Actively Recruiting

Phase 1
Age: 2Years - 65Years
All Genders
NCT03961243

Lentiviral FIX Gene Therapy

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

10

Participants Needed

1

Research Sites

156 weeks

Total Duration

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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 IX into patients with hemophilia B, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.

CONDITIONS

Official Title

Lentiviral FIX Gene Therapy

Who Can Participate

Age: 2Years - 65Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Able to provide informed consent and comply with study requirements
  • Male participants aged 2 years or older with confirmed hemophilia B (factor IX level 2 IU/dL or 2% of normal or less)
  • Experience an average of at least 4 bleeding events per year requiring factor IX infusions (episodic or prophylactic)
  • No measurable factor IX inhibitor and no prior history of inhibitors to factor IX protein
  • 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 or bilirubin
  • History of inhibitor against factor IX
  • Active hepatitis B or C infection currently on antiviral therapy
  • HIV-1 or HIV-2 positive with CD4 count 200/mm3 or less (stable HIV+ with CD4 count above 200/mm3 and undetectable viral load allowed)
  • Any active infection or immunosuppressive disorder
  • Participation in a gene transfer trial within the last 6 months or any investigational drug trial within the last 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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