About this trial
The study will evaluate safety and tolerance of intravenous delivery of GC304 gene therapy drug as a treatment of primary hypertriglyceridemic patients with previous onset of acute pancreatitis.
Eligibility criteria
Qualifiers
Diagnosed as primary hypertriglyceridemia poorly managed by regular treatment and dietary control, with episode of acute pancreatitis twice or once of severe acute pancreatitis within 5 years;
Fasting plasma triglycerides (TG) levels above 5.65 mmol/L (intake of dietary fat <30 g within 24 hours before blood taken);
Homozygous or heterozygous mutations in GPIHBP1 or LPL genes by genetic screening;
The patients within reproductive age take effective contraceptive measures voluntarily entering screening stage until 6 months after the trial;
Disqualifiers
Patient who is known to be allergic to any ingredient of a trial drug (including immunosuppressants) or has any disease prohibited from the treatment;
Patient who is having active bacteria, fungi, viruses or other infections;
Patient who is intolerant of immunosuppressive drugs or steroids;
unrelieved abdominal pain caused by acute onset of pancreatitis or by other causes;
Trial design
Treatments tested in this trial
- GC304