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



Status:Active, not recruiting
Conditions:Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:18 - 75
Updated:10/3/2018
Start Date:February 11, 2013
End Date:October 28, 2030

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A Phase 1/2 Open-Label, Single Ascending Dose Trial of a Self-Complementing Optimized Adeno-associated Virus Serotype 8 Factor IX Gene Therapy (AskBio009) in Adults With Hemophilia B

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.

Hemophilia B is a genetic X-linked bleeding disorder caused by a deficiency in blood-clotting
Factor IX (FIX) activity. FIX is synthesized in the liver and circulates in the blood as a
proenzyme. Current treatment for hemophilia B is based on replacement of the deficient FIX
with IV injections of recombinant FIX protein prophylactically or as needed to treat bleeding
episodes. This clinical program will test a gene transfer approach involving the use of a
gene delivery vector carrying a FIX gene. This first-in-humans study is intended to evaluate
the safety, kinetics, and if possible, the dose of AskBio009 required to achieve stable
plasma FIX activity between 10% and 40% of normal activity.

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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
We found this trial at
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201 Dowman Dr
Atlanta, Georgia 30303
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4650 Sunset Blvd
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 (323) 660-2450
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1 Gustave L Levy Pl # 271
New York, New York 10029
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Mount Sinai Med Ctr Founded in 1852, The Mount Sinai Hospital is a 1,171-bed, tertiary-care...
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Boston, Massachusetts 02115
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171 Ashley Avenue
Charleston, South Carolina 29425
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1653 W. Congress Parkway
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Houston, Texas 77030
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Los Angeles, California 90007
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Portland, Oregon 97239
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2315 Stockton Blvd.
Sacramento, California 95817
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San Diego, California 92103
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