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Clinical Trial Report
Gene Therapy Trial Report
Summary
Lentiviral Gene Therapy for p47 AR-CGD
NCTID
NCT05207657
(View at clinicaltrials.gov)
Description
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.
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Development Status
Active
Indication
Autosomal Recessive Chronic Granulomatous Disease (CGD)
Disease Ontology Term
DOID:0070192
Compound Name
PCHIM-p47
Sponsor
Great Ormond Street Hospital for Children NHS Foundation Trust
Funder Type
Other
Recruitment Status
Recruiting
Enrollment Count
5
Results Posted
Not Available
Therapy Information
Target Gene/Variant
NCF1
Therapy Type
Gene transfer
Therapy Route
Ex-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Autologous cells
Target Tissue/Cell
CD34+ cells
Delivery System
Viral transduction
Vector Type
LV
Editor Type
none
Dose 1
Transduced CD34+ cells, minimum dose: 3E6 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2021-06-24
Completion Date
2029-04-01
Last Update
2023-05-16
Participation Criteria
Eligible Age
>=23 Months
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
United Kingdom
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Updated NCTID is NCT06253507 (NIH-sponsored trial)
Resources/Links
Resources/Links
No External Links Available.