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Clinical Trial NCT01687608 for Hemophilia B is active, not recruiting. See the Trial Radar Card View and AI discovery tools for all the details. Or ask anything here.
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Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B Phase 1, Phase 2 30 Gene Therapy Open-Label

Active, not recruiting
Clinical Trial NCT01687608 is designed to study Treatment for Hemophilia B. It is a Phase 1 Phase 2 interventional study that is active, not recruiting, having started on 11 February 2013, with plans to enroll 30 participants. Led by Baxalta now part of Shire, it is expected to complete by 17 January 2030. The latest data from ClinicalTrials.gov was last updated on 20 March 2026.
Brief 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.
Detailed Description
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 t...Show More
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

Conditions
Hemophilia B
Publications
Scientific articles and research papers published about this clinical trial:
Other Study IDs
  • AskBio009-101
  • 231401 (Other Identifier) (Sponsor)
NCT ID Number
NCT01687608
Start Date (Actual)
2013-02-11
Last Update Posted
2026-03-20
Completion Date (Estimated)
2030-01-17
Enrollment (Estimated)
30
Study Type
Interventional
PHASE
Phase 1
Phase 2
Status
Active, not recruiting
Keywords
Hemophilia B
factor IX deficiency
gene therapy
Primary Purpose
Treatment
Design Allocation
N/A
Interventional Model
Single Group
Masking
None (Open Label)
Arms / Interventions
Participant Group/ArmIntervention/Treatment
ExperimentalAskBio009 Dose Escalation
Single Dose of a Self-Complementing Optimized Adeno-associated Virus (AAV) Serotype 8 Factor IX Gene Therapy
AskBio009
Single dose IV injection
Primary Outcome Measures
Outcome MeasureMeasure DescriptionTime Frame
Number of patients experiencing treatment-related adverse events by dose group
Infusion to Week 3 and Infusion to end of study
Change from baseline in clinical laboratory evaluations
Change from baseline at week 3 and change from baseline at the end of study
Secondary Outcome Measures
Outcome MeasureMeasure DescriptionTime Frame
Changes from Baseline in FIX activity levels, FIX protein levels, and Bleeding Episode Severity & Frequency
At multiple timepoints from pre-dose through up to 5 years post-dose
Immune Response to AskBio009
At multiple timepoints from pre-dose through up to 5 years post-dose
Detection of AskBio009 genomes in blood, saliva, urine, stool, and semen
At multiple timepoints from pre-dose through up to 1 years post-dose
Participation Assistant
Eligibility Criteria

Eligible Ages
Adult, Older Adult
Minimum Age
18 Years
Eligible Sexes
Male
  • 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

  • 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
Baxalta now part of Shire logoBaxalta now part of Shire
No contact data.
15 Study Locations in 1 Countries

California

Orthopaedic Hemophilia Treatment Center, Los Angeles, California, 90007, United States
Children's Hospital Los Angeles, Los Angeles, California, 90027, United States
University of California Davis Medical Center, Sacramento, California, 95817, United States
University of California at San Diego Medical Center, San Diego, California, 92103-8651, United States

Colorado

U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center, Aurora, Colorado, 80045, United States

Georgia

Emory University, Atlanta, Georgia, 30322, United States

Illinois

Rush University Medical Center, Chicago, Illinois, 60612, United States

Massachusetts

Children's Hospital of Boston, Boston, Massachusetts, 022105, United States

Minnesota

University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute, Minneapolis, Minnesota, 55455, United States

New York

Mount Sinai Medical Center, New York, New York, 10029, United States

Oregon

The Hemophilia Center, Oregon Health and Science University, Portland, Oregon, 97239, United States

South Carolina

Medical University of South Carolina, Charleston, South Carolina, 29425, United States

Texas

Gulf States Hemophilia and Thrombosis Center, Houston, Texas, 77030, United States

Washington

Bloodworks Northwest, Seattle, Washington, 98104, United States

Wisconsin

BloodCenter of Wisconsin, Milwaukee, Wisconsin, 53226, United States