Tests a gene therapy for hemophilia B using the patient's own stem cells
Tests a gene therapy in people with hemophilia B.
Plain-language title generated from the registry record
Registry title: Lentiviral FIX Gene Therapy
Key facts
- Where
- Shenzhen, ChinaSee the site
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 65 years old?
Source: NCT03961243, who can take part
Where in China
- Shenzhen Geno-immune Medical InstituteShenzhen, ChinaNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Shenzhen Geno-Immune Medical Institute
- Trial code
- NCT03961243
- Record updated
- 2 November 2026
- Original description
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.Read full original descriptionShow less
Lentiviral FIX Gene Therapy for Hemophilia B
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.
Inclusion: 1. Able to provide informed consent and comply with requirements of the study. 2. Males ≥2 years with confirmed diagnosis of hemophilia B (endogenous factor IX ≤2 IU/dL or ≤2% of normal); A minimum average of 4 bleeding events per year requiring episodic treatment of factor IX infusions or prophylactic factor IX infusions; No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein; Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences.
Exclusion: 1. Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase; History of inhibitor against factor IX. 3. Evidence of active hepatitis B or C and currently on antiviral therapy. 4. Have serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 (subjects who are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible to enroll); Any evidence of active infection or any immunosuppressive disorder. 6. Participated in a gene transfer trial within the last 6 months or in a clinical trial with an investigational drug within the last 12 weeks; Unable or unwilling to comply with study assessments.