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

NCT00979238 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2026-04-02

No results posted yet for this 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

  • Hemophilia B

Interventions

GENETIC

Gene Transfer

Peripheral vein infusion of scAAV2/8-LP1-hFIXco vector once per participant

DRUG

scAAV2/8-LP1-hFIXco

Peripheral vein infusion of scAAV2/8-LP1-hFIXco vector once per participant.

Sponsors & Collaborators

Principal Investigators

  • Ulrike Reiss, MD · St. Jude Children's Research Hospital

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-02-22
Primary Completion
2026-03-26
Completion
2026-03-26
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00979238 on ClinicalTrials.gov