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A Phase I Safety Study in Subjects With Severe Hemophilia B (Factor IX Deficiency) Using Adeno-Associated Viral Vector to Deliver the Gene for Human Factor IX Into the Liver


Phase 1/Phase 2
18 Years
N/A
Not Enrolling
Male
Hemophilia B

Thank you

Trial Information

A Phase I Safety Study in Subjects With Severe Hemophilia B (Factor IX Deficiency) Using Adeno-Associated Viral Vector to Deliver the Gene for Human Factor IX Into the Liver

Inclusion Criteria


- Males with severe hemophilia B with Factor IX activity level < 1% of normal.

- Life expectancy of > 1 year.

- Age > 18 years old.

- Ability to give informed consent.

- Greater than twenty exposure days of treatment with Factor IX protein.

- No history or presence of an inhibitor to Factor IX protein.

- Subjects must be able to receive Factor IX protein on a home infusion protocol.

- Subjects must have a normal protime (PT).

- Hepatitis C infected subjects will be evaluated for liver fibrosis based on liver
biopsy data graded on a scale of 0–4 (Poynard et. al., 1997). Subjects who are
Hepatitis C antibody and RNA positive and have not had a liver biopsy within the last
36 months will be required to have one.

- Subjects must have low AAV titer.

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Authority:

United States: Food and Drug Administration

Study ID:

BB IND 9398

NCT ID:

NCT00076557

Start Date:

January 2004

Completion Date:

Related Keywords:

  • Hemophilia B
  • Hemophilia B
  • Factor IX
  • Gene Transfer
  • Adeno-Associated Virus (AAV)
  • Hemophilia B
  • Hemophilia A

Name

Location
Stanford University Stanford, California  94305
The Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
The Hemophilia Center of Western Pennsylvania Pittsburgh, Pennsylvania  15213