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 range18 Years – 65 Years

SexMALE

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;
✓. Subject has no history of FVIII inhibitors.
✓. Subjects agree to use a reliable barrier contraceptive method from the date of signing the informed consent

Exclusion criteria

What they're measuring

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

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

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

Total FⅧ Antibody Levels: Total FⅧ antibody levels will be measured to determine the immunogenicity of FⅧ expression protein;

Timeframe: Five years after infusion

FVIII inhibitor: Factor Ⅷ inhibitor will be measured to determine the immunogenicity of FⅧ expression protein;

Timeframe: 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.

Timeframe: Five years after infusion

The Change of Laboratory Values: Change in serum chemistry values including liver function tests, hematology, and urinalysis;

Trial details

NCT IDNCT07548411

SponsorGritgen Therapeutics Co., Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-12-31

View on ClinicalTrials.gov More Severe Hemophilia A trials

Plain-language summary

Who can participate

Age range18 Years – 65 Years

SexMALE

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;
✓. Subject has no history of FVIII inhibitors.
✓. Subjects agree to use a reliable barrier contraceptive method from the date of signing the informed consent

Exclusion criteria

What they're measuring

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

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

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

Total FⅧ Antibody Levels: Total FⅧ antibody levels will be measured to determine the immunogenicity of FⅧ expression protein;

Timeframe: Five years after infusion

FVIII inhibitor: Factor Ⅷ inhibitor will be measured to determine the immunogenicity of FⅧ expression protein;

Timeframe: 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.

Timeframe: Five years after infusion

The Change of Laboratory Values: Change in serum chemistry values including liver function tests, hematology, and urinalysis;