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.
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
- 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:
- 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
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 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.
- 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)
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
- 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
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07548411
- Acronym / sponsor ID
- GS1191-0445-GTHA-CN01
- Conditions
- Severe Hemophilia A
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Gritgen Therapeutics Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 10 Jul 2023 (actual)
- Primary completion
- 31 Dec 2024 (actual)
- Study completion
- 23 Dec 2029 (estimated)
- First posted
- 23 Apr 2026 (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 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.
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 (9 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:
- 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;
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
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);
Time frame: 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;
Time frame: Five years after infusion
The shedding of GS1191-0445 viral vector: Viral vector titers in serum, saliva, urine, semen and fecal will be monitored;
Time frame: Five years after infusion
Total FⅧ Antibody Levels: Total FⅧ antibody levels will be measured to determine the immunogenicity of FⅧ expression protein;
Time frame: Five years after infusion
FVIII inhibitor: Factor Ⅷ inhibitor will be measured to determine the immunogenicity of FⅧ expression protein;
Time frame: 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.
Time frame: Five years after infusion
The Change of Laboratory Values: Change in serum chemistry values including liver function tests, hematology, and urinalysis;
Time frame: 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);
Time frame: 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;
Time frame: Five years after infusion
The immunogenicity of AAV capsid protein: Collection of Peripheral Blood Mononuclear Cell (PBMC) and serum samples for vector shedding detection;
Time frame: Five years after infusion
6 secondary outcomes
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；
Time frame: Day 4 to Week 52 after infusion
Total Consumption of Exogenous FⅧ Infusion;
Time frame: Weeks 3 to 52 and five years after infusion
Annualized Consumption of FⅧ Infusion；
Time frame: 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;
Time frame: 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
Time frame: Weeks 3 to 52 and five years after infusion
Number of joint bleeding events: To assess joint bleeding events after administration
Time frame: Weeks 3 to 52 and five 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 6 May 2026.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
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 9 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: under way, not looking for participants, as of the sponsor's last update on 6 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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); (time frame: Five years after infusion). The study lists 10 primary outcomes in all. 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 6 May 2026; AzTrials last refreshed its copy on 8 Nov 2026.