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NCT00979238 · ClinicalTrials.gov registry record · Phase 1
Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
A Phase 1 study of Hemophilia B, sponsored by St. Jude Children's Research Hospital.
- Active
- Registry status
- Phase 1
- Development phase
- 14
- Enrollment target
- 8
- Study locations
NCT00979238 is a Phase 1 study of Hemophilia B that is active but no longer recruiting, run by St. Jude Children's Research Hospital. The registered enrollment target is 14 participants, below the 96-participant average among 20 other Hemophilia B trials with a reported enrollment target (85% lower). The trial reports 8 study locations across 7 states.
The verdict
NCT00979238, a Phase 1 study of Hemophilia B, is active but no longer recruiting, sponsored by St. Jude Children's Research Hospital.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 14 participants
- Enrollment target
- 8
- Study locations
Study Summary
The purpose of this study is to determine the safety of giving a normal factor IX gene to treat individuals who have an abnormal or no factor IX gene. Recruitment will be limited to adults (≥ 18 years) with a confirmed diagnosis of hemophilia B (HB), resulting from a missense mutation in the coagulation factor IX (FIX) gene or a nonsense mutation that has not been associated with an inhibitor. Only subjects who have no evidence of active hepatitis or anti-hFIX antibodies, and who have been treated/exposed to Factor IX concentrates for at least ten years and have had an average of 3 bleeding episodes per year requiring FIX administration will be enrolled. Patients will be recruited within the United States for treatment at St. Jude Children's Research Hospital, and patients will be recruited in England and other countries for treatment in London by our British collaborators.
Conditions Studied
Interventions
- GENETIC Gene Transfer
- DRUG scAAV2/8-LP1-hFIXco
Study Locations (8)
Texas
- University of Texas Southwestern - Dallas
- Scott and White Memorial Hospital - Temple
California
- Stanford Medical School - Stanford
Kentucky
- University of Kentucky - Lexington
Oregon
- Oregon Health and Science University - Portland
Pennsylvania
- Hemophilia Center of Western Pennsylvania - Pittsburgh
Tennessee
- St. Jude Children's Research Hospital - Memphis
Other
- Katharine Dormandy Haemophilia Centre and Haemostasis Unit, University College of London - London
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 14 participants |
| Start Date | 2010-02-22 |
| Est. Completion | 2026-03 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00979238
The ClinicalTrials.gov registry entry for NCT00979238 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 14 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 96-participant average among 20 other Hemophilia B trials with a reported enrollment target (85% lower). The listed sponsor is St. Jude Children's Research Hospital, which has 410 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Hemophilia B appearing as the primary indexed condition, and to 2 interventions - of which Gene Transfer is the first listed.
NCT00979238 reports 8 study locations spanning 7 distinct geographic areas - top geographies include Texas, California, Kentucky.
Frequently Asked Questions
What is clinical trial NCT00979238 about?
NCT00979238 is a clinical study titled "Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B". The purpose of this study is to determine the safety of giving a normal factor IX gene to treat individuals who have an abnormal or no factor IX gene. Recruitment will be limited to adults (≥ 18 years) with a confirmed diagnosis of hemophilia B (HB), resulting from a missense mutation in the coagula...
What is the current status of trial NCT00979238?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 14 participants. The study started on 2010-02-22. Estimated completion is 2026-03.
What conditions does trial NCT00979238 study?
This clinical trial studies the following conditions: Hemophilia B.
What interventions are being tested in trial NCT00979238?
The interventions under investigation include: Gene Transfer (GENETIC), scAAV2/8-LP1-hFIXco (DRUG).
Who is sponsoring clinical trial NCT00979238?
This trial is sponsored by St. Jude Children's Research Hospital, which has 410 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00979238 being conducted?
This trial has 8 study locations across California, Kentucky, Oregon, Pennsylvania, Tennessee. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Read our methodology - how this data is sourced, computed, and verified.
Related
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