Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03369444 · ClinicalTrials.gov registry record · Phase 1
A Factor IX Gene Therapy Study (FIX-GT)
A Phase 1 study, sponsored by University College, London.
- Terminated
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
NCT03369444 is a Phase 1 study that was terminated before completion, run by University College, London. The registered enrollment target is 10 participants.
The verdict
NCT03369444, a Phase 1 study, was terminated before completion, sponsored by University College, London.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
Study Summary
Severe haemophilia B (HB) is a bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor; with severe haemophilia B, levels of clotting factor IX (FIX) (nine) are very low and affected individuals can suffer life threatening bleeding episodes. HB mainly affects boys and men (normally one in every 30,000 males). Current treatment for HB involves intravenous infusions of factor IX as regular treatment (Prophylaxis) or 'on demand'. On demand treatment is highly effective at stopping bleeding but cannot fully reverse long-term damage that follows after a bleed. Regular treatment can prevent bleeding, however can be invasive for patients and also expensive. This research study aims to test the safety and effectiveness of a gene therapy which produces Factor IX protein in the body. The gene will be given using an inactivated virus called "the vector" ( FLT180a), in a single infusion. The vector has been developed from a virus known as an adeno- associated virus, that has been changed so that it is unable to cause a viral infection in humans. This "inactivated" virus is further altered to carry the Factor IX gene and to make its way within liver cells where Factor IX protein is normally made. Up to three different doses cohorts of FLT180a will be tested, in up to 24 patients with severe haemophilia B. Patients will be recruited from haemophilia centres in the EU and US. Patients will be in the trial for approximately 40 weeks and will undergo procedures including physical examinations, bloods tests, ECGs and liver ultrasounds.
Interventions
- BIOLOGICAL FLT180a
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2017-12-05 |
| Est. Completion | 2020-10-20 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03369444
The ClinicalTrials.gov registry entry for NCT03369444 describes a study currently listed as terminated, 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 College, London, which has 8 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which FLT180a is the first listed.
NCT03369444 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03369444 about?
NCT03369444 is a clinical study titled "A Factor IX Gene Therapy Study (FIX-GT)". Severe haemophilia B (HB) is a bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor; with severe haemophilia B, levels of clotting factor IX (FIX) (nine) are very low and affected individuals can s...
What is the current status of trial NCT03369444?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2017-12-05. Estimated completion is 2020-10-20.
What interventions are being tested in trial NCT03369444?
The interventions under investigation include: FLT180a (BIOLOGICAL).
Who is sponsoring clinical trial NCT03369444?
This trial is sponsored by University College, London, which has 8 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.