Actively Recruiting

Phase Not Applicable
Age: 18Years +
MALE
NCT04728841

Gene Therapy for Chinese Hemophilia A

Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-08-01

12

Participants Needed

1

Research Sites

386 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

Gene Therapy for Chinese Hemophilia A

Who Can Participate

Age: 18Years +
MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Be able to understand the study purpose and risks and provide informed consent
  • Male subjects aged 18 years or older
  • Diagnosed with hemophilia A with endogenous FVIII activity levels at or below 1 IU/dL at screening
  • No history of hypersensitivity or anaphylaxis to FVIII products
  • No measurable FVIII inhibitor as confirmed by laboratory tests or documented history
  • Acceptable laboratory values: Hemoglobin  11 g/dL; Platelets  100 x 10^9/L; AST, ALT, alkaline phosphatase  1.25 times upper limit of normal; Bilirubin  1.25 times upper limit of normal; Creatinine  2 mg/dL
  • Agree to use reliable barrier contraception until 52 weeks after infusion and until three consecutive semen samples test negative for vector sequences
Not Eligible

You will not qualify if you...

  • Positive for Hepatitis B or C with clinical significance
  • Currently receiving antiviral therapy for hepatitis B or C
  • History of chronic infections or other chronic diseases that pose a risk
  • Participation in gene therapy trials within the last 52 weeks or investigational drug trials within 30 days
  • Conditions that prevent tolerance of prednisone or prednisolone treatments
  • History of arterial or venous thromboembolic events
  • Known inherited or acquired thrombophilia or conditions increasing thromboembolism risk
  • Major surgery planned within one year after GS001 infusion
  • Hypersensitivity to study vector
  • Major diseases or conditions deemed unsuitable by the investigator
  • Unable or unwilling to comply with study visits and assessments
  • Other bleeding disorders unrelated to hemophilia A

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, Tianjin Municipality, China, 300020

Actively Recruiting

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Research Team

W

Wei Liu, MD

CONTACT

L

Lei Zhang, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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Gene Therapy for Chinese Hemophilia A | DecenTrialz