Lentiviral FVIII Gene Therapy
ConditionHemophilia A
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 VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.
Eligibility criteria
Qualifiers
Able to provide informed consent and comply with requirements of the study.
Males ≥2 years with confirmed diagnosis of hemophilia A (endogenous factor VIII ≤2 IU/dL or ≤2% of normal).
A minimum average of 4 bleeding events per year requiring episodic treatment of factor VIII infusions or prophylactic factor VIII infusions.
No measurable factor VIII inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor VIII protein.
Disqualifiers
Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase.
History of inhibitor against factor VIII.
Evidence of active hepatitis B or C and currently on antiviral therapy.
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).
Trial design
Treatments tested in this trial
- YUVA-GT-F801
Treatment groups
10 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov