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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for HEMOPHILIA B are added.
By submitting, you agree to our Terms of use
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
-
Bloodworks Northwest
Seattle, Washington, 98104, United States
-
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
-
Children's Hospital of Boston
Boston, Massachusetts, 022105, United States
-
Emory University
Atlanta, Georgia, 30322, United States
-
Gulf States Hemophilia and Thrombosis Center
Houston, Texas, 77030, United States
-
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
-
Mount Sinai Medical Center
New York, New York, 10029, United States
-
Orthopaedic Hemophilia Treatment Center
Los Angeles, California, 90007, United States
-
Rush University Medical Center
Chicago, Illinois, 60612, United States
-
The Hemophilia Center, Oregon Health and Science University
Portland, Oregon, 97239, United States
-
U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center
Aurora, Colorado, 80045, 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
-
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.
- 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
- Monthly shot could free kids with hemophilia from frequent infusions