Clinical Trial
Study Details
Haploidentical 5+/10 HSCT from a relative, α/β T-depleted
10/10 HSCT - matched family donor
HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory. With the lack of a suitable donor for the vast majority of patients, the major question of this trial is, if a haploidentical αß/CD19+ T-cell depleted HSCT can be a valid alternative to a MSD HSCT. The main challenge in non-malignant diseases is to offer a safe and GvHD-free HSCT without rejection.
Study Overview
Can an α/ß depleted T-Haplo-HSCT with regard to disease free survival, adverse events and safety be considered equivalent to a matched sibling donor transplantation (MSD), in order to offer cure for the majority of patients with sickle cell disease.
The main questions of this trial are:
* Safety of a α/ß T-depleted haploidentical HSCT
* Incidence of acute and chronic GvHD
* Rate of rejection
* Immune reconstitution
* Fertility It is expected that the use of TCRαβ+ and CD19+ depleted haploidentical cell grafts in combination with the less aggressive and well tolerated conditioning regimen needed for patient preparation will be associated with a low risk of grade II-IV aGVHD and no extensive cGvHD, no graft failure and increase speed, spectrum and functionality of immune system reconstitution. This is supposed to reduce the incidence of severe infections leading to lower rates of transplantation related mortality (TRM).
Contacts and Locations
St. Anna Kinderspital
Vienna
Austria
University Hospital Aachen, Children's Hospital
Aachen
Germany
Charité University medicine, Clinic for Hematology, Oncology
Berlin
Germany
University Hospital Duesseldorf, Clinic for Pediatric Oncology, - Hemtaology and Clinical Immunology
40225 Düsseldorf
Germany
University Hospital of Frankfurt, Clinic for Paediatrics and Adolescent Medicine
Frankfurt
Germany
University Hospital Heidelberg, Department of Pediatric Hematology, Oncology and Immunology
69120 Heidelberg
Germany
University Hospital Regensburg, Dept. of Ped. Hematology, Oncology and Stem Cell Transplantation
93053 Regensburg
Germany
University Children's Hospital Tübingen
72076 Tübingen
Germany
University Children's Hospital Würzburg
97080 Würzburg Würzburg
Germany