Hemophilia APhase 3NCT06833983
To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
A Phase 3 study testing GS1191-0445 injection in people with Hemophilia A, run by Gritgen Therapeutics Co., Ltd. at 13 sites in China.
Trial summary
This study is a single-arm, open-label, multicenter study evaluating the efficacy and safety of GS1191-0445 injection as a single dose in Chinese subjects with hemophilia A. GS1191-0445 is an AAV8-based gene therapy vector designed to express B-domain deleted human factor VIII (FVIII) under the regulation of a human… Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- 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
- 50 participants
- Where
- 13 sitesChina
- Study dates
- 31 Mar 2025to 30 Nov 2030
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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, and ≤65 years old:
- Participants with confirmed 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 no prior 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
13 sites in 1 country. Contact the study team or a site near you to ask about taking part.
13 sites in 1 country
China13 sites, 13 recruiting
- Not yet recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100005
Tienan Zhu, Doctor, +8618611743292, Zhutn@pumch.cn
- Not yet recruiting
Xiangya Hospital of Central South University
Changsha, Hunan, 410028
Xielan Zhao, +8613707489198, zhaoxl9198@163.com
- Not yet recruiting
Nanfang Hospital Southern Medical University
Guangzhou, Guangdong, 510515
Jing Sun, +8613316202696, jsun-cn@qq.com
- Not yet recruiting
Anhui Provincial Hospital
Hefei, Anhui, 230001
Changcheng Zheng, +8613956961162, zhengchch1123@ustc.edu.cn
- Recruiting
Central Hospital Affiliated to Shandong First Medical University
Jinan, Shandong, 250013
Yun Chen, +8613370582720, chyun008@163.com
- Not yet recruiting
The second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, 650101
Zeping Zhou, +8618788571605, Zhouzeping@kmmu.edu.cn
- Not yet recruiting
First Hospital of Lanzhou University
Lanzhou, Gansu, 730099
Yaming Xi, +8613919110815, xiyaming02@163.com
- Not yet recruiting
Jiangxi Provincial People's Hospital
Nanchang, Jiangxi, 344001
Chenghao Jin, +8613699500207, jinch227@aliyun.com
- Not yet recruiting
Nanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School
Nanjing, Jiangsu, 210000
Rongfu Zhou, +8613605142342, rfzhoucn@163.com
- Not yet recruiting
North China University of Science and Technology Affiliated Hospital
Tangshan, Hebei, 063000
Zhenyu Yan, +8618831508262, hbyzy2011@163.com
- Recruiting
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, 300020
Lei Zhang, Doctor, +8613502118379, zhanglei1@ihcams.ac.cn
- Not yet recruiting
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, 430022
Yu Hu, +8613986183871, dr_huyu387@163.com
- Not yet recruiting
Henan Cancer Hospital
Zhengzhou, Henan, 450003
Hu Zhou, +8613939068863, Togerzhoupumc@163.com
About this study
Official title: A Single-arm, Open-label, Multicenter Study Evaluating the Efficacy and Safety of GS1191-0445 Injection as a Single Dose in Chinese Subjects With Hemophilia A
This study is a single-arm, open-label, multicenter study evaluating the efficacy and safety of GS1191-0445 injection as a single dose in Chinese subjects with hemophilia A.
GS1191-0445 is an AAV8-based gene therapy vector 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 targets hepatocytes and facilitates the specific expression and secretion of FVIII into the bloodstream.
- Conditions
- 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 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.
Annualized Bleeding Rate (ABR)
To evaluate the efficacy of a single intravenous administration of GS1191-0445 in subjects with hemophilia A
Measured: Weeks 3 to 52 after infusion
Secondary outcomes (3)
Incidence of adverse events (AE)
The incidence of adverse events (AE) within 52 weeks after GS1191-0445 infusion, including serious adverse events (SAE) and adverse events of special concern (AESI)
Measured: Within 52 weeks after infusion
Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion
Evaluate the safety of a single intravenous infusion of GS1191-0445 injection in hemophilia A subjects
Measured: Within 52 weeks after infusion
Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years
Evaluate the changes in annualized bleeding rate (ABR) and FVIII activity from baseline during the 2nd to 5th year after GS1191-0445 injection
Measured: 2-5 years after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2025
- Study start31 Mar 2025
- Primary completion30 Nov 2026 (estimated)
- Study completion30 Nov 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gritgen Therapeutics Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06833983
- Sponsor's protocol ID
- GS1191-0445-GTHA-CN02
- Status last verified
- Sep 2025
- First submitted
- 28 Jan 2025
- Last updated
- 9 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Open-label, Multicenter Study Evaluating the Efficacy and Safety of GS1191-0445 Injection as a Single Dose in Chinese Subjects With Hemophilia A
- ClinicalTrials.gov ID
- NCT06833983
- Acronym / sponsor ID
- GS1191-0445-GTHA-CN02
- Conditions
- Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Gritgen Therapeutics Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 31 Mar 2025 (actual)
- Primary completion
- 30 Nov 2026 (estimated)
- Study completion
- 30 Nov 2030 (estimated)
- First posted
- 19 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a single-arm, open-label, multicenter study evaluating the efficacy and safety of GS1191-0445 injection as a single dose in Chinese subjects with hemophilia A.
GS1191-0445 is an AAV8-based gene therapy vector 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 targets hepatocytes and facilitates the specific expression and secretion of FVIII into the bloodstream.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 3E12 vg/kg | Experimental | Drug: GS1191-0445 injection |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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, and ≤65 years old:
- Participants with confirmed 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 no prior history of FVIII inhibitors;
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GS1191-0445 injectionA single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg | Drug | 3E12 vg/kg |
Primary outcome
Annualized Bleeding Rate (ABR)
Time frame: Weeks 3 to 52 after infusion
To evaluate the efficacy of a single intravenous administration of GS1191-0445 in subjects with hemophilia A
3 secondary outcomes
Incidence of adverse events (AE)
Time frame: Within 52 weeks after infusion
Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion
Time frame: Within 52 weeks after infusion
Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years
Time frame: 2-5 years after infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing GS1191-0445 injection in people with Hemophilia A, run by Gritgen Therapeutics Co., Ltd. at 13 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yanfang Li (+8613817454595, yanfang.li@gritgen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Bleeding Rate (ABR) (time frame: Weeks 3 to 52 after infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Dec 2025.