NCT03961243Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Lentiviral FIX Gene Therapy for Hemophilia B
Source: ClinicalTrials.gov · Last updated 2 Nov 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- First submitted
- 22 May 2019
- Start date
- 1 Jun 2027
- Primary completion date
- 31 May 2028
- Lead sponsor
- Shenzhen Geno-Immune Medical Institute
- Diseases
- Haemophilia
- NCT number
- NCT03961243
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 10
- From 1 Jun 2027 to the primary completion date, 31 May 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Lab values
- 1\. Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase.
- 2\. 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).
- 5\. 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.
- 7\. Unable or unwilling to comply with study assessments.
Who the study is looking for
- 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).
- 3\. A minimum average of 4 bleeding events per year requiring episodic treatment of factor IX infusions or prophylactic factor IX infusions.
- 4\. No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein.
- 5\. Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences.
Age in the registry: 2 years to 65 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Shenzhen Geno-immune Medical Institute Coordinator of this site? Confirm its status | Shenzhen | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.