Ignite Creation Date:
2024-05-06 @ 2:45 PM
Last Modification Date:
2024-10-26 @ 1:37 PM
Study NCT ID:
NCT04418414
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-02-20
First Post:
2020-05-28
Brief Title:
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
Sponsor:
Expression Therapeutics LLC
Organization:
Expression Therapeutics LLC
Study Overview
Official Title:
ET3-201 Phase 1 Study of Hematopoietic Stem Cell Transplantation HSCT Gene Therapy Incorporating a Lentiviral Vector LV Encoding a High Expressing Factor VIII Transgene for Treatment of Severe Hemophilia A
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-02
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:
This is a first-in-human non-randomized open label single treatment Phase 1 study in approximately 7 patients with severe hemophilia A The study will evaluate gene therapy by transplantation of autologous CD34 hematopoietic stem cells transduced ex vivo with the CD68-ET3 lentiviral vector
Detailed Description:
Eligible subjects will undergo CD34 hematopoietic stem cell collection These cells will be transduced ex vivo with CD68-ET3 lentiviral vector and subsequently following a conditioning regimen of busulfan and anti-thymocyte globulin the transduced cells will be infused to patients After completion of study treatment patients are followed up periodically for up to 15 years
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None