Ignite Creation Date:
2024-05-05 @ 7:20 PM
Last Modification Date:
2024-10-26 @ 9:47 AM
Study NCT ID:
NCT00658385
Status:
COMPLETED
Last Update Posted:
2016-11-25
First Post:
2008-04-10
Brief Title:
Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor GCSF Mobilization of CD34 Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
Sponsor:
Memorial Sloan Kettering Cancer Center
Organization:
Memorial Sloan Kettering Cancer Center
Study Overview
Official Title:
A Pilot Trial to Assess the Feasibility and Safety of GCSF Mobilization of CD34 Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
Status:
COMPLETED
Status Verified Date:
2016-10
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:
Betathalassemia major is a disease of the blood and bone marrow You were born with it and it has made you unable to make normal hemoglobin and red cells You have been receiving red blood cell transfusions all your life These transfusions do not cure your disease The problem with transfusions is that they contain a lot of iron With time iron builds up in your body and will eventually hurt some of your organs Because of this buildup of iron you are taking medicine that helps your body get rid of the extra iron
Today the only other treatment is bone marrow or stem cell transplant It can only be done when a matched donor is available This is most often a brother sister or parent Bone marrow transplant may cure betathalassemia major If you have a transplant and it is successful you will no longer have the disease Without a matched sibling or parent the standard treatment is to keep having transfusions
In the near future we will be testing a new treatment for making normal hemoglobin and normal red blood cells We have recreated the healthy hemoglobin gene in a test tube We are able to use it and put it back into cells This is called gene therapy We have been able to put this gene into the stem cells of mice with thalassemia These mice were cured We now plan to take that gene and put it into stem cells from people who have betathalassemia major We will then inject those stem cells back into that persons blood
In general we can obtain more stem cells from the blood of a person than from the bone marrow In order to do so we must give that person a blood growth factor The growth factor stimulates the bone marrow to make more stem cells That growth factor is called granulocyte colony stimulating factor GCSF or Filgrastim
The purpose of this trial is to find out if the drug GCSF has any side effects on you and if you will make more stem cells in response to it This trial is not a gene therapy trial This trial will not help your thalassemia
Today the only other treatment is bone marrow or stem cell transplant It can only be done when a matched donor is available This is most often a brother sister or parent Bone marrow transplant may cure betathalassemia major If you have a transplant and it is successful you will no longer have the disease Without a matched sibling or parent the standard treatment is to keep having transfusions
In the near future we will be testing a new treatment for making normal hemoglobin and normal red blood cells We have recreated the healthy hemoglobin gene in a test tube We are able to use it and put it back into cells This is called gene therapy We have been able to put this gene into the stem cells of mice with thalassemia These mice were cured We now plan to take that gene and put it into stem cells from people who have betathalassemia major We will then inject those stem cells back into that persons blood
In general we can obtain more stem cells from the blood of a person than from the bone marrow In order to do so we must give that person a blood growth factor The growth factor stimulates the bone marrow to make more stem cells That growth factor is called granulocyte colony stimulating factor GCSF or Filgrastim
The purpose of this trial is to find out if the drug GCSF has any side effects on you and if you will make more stem cells in response to it This trial is not a gene therapy trial This trial will not help your thalassemia
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