Summary

for males ages 18-75 (full criteria)
at San Diego, California and other locations
study started
estimated completion:

Description

Summary

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.

Official Title

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

Details

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.

Keywords

Hemophilia B factor IX deficiency gene therapy Hemophilia A AskBio009 AskBio009 Dose Escalation

Eligibility

You can join if…

Open to males ages 18-75

  • 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 CAN'T join 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

Locations

  • University of California at San Diego Medical Center
    San Diego California 92103-8651 United States
  • Orthopaedic Hemophilia Treatment Center
    Los Angeles California 90007 United States
  • Children's Hospital Los Angeles
    Los Angeles California 90027 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Baxalta now part of Shire
ID
NCT01687608
Phase
Phase 1/2
Study Type
Interventional
Last Updated
November 6, 2017