Actively Recruiting

Phase 1
Age: 2Years - 65Years
All Genders
NCT03217032

Lentiviral FVIII Gene Therapy

Led by Shenzhen Geno-Immune Medical Institute · Updated on 2026-04-24

10

Participants Needed

1

Research Sites

156 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.

CONDITIONS

Official Title

Lentiviral FVIII Gene Therapy

Who Can Participate

Age: 2Years - 65Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Able to provide informed consent and follow study requirements
  • Male patients aged 2 years or older with confirmed hemophilia A (factor VIII 62 IU/dL or 62% of normal)
  • Have an average of at least 4 bleeding events per year needing factor VIII infusions
  • No measurable factor VIII inhibitor and no prior history of inhibitors
  • Agree to use reliable barrier contraception until 3 consecutive tests show no vector sequences
Not Eligible

You will not qualify if you...

  • Significant liver dysfunction indicated by abnormal liver enzymes
  • History of inhibitor against factor VIII
  • Active hepatitis B or C infection currently on antiviral treatment
  • HIV positive with CD4 count 60/mm3; stable HIV patients with CD4 count >200/mm3 and undetectable viral load are eligible
  • Any active infection or immunosuppressive disorder
  • Participation in another gene transfer trial within 6 months or clinical trial with investigational drug within 12 weeks
  • Unable or unwilling to comply with study assessments

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

Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, China, 518000

Actively Recruiting

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Research Team

L

Lung-Ji Chang, PhD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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Lentiviral FVIII Gene Therapy | DecenTrialz