Ignite Creation Date:
2024-05-05 @ 4:37 PM
Last Modification Date:
2024-10-26 @ 9:22 AM
Study NCT ID:
NCT00272857
Status:
COMPLETED
Last Update Posted:
2017-06-23
First Post:
2006-01-04
Brief Title:
Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia
Sponsor:
Boston Childrens Hospital
Organization:
Boston Childrens Hospital
Study Overview
Official Title:
Gene Transfer From Patients With Fanconi Anemia Genotype A A Pilot Study
Status:
COMPLETED
Status Verified Date:
2017-06
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:
Fanconi anemia FA is a disease that affects an individuals bone marrow It is caused by a defective gene in the bone marrow cells that produce various types of blood cells Individuals with FA may experience fatigue bleeding and increased infections The purpose of this study is to evaluate the safety and effectiveness of a gene transfer procedure in generating new healthy cells in individuals with FA
Detailed Description:
FA is a rare inherited disease that is caused by a gene defect and that 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 Bone marrow transplants are one common treatment for FA However there are many risks associated with transplantation including rejection of the transplanted cells and graft-versus-host disease a serious side effect in which donor cells attack the recipients tissues This study will use an experimental gene transfer procedure performed in a laboratory to insert a new FA gene into the participants bone marrow cells The gene-corrected bone marrow cells will then be re-infused into the participant and participants will be observed for successful gene transfer The purpose of this study is to evaluate the safety and effectiveness of the FA gene transfer procedure and to determine the ability of the gene-corrected cells at generating new healthy blood cells in individuals with FA
This study will enroll individuals with FA Participants will be required to have the initial bone marrow transfer procedure performed at Cincinnati Childrens Hospital but will be allowed to see their own doctor for the majority of study visits Participants will first attend a screening visit which will include a physical exam blood draw for laboratory testing and a bone marrow biopsy Bone marrow cells will be collected from eligible participants and sent to a laboratory for the FA gene transfer procedure Several days later the gene-corrected cells will be re-infused back into the participants via an intravenous catheter Side effects will be closely monitored for 12 hours following the procedure and participants may be required to spend an overnight in the hospital Following discharge from the hospital participants will be required to stay in the Cincinnati area for 3 days to undergo daily evaluations and physical examinations Participants will continue to be followed very closely for the first year after cell re-infusion Study visits will be held weekly for the first 3 weeks and then every 3 months for the remainder of the year Visits will include physical examinations blood collection and liver function testing Bone marrow testing will occur at Months 3 6 and 12 Follow-up visits will occur yearly for up to 15 years and will include blood collection a physical exam and review of medical history
This study will enroll individuals with FA Participants will be required to have the initial bone marrow transfer procedure performed at Cincinnati Childrens Hospital but will be allowed to see their own doctor for the majority of study visits Participants will first attend a screening visit which will include a physical exam blood draw for laboratory testing and a bone marrow biopsy Bone marrow cells will be collected from eligible participants and sent to a laboratory for the FA gene transfer procedure Several days later the gene-corrected cells will be re-infused back into the participants via an intravenous catheter Side effects will be closely monitored for 12 hours following the procedure and participants may be required to spend an overnight in the hospital Following discharge from the hospital participants will be required to stay in the Cincinnati area for 3 days to undergo daily evaluations and physical examinations Participants will continue to be followed very closely for the first year after cell re-infusion Study visits will be held weekly for the first 3 weeks and then every 3 months for the remainder of the year Visits will include physical examinations blood collection and liver function testing Bone marrow testing will occur at Months 3 6 and 12 Follow-up visits will occur yearly for up to 15 years and will include blood collection a physical exam and review of medical history
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 |
|---|---|---|---|
| R01HL081499-01A1 | NIH | CCHMC | httpsreporternihgovquickSearchR01HL081499-01A1 |
| R01HL081499 | NIH | None | None |
| CCHMCEH002 | OTHER_GRANT | None | None |