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New treatment option for Hemophilia A
Official title Gene Therapy for Chinese Hemophilia A
ClinicalTrials.gov ID: NCT04728841
What this study is testing
What is Injection of GS001?
Injection of GS001 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for hemophilia a.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with \<1 IU/dl residual FVIII levels.
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older, men only
You may be able to join if
- Be able to understand the purpose and risks of the study and provide informed consent according to national and local privacy laws;
- Male people and ≥ 18 years of age;
- Have hemophilia A with ≤1 IU/dL (≤1%) endogenous FVIII activity levels at the time of screening. If the screening result is \>1% due to previous...
- No history of hypersensitivity or anaphylaxis associated with FVIII product administration;
- Have no measurable FVII inhibitor as assessed by laboratory two times that were at least one week apart; or documented no prior history of FVIII...
You likely can't join if
- Have Hepatitis B, hepatitis C or HBsAg, HCVAb, HBV-DNA, HCV-RNA are positive and have clinical significance. Both natural clearers and those who have...
- Currently Receiving antiviral therapy for hepatitis B and C;
- Have history of chronic infections or other chronic diseases that may pose a risk to the study participation;
- Have participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the...
- The subject has any concurrent diseases that cannot tolerate treatments of prednisone or prednisolone as judged by the investigator;
- History of arterial or venous thromboembolic events (e.g., deep vein thrombosis, non-hemorrhagic stroke, pulmonary embolism, myocardial infarction...
See the full eligibility criteria
- Be able to understand the purpose and risks of the study and provide informed consent according to national and local privacy laws;
- Male people and ≥ 18 years of age;
- Have hemophilia A with ≤1 IU/dL (≤1%) endogenous FVIII activity levels at the time of screening. If the screening result is \>1% due to previous treatment with FVIII product, then it may be confirmed by documented...
- No history of hypersensitivity or anaphylaxis associated with FVIII product administration;
- Have no measurable FVII inhibitor as assessed by laboratory two times that were at least one week apart; or documented no prior history of FVIII inhibitor after 150 EDs and no clinical signs or symptoms of decreased...
- Have acceptable laboratory values sampled at screening and repeated prior to Day 0; A. Hemoglobin ≥ 11 g/dL; B. Platelets ≥ 100 x 10\^9/L; C. AST, ALT, alkaline phosphatase ≤ 1.25 upper limit of normal (ULN); D...
- Agree to use reliable barrier contraception until the end of the 52 weeks observation period, and three consecutive semen samples are negative for vector sequences after GS001 infusion.
- Have Hepatitis B, hepatitis C or HBsAg, HCVAb, HBV-DNA, HCV-RNA are positive and have clinical significance. Both natural clearers and those who have cleared HCV on antiviral therapy are deemed eligible;
- Currently Receiving antiviral therapy for hepatitis B and C;
- Have history of chronic infections or other chronic diseases that may pose a risk to the study participation;
- Have participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the past 30 days;
- The subject has any concurrent diseases that cannot tolerate treatments of prednisone or prednisolone as judged by the investigator;
- History of arterial or venous thromboembolic events (e.g., deep vein thrombosis, non-hemorrhagic stroke, pulmonary embolism, myocardial infarction, arterial embolism);
- Known inherited or acquired thrombophilia, including conditions associated with increased risk of thromboembolism, such as atrial fibrillation;
- Major surgery planned in 1 year period following the infusion with GS001;
- Hypersensitivity to the study vector;
- Have clinically major diseases or any other unspecified conditions that, in the opinion of the Investigator, makes the subject unsuitable for participating in the study;
- Patients who are unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol;
- Evidence of other bleeding disorders not associated with hemophilia A.
The study team makes the final eligibility decision.
Where it's taking place
- Tianjin, Tianjin Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Tianjin, Tianjin Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.