Severe Hemophilia APhase 1/2NCT07548411

To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A

A Phase 1/2 study testing GS1191-0445 injection in people with Severe Hemophilia A, run by Gritgen Therapeutics Co., Ltd. at 2 sites in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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Trial summary

This study is a single-arm, open-label study evaluating the safety and efficacy of GS1191-0445 injection as a single dose in Chinese subjects with severe hemophilia A. GS1191-0445 is an adeno-associated virus 8 (AAV8)-delivered gene therapy designed to express B-domain deleted human factor VIII (FVIII) under the… Read more about this study

Who is it for?: Ages 18 to 65 years, men only
Conditions the study is for: Severe Hemophilia A
What is being tested?: GS1191-0445 injection
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 7 participants
Where: 2 sitesChina
Study dates: 10 Jul 2023to 23 Dec 2029
Phase: Phase 1/2

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 65 years
Men only
No healthy volunteers
Adult, Older adult

Who may be able to join

Understand the purpose and risks of the study and provide informed consent in accordance with national and local privacy laws:
Subject must be male, aged ≥18 years old at the time of signing informed consent:
Participants with confirmed severe hemophilia A in their pre-admission history and based on clinical laboratory examination;
Subjects had used FVIII products for at least 150 exposure days (ED) before enrollment;
Subject has received continuous prophylactic treatment with exogenous FVIII for one year prior to enrollment or has been treated with exogenous FVIII on demand;
Subject has no history of hypersensitivity or allergic reactions related to the administration of FVIII agents;

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Subject has no history of FVIII inhibitors.
Subjects agree to use a reliable barrier contraceptive method from the date of signing the informed consent
Subject is willing and able to follow planned visits, treatment plans, and other study procedures.

Who cannot join

The subject has any hemorrhagic disorder not related to hemophilia A,
Abnormal liver function test results of subjects during screening.
Abnormal laboratory examination of subjects during screening
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;
Active systemic immune disease.

Trial locations and contacts

2 sites in 1 country.

China2 sites

Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, 300020
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, 430022

Other trials in China

About this study

Official title: A Single-arm, Open-label Study Evaluating the Safety and Efficacy of a Single Dose of GS1191-0445 Injection in Chinese Subjects With Severe Hemophilia A

This study is a single-arm, open-label study evaluating the safety and efficacy of GS1191-0445 injection as a single dose in Chinese subjects with severe hemophilia A.

GS1191-0445 is an adeno-associated virus 8 (AAV8)-delivered gene therapy designed to express B-domain deleted human factor VIII (FVIII) under the regulation of a human liver-specific promoter. Following a single intravenous administration, AAV8 gene expression cassette, which transfects hepatocytes and facilitates the specific expression and secretion of FVIII into the blood.

Conditions: Severe Hemophilia A
Being tested: GS1191-0445 injection
Medical subject headings: Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

GS1191-0445 injectionDrug
A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg

Study groups in NCT07548411
Study group	Role	What they receive
3E12 vg/kg	Experimental	Not described Receives: Drug: GS1191-0445 injection

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of participants with Adverse Events (AE) as assessed by CTCAE v5.0, including Adverse Event of Special Interests (AESI) and Serious Adverse Events (SAE);
Measured: Five years after infusion
Number of Participants with Thrombosis Risk: In subjects with >150% FⅧ:C post-GS1191-0445 infusion, VTE risk will be assessed via Caprini model, coagulation function, D-dimer, FDP, and TAT;
Measured: Five years after infusion
The shedding of GS1191-0445 viral vector: Viral vector titers in serum, saliva, urine, semen and fecal will be monitored;
Measured: Five years after infusion
Total FⅧ Antibody Levels: Total FⅧ antibody levels will be measured to determine the immunogenicity of FⅧ expression protein;
Measured: Five years after infusion
FVIII inhibitor: Factor Ⅷ inhibitor will be measured to determine the immunogenicity of FⅧ expression protein;
Measured: Five years after infusion
The number of dose-limiting toxicity (DLT) events will be determined by the Safety Review Committee (SRC), at least 12 weeks after GS1191-0445 infusion.
Measured: Five years after infusion
The Change of Laboratory Values: Change in serum chemistry values including liver function tests, hematology, and urinalysis;
Measured: Five years after infusion
Changes for vital signs: Includes sitting blood pressure (mmHg), respiratory rate (breaths/min), body temperature (°C), and pulse rate (beats/min);
Measured: Five years after infusion
Changes for physical examination: Includes skin, mucous membranes, lymph nodes, head and neck, chest (heart, lungs), abdomen, muscles, nervous system, spine/extremities;
Measured: Five years after infusion
The immunogenicity of AAV capsid protein: Collection of Peripheral Blood Mononuclear Cell (PBMC) and serum samples for vector shedding detection;
Measured: Five years after infusion

Secondary outcomes (6)

Vector-derived FⅧ Activity Level: Validated methods will be used to measure vector-derived FⅧ activity, including peak and steady state following GS1191-0445 infusion；
Measured: Day 4 to Week 52 after infusion
Total Consumption of Exogenous FⅧ Infusion;
Measured: Weeks 3 to 52 and five years after infusion
Annualized Consumption of FⅧ Infusion；
Measured: Weeks 3 to 52 and five years after infusion
Number of bleeding events requiring exogenous FⅧ infusion: To assess the number of bleeding events requiring exogenous FⅧ infusion after administration;
Measured: Weeks 3 to 52 and five years after infusion
Number of bleeding events: To assess bleeding events, including spontaneous, traumatic and untreated bleeding events after administration
Measured: Weeks 3 to 52 and five years after infusion
Number of joint bleeding events: To assess joint bleeding events after administration
Measured: Weeks 3 to 52 and five years after infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

10 Jul 202323 Dec 2029

First posted23 Apr 2026
Study start10 Jul 2023
Primary completion31 Dec 2024
Study completion23 Dec 2029 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Severe Hemophilia AStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 7 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07548411
Sponsor's protocol ID: GS1191-0445-GTHA-CN01
Status last verified: Apr 2026
First submitted: 26 Jan 2026
Last updated: 6 May 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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