Ignite Creation Date:
2024-05-06 @ 2:11 PM
Last Modification Date:
2024-10-26 @ 1:27 PM
Study NCT ID:
NCT04248439
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-04-10
First Post:
2020-01-24
Brief Title:
Gene Therapy for Fanconi Anemia Complementation Group A
Sponsor:
Rocket Pharmaceuticals Inc
Organization:
Rocket Pharmaceuticals Inc
Study Overview
Official Title:
A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34 Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-04
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:
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia subtype A FA-A
Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo outside the body with a lentiviral vector carrying the FANCA gene After transduction the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure
Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo outside the body with a lentiviral vector carrying the FANCA gene After transduction the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure
Detailed Description:
This is a pediatric open-label Phase II clinical trial to assess the efficacy of a hematopoietic gene therapy consisting of autologous CD34 enriched cells transduced with a lentiviral vector carrying the FANCA gene in subjects with FA-A
Enriched CD34 hematopoietic stem cells will be transduced ex vivo with the therapeutic lentiviral vector and infused via intravenous infusion following transduction without any prior conditioning
Enriched CD34 hematopoietic stem cells will be transduced ex vivo with the therapeutic lentiviral vector and infused via intravenous infusion following transduction without any prior conditioning
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