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NCT02234934 · ClinicalTrials.gov registry record · Phase 1
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
A Phase 1 study, sponsored by University of California, Los Angeles.
- Completed
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
NCT02234934 is a Phase 1 study that has completed, run by University of California, Los Angeles. The registered enrollment target is 10 participants.
The verdict
NCT02234934, a Phase 1 study, has completed, sponsored by University of California, Los Angeles.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
Study Summary
Chronic Granulomatous Disease (CGD) is an inherited immunodeficiency disorder which results from defects that prevent white blood cells from effectively killing bacteria, fungi and other microorganisms. Chronic granulomatous inflammation may compromise vital organs and account for additional morbidity. CGD is thought to affect approximately 1 in 200,000 persons, although the real incidence might be higher due to under-diagnosis of milder phenotypes. The first gene therapy approaches in X-CGD have shown that effective gene therapy requires bone-marrow (BM) conditioning with chemotherapy to make space for the gene-modified cells to engraft. These studies demonstrated that transplantation of gene modified stem cells led to production of white blood cells that could clear existing infections. However, some trials using mouse-derived retroviral vectors were complicated by the development of myelodysplasia and leukemia-like growth of blood cells. This trial will evaluate a new lentiviral vector that may be able to correct the defect, but have much lower risk for the complication. This study is a two-part, prospective non-controlled, non-randomized Phase I/II clinical trial to assess the safety, feasibility and efficacy of cellular gene therapy in patients with chronic granulomatous disease using transplantation of autologous bone marrow CD34+ cells transduced ex vivo by the G1XCGD lentiviral vector containing the human CGD gene. Primary objectives include evaluation of safety and evaluation of efficacy by biochemical and functional reconstitution in progeny of engrafted cells and stability at 12 months. Secondary objectives include evaluation of clinical efficacy, longitudinal evaluation of clinical effect in terms of augmented immunity against bacterial and fungal infection, transduction of CD34+ hematopoietic cells from X-CGD patients by ex vivo lentivirus-mediated gene transfer, and evaluation of engraftment kinetics and stability. Approximately 3-6 patients will be
Interventions
- BIOLOGICAL Lentiviral G1XCGD Gene Therapy
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2015-10-29 |
| Est. Completion | 2024-12-01 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02234934
The ClinicalTrials.gov registry entry for NCT02234934 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is University of California, Los Angeles, which has 897 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Lentiviral G1XCGD Gene Therapy is the first listed.
NCT02234934 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT02234934 about?
NCT02234934 is a clinical study titled "Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease". Chronic Granulomatous Disease (CGD) is an inherited immunodeficiency disorder which results from defects that prevent white blood cells from effectively killing bacteria, fungi and other microorganisms. Chronic granulomatous inflammation may compromise vital organs and account for additional morbidi...
What is the current status of trial NCT02234934?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2015-10-29. Estimated completion is 2024-12-01.
What interventions are being tested in trial NCT02234934?
The interventions under investigation include: Lentiviral G1XCGD Gene Therapy (BIOLOGICAL).
Who is sponsoring clinical trial NCT02234934?
This trial is sponsored by University of California, Los Angeles, which has 897 total clinical trials registered on ClinicalTrials.gov.
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