For Healthcare Professionals

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

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About the study

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.
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

  1. Males age 18-75 years, inclusive
  2. 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
  3. Plasma FIX activity ≤2% (<1% for first cohort; then per protocol)
  4. 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

  1. 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)
  2. Documented prior allergic reaction to any FIX product
  3. Detectable AAV8 neutralizing antibodies
  4. Markers of hepatic inflammation or overt or occult cirrhosis as evidenced by one or more of the following:
  5. Platelet count <175,000/μL
  6. Albumin ≤3.5 g/dL
  7. Total bilirubin >1.5 x ULN and direct bilirubin ≥0.5 mg/dL
  8. Alkaline phosphatase >2.0 x ULN
  9. ALT or AST >2.0 x ULN (except for subjects who are HIV infected)
  10. Liver biopsy in the past indicating moderate or severe fibrosis (Metavir staging of 2 or greater)
  11. History of ascites, varices, variceal hemorrhage or hepatic encephalopathy
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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply


Contact the study center to learn if this study is a good match for you.
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Study’s details


Contition

Hemophilia B

Age

18+

Phase

Phase 1/Phase 2

Participants needed

30

Est. Completion Date

Oct 2030

Treatment type

Interventional


Sponsor

Takeda

ClinicalTrials.gov identifier

NCT01687608

Study number

AskBio009-101

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