Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Hemophilia B

In brief

The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B.

Key facts

Study ID

NCT01687608

Run by

Baxalta now part of Shire

People needed

30

Starts

2013-02-11

Expected to finish

2030-01-17

Last updated by the study team

2026-03-20

Who can join

Age: 18 and older, up to 75. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Males age 18-75 years, inclusive
• Established hemophilia B with ≥3 hemorrhages per year requiring treatment with exogenous FIX OR use of FIX prophylaxis because of history of frequent bleeding episodes
• Plasma FIX activity ≤2% (<1% for first cohort; then per protocol)
• Negative for active Hepatitis C virus (HCV), defined as Hepatitis C virus antibody negative and negative (undetectable) PCR test for plasma Hepatitis C virus ribonucleic acid (RNA) OR if Hepatitis C virus antibody positive must have ≥2 consecutive negative (undetectable) PCR tests for plasma HCV RNA at least 3 months apart, and negative at screening

You may not qualify if…

• Family history of inhibitor to FIX protein or personal laboratory evidence of having developed inhibitors to FIX protein at any time (>0.6 Bethesda Units on any single test)
• Documented prior allergic reaction to any FIX product
• Detectable AAV8 neutralizing antibodies
• Markers of hepatic inflammation or overt or occult cirrhosis as evidenced by one or more of the following:
• Platelet count <175,000/μL
• Albumin ≤3.5 g/dL
• Total bilirubin >1.5 x ULN and direct bilirubin ≥0.5 mg/dL
• Alkaline phosphatase >2.0 x ULN
• ALT or AST >2.0 x ULN (except for subjects who are HIV infected)
• Liver biopsy in the past indicating moderate or severe fibrosis (Metavir staging of 2 or greater)
• History of ascites, varices, variceal hemorrhage or hepatic encephalopathy

Where it is running

• Orthopaedic Hemophilia Treatment Center — Los Angeles, California, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States
• University of California Davis Medical Center — Sacramento, California, United States
• University of California at San Diego Medical Center — San Diego, California, United States
• U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center — Aurora, Colorado, United States
• Emory University — Atlanta, Georgia, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Children's Hospital of Boston — Boston, Massachusetts, United States
• University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute — Minneapolis, Minnesota, United States
• Mount Sinai Medical Center — New York, New York, United States
• The Hemophilia Center, Oregon Health and Science University — Portland, Oregon, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• Gulf States Hemophilia and Thrombosis Center — Houston, Texas, United States
• Bloodworks Northwest — Seattle, Washington, United States
• BloodCenter of Wisconsin — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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