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

NCT ID NCT06111638

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.

Disclaimer Read more

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 A are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Affiliated Hospital of Guizhou Medical University

    RECRUITING

    Guiyang, Guizhou, 550001, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College

    RECRUITING

    Tianjin, Tianjin Municipality, 300020, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

    RECRUITING

    Beijing, Beijing Municipality, 100005, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Sichuan Provincial People's Hospital

    RECRUITING

    Chengde, Sichuan, 610031, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Southern Hospital, Southern Medical University

    RECRUITING

    Guangzhou, Guangdong, 510515, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • The Affiliated Hospital of Northwest University Xi'an No.3 Hospital

    RECRUITING

    Xi’an, Shanxi, 710000, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • The First Affiliated Hospital of Soochow University

    RECRUITING

    Suzhou, Jiangsu, 215006, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • The Second Affiliated Hospital of Kunming Medical University

    RECRUITING

    Kunming, Yunnan, 650032, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Wuhan Union Hospital Affiliated to Huazhong University of Science and Technology

    RECRUITING

    Wuhan, Hubei, 430000, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Xiangya Hospital Central South University

    RECRUITING

    Changsha, Hunan, 410008, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••