Ignite Creation Date:
2024-05-06 @ 2:57 AM
Last Modification Date:
2024-10-26 @ 11:26 AM
Study NCT ID:
NCT02165007
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-09-21
First Post:
2014-06-13
Brief Title:
Haploidentical Hematopoietic Stem Cell Transplantation
Sponsor:
Catherine Bollard
Organization:
Childrens National Research Institute
Study Overview
Official Title:
HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE AND THALASSEMIA USING CD34 POSITIVE SELECTED GRAFTS
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-09
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:
The study is designed as a PilotPhase 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
Detailed Description:
Research subjects will undergo reduced intensity conditioning Hydroxyurea ATG Fludarabine Thiotepa Melphalan followed by infusion of a peripheral blood stem cell graft collected from haploidentical family donors that are CD34 positively selected using the CliniMACS device Sirolimus will be used for GVHD prophylaxis and given for 9 months post-transplant and then tapered off by one year
The use of the CliniMACS device for CD34 selection will be performed at CNMC through cross-reference of the master file for CliniMACS CD34 Reagent by Milteyni Biotech BB-MF 8061
CliniMACs is an electromechanical device intended to isolate certain cell subsets from mixed cell populations When used in combination with the CliniMACs CD34 reagent it is possible to prepare extremely pure populations of CD34 cells with upwards of 5 logs depletion of contaminating T cells within a closed and sterile system
We intend to use this system to select cells from HLA haploidentical related donors who have been mobilized with G-CSF prior to stem cell collection Since previous investigations of this strategy in adult patients have not translated into enhanced long term survival we intend to limit this protocol to patients under the age of 22 as they have more rapid immune reconstitution
The use of the CliniMACS device for CD34 selection will be performed at CNMC through cross-reference of the master file for CliniMACS CD34 Reagent by Milteyni Biotech BB-MF 8061
CliniMACs is an electromechanical device intended to isolate certain cell subsets from mixed cell populations When used in combination with the CliniMACs CD34 reagent it is possible to prepare extremely pure populations of CD34 cells with upwards of 5 logs depletion of contaminating T cells within a closed and sterile system
We intend to use this system to select cells from HLA haploidentical related donors who have been mobilized with G-CSF prior to stem cell collection Since previous investigations of this strategy in adult patients have not translated into enhanced long term survival we intend to limit this protocol to patients under the age of 22 as they have more rapid immune reconstitution
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None