One-Time gene therapy could free hemophilia patients from frequent infusions

NCT ID NCT06111638

Recruiting now Disease control Sponsor: Shanghai Xinzhi BioMed Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This trial tests a one-time gene therapy called BBM-H803 for adults with severe hemophilia A. The treatment uses a harmless virus to deliver a working gene to the liver, so the body can produce its own clotting factor VIII. The study involves 55 participants and will check safety, liver function, and how well it 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: BBM-H803 (a gene therapy using a harmless virus to deliver a working factor VIII gene to the liver)
What this could lead to: If successful, this could allow people with severe hemophilia A to produce their own clotting factor, reducing or stopping the need for regular factor infusions.
What could go wrong: This is an early-to-mid stage trial with only 55 participants. Gene therapy may not work for everyone, and there are risks like liver inflammation or an immune reaction to the virus.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

hemophilia A

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEMOPHILIA A

Contacts and locations

Locations

Affiliated Hospital of Guizhou Medical University
RECRUITING

Guiyang, Guizhou, 550001, China
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
RECRUITING

Tianjin, Tianjin Municipality, 300020, China
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
RECRUITING

Beijing, Beijing Municipality, 100005, China
Sichuan Provincial People's Hospital
RECRUITING

Chengde, Sichuan, 610031, China
Southern Hospital, Southern Medical University
RECRUITING

Guangzhou, Guangdong, 510515, China
The Affiliated Hospital of Northwest University Xi'an No.3 Hospital
RECRUITING

Xi’an, Shanxi, 710000, China
The First Affiliated Hospital of Soochow University
RECRUITING

Suzhou, Jiangsu, 215006, China
The Second Affiliated Hospital of Kunming Medical University
RECRUITING

Kunming, Yunnan, 650032, China
Wuhan Union Hospital Affiliated to Huazhong University of Science and Technology
RECRUITING

Wuhan, Hubei, 430000, China
Xiangya Hospital Central South University
RECRUITING

Changsha, Hunan, 410008, China

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