Ignite Creation Date:
2024-05-06 @ 12:42 AM
Last Modification Date:
2024-10-26 @ 10:53 AM
Study NCT ID:
NCT01639690
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-07-12
First Post:
2012-07-11
Brief Title:
ß-Thalassemia Major with Autologous CD34 Hematopoietic Progenitor Cells Transduced with TNS9355 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene
Sponsor:
Memorial Sloan Kettering Cancer Center
Organization:
Memorial Sloan Kettering Cancer Center
Study Overview
Official Title:
A Phase I Clinical Trial for the Treatment of ß-Thalassemia Major with Autologous CD34 Hematopoietic Progenitor Cells Transduced with TNS9355 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-09
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 patient has inherited ß-thalassemia major through the genes These genes have mistakes in them so the body cannot make normal red blood cells Stem cells are made in the bone marrow They are the earliest form of blood cells
This study is being done to see if the investigators can make the stem cells produce normal red blood cells and hemoglobin The investigators do this by collecting the stem cells The genes with mistakes are removed from the cells These cells are then treated so they have the corrected gene for making normal hemoglobin These treated cells are given back to the patient through an injection shot in the vein This is also known as gene transfer In order for the body to accept these cells the patient will need to receive a low dose of a drug called busulfan It is a drug that will prepare the body to receive the new stem cells
This study will let the investigators know
If it is safe to give the patient the treated stem cells
If the treated stem cells will go into the bone marrow without causing side effects
Gene transfer has been used for the past five years It has been successful in treating many blood disorders At least 20 patients have received the type of treatment that the patient will get on this study This treatment for B-thalassemia major was developed at Memorial Sloan Kettering MSK It was studied for a long time in the lab before being given to patients
This study is being done to see if the investigators can make the stem cells produce normal red blood cells and hemoglobin The investigators do this by collecting the stem cells The genes with mistakes are removed from the cells These cells are then treated so they have the corrected gene for making normal hemoglobin These treated cells are given back to the patient through an injection shot in the vein This is also known as gene transfer In order for the body to accept these cells the patient will need to receive a low dose of a drug called busulfan It is a drug that will prepare the body to receive the new stem cells
This study will let the investigators know
If it is safe to give the patient the treated stem cells
If the treated stem cells will go into the bone marrow without causing side effects
Gene transfer has been used for the past five years It has been successful in treating many blood disorders At least 20 patients have received the type of treatment that the patient will get on this study This treatment for B-thalassemia major was developed at Memorial Sloan Kettering MSK It was studied for a long time in the lab before being given to patients
Detailed Description:
None
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