Ignite Creation Date:
2024-05-05 @ 11:07 AM
Last Modification Date:
2024-10-26 @ 9:05 AM
Study NCT ID:
NCT00005896
Status:
UNKNOWN
Last Update Posted:
2005-06-24
First Post:
2000-06-02
Brief Title:
Phase I Pilot Study of CD34 Enriched Fanconis Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconis Anemia
Sponsor:
University of Minnesota
Organization:
Office of Rare Diseases ORD
Study Overview
Official Title:
None
Status:
UNKNOWN
Status Verified Date:
2001-03
Last Known Status:
ACTIVE_NOT_RECRUITING
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:
OBJECTIVES I Determine the safety of transferring the Fanconi anemia complementation group C FACC gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconis anemia complementation group C
II Determine the extent of engraftment following this treatment regimen without prior ablation of recipient marrow in these patients
III Determine the ability of this treatment regimen to correct the cell phenotype and improve hematopoietic function in these patients
II Determine the extent of engraftment following this treatment regimen without prior ablation of recipient marrow in these patients
III Determine the ability of this treatment regimen to correct the cell phenotype and improve hematopoietic function in these patients
Detailed Description:
PROTOCOL OUTLINE Patients receive filgrastim G-CSF subcutaneously daily on days 0-6 followed by apheresis to collect peripheral blood stem cells PBSC on days 5-7 PBSCs are processed in vitro for enrichment of CD34 cells and transduced with a Fanconis anemia complementation C FACC retroviral vector on days 5-10 Patients receive transduced PBSCs IV over no more than 2 hours on days 8-10 PBSC infusions may be repeated no more than every 2 months for up to 4 courses total
Patients are followed monthly for 3 months every 3 months for 9 months every 6 months for the next year and then yearly thereafter
Patients are followed monthly for 3 months every 3 months for 9 months every 6 months for the next year and then yearly thereafter
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| UNM-MT-9710 | None | None | None |
| UMN-MT-1997-10 | None | None | None |