Ignite Creation Date:
2024-05-05 @ 4:36 PM
Last Modification Date:
2024-10-26 @ 9:21 AM
Study NCT ID:
NCT00271089
Status:
COMPLETED
Last Update Posted:
2016-07-12
First Post:
2005-12-29
Brief Title:
Blood Cell Collection for Future Use in Individuals With Fanconi Anemia
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Heart Lung and Blood Institute NHLBI
Study Overview
Official Title:
Collection of Hematopoietic Cells From Patients With Fanconi Anemia FA for Future Autologous Reinfusion and Research
Status:
COMPLETED
Status Verified Date:
2007-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Fanconi anemia FA is a disease that affects an individuals bone marrow It is caused by a defective gene in the CD34 cells which are responsible for producing various types of blood cells Individuals with FA may experience fatigue bleeding and increased infections The purpose of this study is to collect and purify blood cells from individuals with FA and store them for future therapeutic use
Detailed Description:
FA is a rare inherited disease that is caused by a gene defect to the CD34 cells It primarily affects an individuals bone marrow resulting in decreased production of blood cells The lack of white blood cells affects an individuals ability to fight infections the lack of platelets may result in bleeding and the lack of red blood cells usually leads to anemia FA is typically diagnosed in childhood and there is a high fatality rate This study will use two methods to collect purify and store participants CD34 cells for future use in case of severe bone marrow failure The collected cells will also be used by researchers to better understand the causes of FA and to possibly develop new treatments
This study will enroll individuals with FA All participants will undergo a bone marrow biopsy within 3 months of study entry Based on the results of this biopsy participants will undergo either a bone marrow harvest procedure or a cytokine mobilized peripheral blood stem cell PBSC collection procedure Prior to both procedures medical history will be reviewed blood will be drawn liver and kidney function will be evaluated and a physical examination will be performed Participants who undergo the bone marrow harvest procedure will be admitted to the hospital with a possible overnight stay for observation The following day participants will have a physical examination and blood draw for laboratory testing A blood andor platelet transfusion may be required following the procedure
Participants who undergo the PBSC procedure will be required to receive injections of G-CSF a protein found normally in the body twice a day for 4 to 8 days prior to the procedure G-CSF has been found to help increase the amount of CD34 cells in the blood Once the CD34 level is within a certain range the PBSC procedure will begin through an IV placed in the arm or a temporary collection catheter placed under the participants collarbone Blood cells will be collected with some cells separated out and the remainder of the cells infused back into the participant The length of this procedure will vary for each participant it will take 3 to 6 hours a day for 1 to 4 days Participants may require blood andor platelet transfusions prior to and during the procedure
Following the bone marrow harvest and PBSC procedures CD34 cells will be isolated in a laboratory The majority of the cells will be frozen and stored for future use by the participants A small portion of the cells will be available for researchers to perform experimental research to better understand FA
The study completion date listed in this record was obtained from the End Date entered in the Protocol Registration and Results System PRS record
This study will enroll individuals with FA All participants will undergo a bone marrow biopsy within 3 months of study entry Based on the results of this biopsy participants will undergo either a bone marrow harvest procedure or a cytokine mobilized peripheral blood stem cell PBSC collection procedure Prior to both procedures medical history will be reviewed blood will be drawn liver and kidney function will be evaluated and a physical examination will be performed Participants who undergo the bone marrow harvest procedure will be admitted to the hospital with a possible overnight stay for observation The following day participants will have a physical examination and blood draw for laboratory testing A blood andor platelet transfusion may be required following the procedure
Participants who undergo the PBSC procedure will be required to receive injections of G-CSF a protein found normally in the body twice a day for 4 to 8 days prior to the procedure G-CSF has been found to help increase the amount of CD34 cells in the blood Once the CD34 level is within a certain range the PBSC procedure will begin through an IV placed in the arm or a temporary collection catheter placed under the participants collarbone Blood cells will be collected with some cells separated out and the remainder of the cells infused back into the participant The length of this procedure will vary for each participant it will take 3 to 6 hours a day for 1 to 4 days Participants may require blood andor platelet transfusions prior to and during the procedure
Following the bone marrow harvest and PBSC procedures CD34 cells will be isolated in a laboratory The majority of the cells will be frozen and stored for future use by the participants A small portion of the cells will be available for researchers to perform experimental research to better understand FA
The study completion date listed in this record was obtained from the End Date entered in the Protocol Registration and Results System PRS record
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 |
|---|---|---|---|
| CCHMCEH001 | None | None | None |
| R01HL081499-01A1 | NIH | None | httpsreporternihgovquickSearchR01HL081499-01A1 |