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ACTIVE NOT RECRUITING Phase 1

Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B

NCT00979238 · View on ClinicalTrials.gov ↗

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

FieldValue
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. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 14 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is St. Jude Children's Research Hospital, which has 441 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT00979238 reports 8 study locations spanning 7 distinct geographic areas — top geographies include Texas, California, Kentucky. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT00979238?

The interventions under investigation include: Gene Transfer (GENETIC), scAAV2/8-LP1-hFIXco (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT00979238?

This trial is sponsored by St. Jude Children's Research Hospital, which has 441 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial