Ignite Creation Date:
2024-05-06 @ 12:39 PM
Last Modification Date:
2024-10-26 @ 1:02 PM
Study NCT ID:
NCT03812263
Status:
COMPLETED
Last Update Posted:
2023-11-15
First Post:
2019-01-18
Brief Title:
A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
Sponsor:
Rocket Pharmaceuticals Inc
Organization:
Rocket Pharmaceuticals Inc
Study Overview
Official Title:
Gene Therapy for Leukocyte Adhesion Deficiency-I LAD-I A Phase III Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene
Status:
COMPLETED
Status Verified Date:
2023-11
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 primary purpose of the Phase I portion of the study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34 enriched cells transduced with the therapeutic lentiviral vector Chim-CD18-WPRE RP-L201 The primary objectives of the Phase II portion of the study are evaluation of survival as determined by the proportion of subjects alive at age 2 24 months and at least 1-year post-infusion without allogeneic hematopoietic stem cell transplant HSCT and characterization of the safety and toxicity associated with the infusion
Detailed Description:
This is a pediatric non-randomized open-label Phase III clinical trial The Phase I portion will include a safety evaluation and preliminary assessment of the efficacy of hematopoietic gene therapy consisting of autologous CD34 enriched cells transduced with a lentiviral vector carrying the ITGB2 gene in subjects with severe LAD-I CD34 cells will be transduced ex vivo with the therapeutic vector followed by cryopreservation If the number of CD34 cells that are available for infusion is at least 2x10e6 total CD34 cellskg subjects will undergo myeloablative conditioning with intravenous busulfan Subjects will then receive infusion of gene-corrected hematopoietic cells approximately 24 hours following the final busulfan dose
The active agent is a self-inactivating lentiviral vector carrying the therapeutic ITGB2 gene encoding for the human CD18 receptor β2 integrin subunit The therapeutic product is the subjects autologous hematopoietic stem cells that have been transduced with the lentiviral vector
The active agent is a self-inactivating lentiviral vector carrying the therapeutic ITGB2 gene encoding for the human CD18 receptor β2 integrin subunit The therapeutic product is the subjects autologous hematopoietic stem cells that have been transduced with the lentiviral vector
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