One-Shot gene therapy could free hemophilia b patients from frequent injections
NCT ID NCT05203679
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This clinical trial is testing a one-time gene therapy called BBM-H901 for people with hemophilia B, a bleeding disorder caused by low levels of clotting factor IX. The therapy uses a harmless virus to deliver a working copy of the factor IX gene to the liver, so the body can produce its own clotting factor. The study involves 32 adult men with severe hemophilia B and will monitor safety, liver function, and how well the treatment reduces bleeding episodes over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
BBM-H901 (a gene therapy using a harmless virus to deliver a working copy of the factor IX gene to the liver)
What this could lead to
If it works, this could give people with hemophilia B a lasting increase in clotting factor, reducing or eliminating the need for regular injections.
What could go wrong
This is an early-to-mid stage trial with only 32 participants. The gene therapy may not work for everyone, and there are risks like liver inflammation or an immune reaction that could reduce its effect.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Anhui Provincial Hospital
Hefei, Anhui, 230022, China
-
Henan Cancer Hospital
Zhengzhou, Henan, 450003, China
-
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, 300020, China
-
Nanfang Hospital Southern Medical University
Guangzhou, Guangdong, 510515, China
-
North China University of Science and Technology Affiliated Hospital
Tangshan, Hebei, 063000, China
-
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100005, China
-
The Second Hospital of Shanxi Medical University
Taiyuan, Shanxi, 030001, China
-
The Second People's Hospital of Shenzhen
Shenzhen, Guangdong, 518025, China
-
The second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, 650032, China