Lentiviral FIX Gene Therapy
ConditionHemophilia B
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age2-65
SponsorShenzhen Geno-Immune Medical Institute
About this trial
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.
Eligibility criteria
Qualifiers
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).
Disqualifiers
1. Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase.
Trial design
Treatments tested in this trial
- YUVA-GT-F901
Treatment groups
10 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov