Ignite Creation Date:
2024-05-06 @ 5:07 PM
Last Modification Date:
2024-10-26 @ 2:23 PM
Study NCT ID:
NCT05207657
Status:
RECRUITING
Last Update Posted:
2023-05-16
First Post:
2021-06-24
Brief Title:
Lentiviral Gene Therapy for p47 AR-CGD
Sponsor:
Great Ormond Street Hospital for Children NHS Foundation Trust
Organization:
Great Ormond Street Hospital for Children NHS Foundation Trust
Study Overview
Official Title:
Phase III Non-randomised Single-centre Open-label Study of pCHIM-p47 Lentiviral Vector Transduced CD34 Cells in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease
Status:
RECRUITING
Status Verified Date:
2023-03
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:
Chronic Granulomatous Disease CGD is a rare inherited disorder in which patients suffer from severe infection and inflammation The first indications of disease usually appear in early childhood The basic defect has been found to be lie in specialised white blood cells called phagocytic cells which are responsible for engulfing and destroying germs In CGD there is a defect in an enzyme known as the NADPH-oxidase that is responsible for generating bleach like substances that are important for killing some important germs In one form of the disease known as p47 AR-CGD which accounts for 30 of patients there are defined mistakes in a gene called NCF1 This gene is needed to form a key component of NADPH-oxidase
In many cases patients can be protected from infection by constant intake of antibiotics However in others potentially life-threatening infections break through In some cases patients also develop serious inflammation requiring high doses of drugs such as steroids CGD can be cured by bone marrow transplant and the best results are available when a matched sibling donor is available Transplant from unmatched donors have a much worse outcome and as a result alternative treatments for patients without a matched donor are highly desirable
Gene therapy of p47 AR-CGD is performed by introducing a normal copy of the human NCF-1 gene into the blood forming stem cells in the patients bone marrow by using a gene carrier in this study called a lentiviral vector After treatment of the bone marrow cells in a specialised laboratory they are given back to the patient and will grow into functional phagocytic cells There have been no previous clinical trials for patients with p47 AR-CGD however there have been previous gene therapy clinical trials conducted in the UK for patients with the most common form of CGD known as X-CGD
In many cases patients can be protected from infection by constant intake of antibiotics However in others potentially life-threatening infections break through In some cases patients also develop serious inflammation requiring high doses of drugs such as steroids CGD can be cured by bone marrow transplant and the best results are available when a matched sibling donor is available Transplant from unmatched donors have a much worse outcome and as a result alternative treatments for patients without a matched donor are highly desirable
Gene therapy of p47 AR-CGD is performed by introducing a normal copy of the human NCF-1 gene into the blood forming stem cells in the patients bone marrow by using a gene carrier in this study called a lentiviral vector After treatment of the bone marrow cells in a specialised laboratory they are given back to the patient and will grow into functional phagocytic cells There have been no previous clinical trials for patients with p47 AR-CGD however there have been previous gene therapy clinical trials conducted in the UK for patients with the most common form of CGD known as X-CGD
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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