Lentiviral FIX Gene Therapy
ConditionHemophilia B
Trial statusNot yet recruiting
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
This trial does not accept healthy volunteersQualifiers
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
Design model
Single group
Treatments tested in this trial
YUVA-GT-F901
Biological/VaccineLentiviral factor IX gene modified autologous hematopoietic and mesenchymal stem cells
Treatment groups
10 Participants
are divided into 1 treatment groupGroup A: YUVA-GT-F901Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Number of participants experiencing drug-related adverse events
As assessed by physical exam, vital signs, standard clinical labs, and Bethesda assay for FIX inhibitor
Time frame
a yearSecondary outcomes
1
Changes from baseline in circulating FIX activity (IU/dL or % normal)
Time frame
a yearSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: