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One-Shot gene therapy aims to fix bleeding disorder

NCT ID NCT07548411

Disease control Sponsor: Gritgen Therapeutics Co., Ltd. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Summary

This early-stage trial is testing a single-dose gene therapy called GS1191-0445 in 7 Chinese adults with severe hemophilia A. The therapy uses a harmless virus to deliver a working gene to the liver, so the body can produce its own clotting factor. The main goal is to check safety, but researchers will also see if it reduces bleeding episodes and the need for factor infusions.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
GS1191-0445 injection (gene therapy)
What this could lead to
If it works, this could allow people with severe hemophilia A to produce their own clotting factor, reducing or eliminating the need for regular infusions.
What could go wrong
This is a very early, small trial (only 7 people) focused on safety. The gene therapy may not produce enough clotting factor, could cause side effects like liver issues or blood clots, and long-term effects are unknown.

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

7 people

The number who actually took part.

Started

Jul 2023

Expected to finish

Dec 2029

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 65 years

Sex

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Understand the purpose and risks of the study and provide informed consent in accordance with national and local privacy laws: 2. Subject must be male, aged ≥18 years old at the time of signing informed consent: 3. Participants with confirmed severe hemophilia A in their pre-admission history and based on clinical laboratory examination; 4. Subjects had used FVIII products for at least 150 exposure days (ED) before enrollment; 5. Subject has received continuous prophylactic treatment with exogenous FVIII for one year prior to enrollment or has been treated with exogenous FVIII on demand; 6. Subject has no history of hypersensitivity or allergic reactions related to the administration of FVIII agents; 7. Subject has no history of FVIII inhibitors. 8. Subjects agree to use a reliable barrier contraceptive method from the date of signing the informed consent 9. Subject is willing and able to follow planned visits, treatment plans, and other study procedures. Exclusion Criteria: 1. The subject has any hemorrhagic disorder not related to hemophilia A, 2. Abnormal liver function test results of subjects during screening. 3. Abnormal laboratory examination of subjects during screening 4. The subject has acute or chronic hepatitis B virus (HBV) infection or chronic hepatitis C virus (HCV) infection; Or are receiving antiviral treatment for hepatitis B and C; 5. Active systemic immune disease.

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Conditions

The condition(s) this trial relates to.

hemophilia A severe hemophilia A

As listed by the trial registrant

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

Severe hemophilia A

Contacts and locations

Locations

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

    Tianjin, Tianjin Municipality, 300020, China

  • Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

    Wuhan, Hubei, 430022, China

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