MedTrace Logo

Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

NCT00076557 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2007-04-04

No results posted yet for this study

Summary

In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector). Gene therapy is a very new medical technique being used in a number of clinical studies for diseases such as cancer and cystic fibrosis. At this time, the U.S. Food and Drug Administration has approved no gene transfer products for commercial use. To date, 8 subjects have received AAV vector in the muscle for a hemophilia B trial by intramuscular injection, and, to date, 6 subjects have been treated with AAV vector in the current hemophilia B liver trial. Eleven cystic fibrosis subjects have received AAV vector into their nasal sinuses or lungs to date. In this study, AAV human Factor IX vector will be injected into the liver using a catheter inserted into a large blood vessel (called the proper hepatic artery or the right hepatic artery).

Conditions

  • Hemophilia B

Interventions

GENETIC

Adeno-Associated Viral with Human Factor IX

Sponsors & Collaborators

  • Stanford University

    collaborator OTHER

  • Children's Hospital of Philadelphia

    collaborator OTHER

  • The Hemophilia Center of Western Pennsylvania

    collaborator OTHER

  • University of Washington

    collaborator OTHER

  • The University of Texas Health Science Center, Houston

    collaborator OTHER

  • University of Campinas, Brazil

    collaborator OTHER

  • Christian Medical College, Vellore, India

    collaborator OTHER

  • Royal Prince Alfred Hospital, Sydney, Australia

    collaborator OTHER

  • Avigen

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-01-31

Countries

  • United States

Study Locations

More Related Trials

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 NCT00076557 on ClinicalTrials.gov