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

April 30, 2026 updated by: Gritgen Therapeutics Co., Ltd.

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
7

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: 3E12 vg/kg
Drug: GS1191-0445 injection
A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
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
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
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
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
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
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
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
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
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
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
Five years after infusion

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
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
Weeks 3 to 52 and five years after infusion
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
Day 4 to Week 52 after infusion
Total Consumption of Exogenous FⅧ Infusion;
Time Frame: Weeks 3 to 52 and five years after infusion
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
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
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
Weeks 3 to 52 and five years after infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Gritgen Therapeutics Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 10, 2023

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2024

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 23, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 26, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 21, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 23, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 6, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 30, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GS1191-0445-GTHA-CN01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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