Ignite Creation Date:
2024-05-06 @ 1:35 PM
Last Modification Date:
2024-10-26 @ 1:16 PM
Study NCT ID:
NCT04069533
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-22
First Post:
2019-08-23
Brief Title:
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
Sponsor:
Rocket Pharmaceuticals Inc
Organization:
Rocket Pharmaceuticals Inc
Study Overview
Official Title:
A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34 Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene Orphan Drug in Patients With Fanconi Anemia Subtype A
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-05
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 an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric 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 pediatric 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?:
False
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2018-002502-31 | EUDRACT_NUMBER | None | None |