Ignite Creation Date:
2024-05-05 @ 4:53 PM
Last Modification Date:
2024-10-26 @ 9:25 AM
Study NCT ID:
NCT00336362
Status:
COMPLETED
Last Update Posted:
2012-12-20
First Post:
2006-06-12
Brief Title:
Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major
Sponsor:
University of Washington
Organization:
University of Washington
Study Overview
Official Title:
A Pilot Study to Assess the Safety and Efficacy of G-CSF Mobilization With and Without Hydroxyurea Pretreatment in Adults With Beta Thalassemia Major
Status:
COMPLETED
Status Verified Date:
2012-12
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:
Beta thalassemia major is a serious genetic disease of the blood Treatments are limited and although a bone marrow transplant from a compatible donor can be curative only a limited percentage of individuals with this disease have a matched donor available A long-term goal of study researchers is to develop a gene transfer process as a method of curing beta thalassemia major Gene transfer involves obtaining blood stem cells from an individual adding a normal globin gene to the stem cells and putting the cells back into the individual
Before gene transfer methods can be attempted in individuals with beta thalassemia major a safe method of obtaining blood stem cells needs to be developed The purpose of this study is to investigate the safety and feasibility of collecting peripheral blood stem cells PBSC from individuals with beta thalassemia major Research participants will be given G-CSF filgrastim for several days to increase the number of stem cells in the blood a process called mobilization After mobilization participants will undergo a procedure called apheresis to remove the white blood cells Researchers in the laboratory will purify the stem cells from the mixture and test methods of putting a normal globin gene into the stem cells Half of the participants will receive hydroxyurea HU prior to G-CSF mobilization HU is used in splenectomized patients to attempt to reduce the risk of clotting during mobilization In non-splenectomized patients HU is given in an attempt to decrease the size of the spleen
Before gene transfer methods can be attempted in individuals with beta thalassemia major a safe method of obtaining blood stem cells needs to be developed The purpose of this study is to investigate the safety and feasibility of collecting peripheral blood stem cells PBSC from individuals with beta thalassemia major Research participants will be given G-CSF filgrastim for several days to increase the number of stem cells in the blood a process called mobilization After mobilization participants will undergo a procedure called apheresis to remove the white blood cells Researchers in the laboratory will purify the stem cells from the mixture and test methods of putting a normal globin gene into the stem cells Half of the participants will receive hydroxyurea HU prior to G-CSF mobilization HU is used in splenectomized patients to attempt to reduce the risk of clotting during mobilization In non-splenectomized patients HU is given in an attempt to decrease the size of the spleen
Detailed Description:
Primary Objective To determine the safety of PBSC mobilization with G-CSF with or without HU pretreatment in adults with beta thalassemia major
Secondary Objective To determine the number of CD34 stemprogenitor cells that are mobilized under these conditions as well as the ability of these cells to be transduced with a recombinant lentivirus vector for beta-globin and engraft immunodeficient mice
Study Design The ability of G-CSF to safely and effectively mobilize PBSC in adults with beta thalassemia major will be assessed in 12 splenectomized and 12 non-splenectomized patients Of the 12 splenectomized patients 6 will be treated with HU and G-CSF and 6 will be treated only with G-CSF Likewise of the 12 non-splenectomized patients 6 will be treated with HU and G-CSF and 6 will be treated only with G-CSF G-CSF mobilized participants will undergo leukapheresis on 2 consecutive days with a target yield of 2 million CD34 cells per kg of body weight Safety will be assessed by monitoring for study-related toxicity Efficacy will be assessed by measuring the total number of CD34 cells the ability of these cells to be transduced with a recombinant lentivirus vector for beta-globin and the ability of these cells to engraft immunodeficient mice
Population Adults with beta thalassemia major
Sample size A total of 24 subjects will be enrolled 12 splenectomized participants and 12 non-splenectomized participants
End Points This is a pilot study and no specific hypotheses are being tested However the study will allow for qualitative comparisons if outcomes between the various arms are markedly different For example the study will provide qualitative data on the safety and feasibility of utilizing HU and G-CSF to mobilize stem cells in individuals with beta thalassemia major The study will be completed upon full enrollment or when stopping criteria are met within specific study arms
Secondary Objective To determine the number of CD34 stemprogenitor cells that are mobilized under these conditions as well as the ability of these cells to be transduced with a recombinant lentivirus vector for beta-globin and engraft immunodeficient mice
Study Design The ability of G-CSF to safely and effectively mobilize PBSC in adults with beta thalassemia major will be assessed in 12 splenectomized and 12 non-splenectomized patients Of the 12 splenectomized patients 6 will be treated with HU and G-CSF and 6 will be treated only with G-CSF Likewise of the 12 non-splenectomized patients 6 will be treated with HU and G-CSF and 6 will be treated only with G-CSF G-CSF mobilized participants will undergo leukapheresis on 2 consecutive days with a target yield of 2 million CD34 cells per kg of body weight Safety will be assessed by monitoring for study-related toxicity Efficacy will be assessed by measuring the total number of CD34 cells the ability of these cells to be transduced with a recombinant lentivirus vector for beta-globin and the ability of these cells to engraft immunodeficient mice
Population Adults with beta thalassemia major
Sample size A total of 24 subjects will be enrolled 12 splenectomized participants and 12 non-splenectomized participants
End Points This is a pilot study and no specific hypotheses are being tested However the study will allow for qualitative comparisons if outcomes between the various arms are markedly different For example the study will provide qualitative data on the safety and feasibility of utilizing HU and G-CSF to mobilize stem cells in individuals with beta thalassemia major The study will be completed upon full enrollment or when stopping criteria are met within specific study arms
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| EudraCT no 2005-000315-10 | US NIH GrantContract | None | httpsreporternihgovquickSearch2U01HL066947 |
| 2U01HL066947 | NIH | None | None |
| IRB no 27527 | None | None | None |