To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A (NCT07548411) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A
China7 participantsStarted 2023-07-10
Plain-language 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 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.
Who can participate
Age range
18 Years – 65 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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;
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
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);
Timeframe: Five years after infusion
2
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;
Timeframe: Five years after infusion
3
The shedding of GS1191-0445 viral vector: Viral vector titers in serum, saliva, urine, semen and fecal will be monitored;
Timeframe: Five years after infusion
4
Total FⅧ Antibody Levels: Total FⅧ antibody levels will be measured to determine the immunogenicity of FⅧ expression protein;
Timeframe: Five years after infusion
5
FVIII inhibitor: Factor Ⅷ inhibitor will be measured to determine the immunogenicity of FⅧ expression protein;
Timeframe: Five years after infusion
6
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.
. Subjects agree to use a reliable barrier contraceptive method from the date of signing the informed consent
Exclusion criteria
. 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.
7
The Change of Laboratory Values: Change in serum chemistry values including liver function tests, hematology, and urinalysis;
Timeframe: Five years after infusion
8
Changes for vital signs: Includes sitting blood pressure (mmHg), respiratory rate (breaths/min), body temperature (°C), and pulse rate (beats/min);
Timeframe: Five years after infusion
9
Changes for physical examination: Includes skin, mucous membranes, lymph nodes, head and neck, chest (heart, lungs), abdomen, muscles, nervous system, spine/extremities;
Timeframe: Five years after infusion
10
The immunogenicity of AAV capsid protein: Collection of Peripheral Blood Mononuclear Cell (PBMC) and serum samples for vector shedding detection;