Actively Recruiting
Safety and Tolerance Study of Gene Therapy Drug GC304 for Adults with Primary Hypertriglyceridemia and History of Acute Pancreatitis
Led by GeneCradle Inc · Updated on 2025-07-03
7
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerance of the gene therapy drug GC304 in patients with primary hypertriglyceridemia who have experienced acute pancreatitis. This trial focuses on patients with specific gene mutations GPIHBP1 or LPL and aims to understand how well the intravenous gene therapy is tolerated and its safety profile. The study is an open-label, dose-escalation trial conducted in China and sponsored by GeneCradle Inc. GC304, a genetic therapy using a modified virus to deliver a functional LPL gene, is given as a one-time intravenous infusion in three different dose groups. The doses are 3.0x1012, 1.0x1013, and 3.0x1013 vector genomes per kilogram, with three patients in each group. The trial includes a short-term safety evaluation for 52 weeks, followed by an optional long-term follow-up period of up to five years. Participants will be tested at the start of the study and monitored at various points throughout the trial. Researchers will assess safety by tracking treatment-related adverse events and dose tolerance over 12 weeks. They will also measure changes in blood triglyceride levels, antibody responses, and viral vector DNA presence. The study continues with long-term follow-up to monitor ongoing safety and effects up to five years after treatment.
CONDITIONS
Brief Title
Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with primary hypertriglyceridemia poorly controlled by treatment and diet, with at least one episode of acute pancreatitis or severe acute pancreatitis within 5 years
- Fasting plasma triglycerides above 5.65 mmol/L with low dietary fat intake before testing
- Confirmed homozygous or heterozygous mutations in GPIHBP1 or LPL genes
- Patients of reproductive age must use effective contraceptives during screening and up to 6 months after the trial
- Ability and willingness to comply with trial procedures, low-fat diet, and provide biological samples
- Able to understand trial procedures and provide informed consent
You will not qualify if you...
- Allergy to any trial drug components or immunosuppressants
- Active bacterial, fungal, viral, or other infections
- Intolerance to immunosuppressive drugs or steroids
- Serious or uncontrolled illnesses including unresolved abdominal pain, malignancies, autoimmune diseases, epilepsy or mental illnesses, severe heart diseases, poorly controlled diabetes, and high blood pressure despite treatment
- Abnormal lab results exceeding specified limits for liver, kidney, or blood function
- Positive tests for hepatitis B, hepatitis C, HIV, syphilis, or pregnancy
- High levels of AAV5 neutralizing antibodies
- Participation in other clinical trials within 30 days or planned during this trial
- Blood loss over 400 mL within 90 days or recent blood transfusion
- Major surgery within 90 days prior to screening
- High alcohol consumption in the past 3 months or inability to abstain during the trial
- Pregnant or breastfeeding women or those unable to use effective contraception until 3 months post-study
- Poor compliance or judged unsuitable by investigators
Research Team
G
GeneCradle, Inc. China
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