Single gene shot could tame hemophilia b bleeding
NCT ID NCT01687608
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a single intravenous dose of a gene therapy called AskBio009 in up to 30 adults with severe hemophilia B. The therapy delivers a working copy of the factor IX gene to help the body produce its own clotting factor. The main goal is to check safety, but researchers will also measure whether it raises factor IX levels and reduces bleeding episodes.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- AskBio009 (BAX 335) gene therapy
- What this could lead to
- If successful, this could provide long-term control of hemophilia B with a single injection, reducing or eliminating the need for regular factor IX infusions.
- What could go wrong
- This is an early phase 1/2 trial with only 30 participants, so results may not apply to everyone. Gene therapy can cause immune reactions or may not raise factor IX levels enough to stop bleeding.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
30 people
The number who actually took part.
- Started
-
Feb 2013
- Expected to finish
-
Jan 2030
An estimate. End dates often move.
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 75 years
- Sex
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Male participants only
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
BloodCenter of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Bloodworks Northwest
Seattle, Washington, 98104, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Boston
Boston, Massachusetts, 022105, United States
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Emory University
Atlanta, Georgia, 30322, United States
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Gulf States Hemophilia and Thrombosis Center
Houston, Texas, 77030, United States
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Mount Sinai Medical Center
New York, New York, 10029, United States
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Orthopaedic Hemophilia Treatment Center
Los Angeles, California, 90007, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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The Hemophilia Center, Oregon Health and Science University
Portland, Oregon, 97239, United States
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U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center
Aurora, Colorado, 80045, United States
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University of California Davis Medical Center
Sacramento, California, 95817, United States
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University of California at San Diego Medical Center
San Diego, California, 92103-8651, United States
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University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute
Minneapolis, Minnesota, 55455, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- A Once-a-Week shot could transform hemophilia Care—Even for those with inhibitors
- Gene Editing's lasting impact: a 10-Year safety watch
- Can a single gene shot free hemophilia b patients from regular infusions?
- Can a video call replace the clinic for hemophilia pain relief?
- Newborn screening study aims to catch rare diseases at birth