2022
Bone Marrow Disease(s): 
  • paroxysmal nocturnal hemoglobinuria (PNH)
Intervention: 
Experimental: Reduced Toxicity Ablative Regimen For use in patients with a matched sibling donor or unrelated UCB donor and DBA patients who are <12 years and/or have mild/moderate iron exposure. Drug: Reduced Toxicity Ablative Regimen Anti-thymocyte Globulin (ATG) Fludarabine Busulfan Stem Cell Infusion Day 0 Experimental: Reduced Intensity Preparative Regimen For use in patients with unrelated donor bone marrow and for DBA patients who are >12 years and/or have significant iron exposure. Drug: Reduced Intensity Preparative Regimen Alemtuzumab Cyclophosphamide Fludarabine Total Body Irradiation (TBI) Stem Cell Infusion Day 0 Experimental: Myeloablative Preparative Regimen For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia. Drug: Myeloablative Preparative Regimen Alemtuzumab Cyclophosphamide Busulfan Stem Cell Infusion Day 0