Actively Recruiting

Early Phase 1
Age: 18Years +
MALE
NCT05454774

A Study of FVIII Gene Therapy for Hemophilia A

Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-02-21

8

Participants Needed

1

Research Sites

269 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This is a single-arm, open-label, clinical study to evaluate the safety, tolerability of BBM 002 injection in Hemophilia A subjects with residual factor VIII (FVIII) levels ≤2 International unit per deciliter (IU/dl) . BBM 002 injection is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII (hFVIII) transgene and raises circulating levels of endogenous FVIII.

CONDITIONS

Official Title

A Study of FVIII Gene Therapy for Hemophilia A

Who Can Participate

Age: 18Years +
MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Male aged 18 years or older
  • Diagnosed with Hemophilia A with endogenous factor VIII activity levels of 2 IU/dL or less
  • At least 150 prior exposure days to recombinant and/or plasma-derived factor VIII products
  • Documented bleeding events and/or factor VIII infusions in the past 12 weeks
  • No history of hypersensitivity or anaphylaxis related to factor VIII or intravenous immunoglobulin
  • No factor VIII inhibitor detected in two consecutive tests or prior medical history
  • Agree to use reliable barrier contraception from informed consent signing to 52 weeks after BBM 002 infusion
  • Good compliance and willingness to participate in gene therapy clinical trials
Not Eligible

You will not qualify if you...

  • Positive for hepatitis B surface antigen or hepatitis B virus DNA
  • Positive for hepatitis C virus antibody or RNA
  • Currently receiving antiviral therapy for hepatitis B or C
  • Other coagulation disorders besides Hemophilia A
  • Use of systemic immunosuppressants other than glucocorticoids within 30 days before enrollment
  • Vaccination within 30 days prior to screening
  • Presence of significant liver disease or abnormalities
  • Planned major surgery during the 52-week study period
  • Previous participation in gene therapy trials or investigational drug studies within 5 half-lives
  • Alcohol or drug dependence or inability to abstain during the study
  • Any other major clinically significant disease or condition deemed unsuitable by investigators

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

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, China, 300020

Actively Recruiting

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Research Team

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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