NCT05860569 Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis
| NCT ID | NCT05860569 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | GeneCradle Inc |
| Condition | Hypertriglyceridemia, Familial |
| Study Type | INTERVENTIONAL |
| Enrollment | 7 participants |
| Start Date | 2024-09-20 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 7 participants in total. It began in 2024-09-20 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
Inclusion Criteria: * 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; * The patients fully understand and are able to comply with the requirements of the treatment and are willing to complete the trial as planned, including voluntary compliance with the trial procedures, acceptance of low-fat dietary requirements, and provide of biological samples. * Be able to understand the procedures and methods of the trial and voluntarily participate with the signature of the informed consent by the patient or his/her guardian. Exclusion Criteria: * 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; * Patient who is with any of the following clinical history of serious illness or existing serious illness: 1. unrelieved abdominal pain caused by acute onset of pancreatitis or by other causes； 2. disease history of malignancy or currently suffering from any malignant tumor; 3. autoimmune diseases; 4. disease history of epilepsy or mental illness (e.g. schizophrenia, depression, mania, anxiety, etc.); 5. heart diseases: cardiomyopathy and myocarditis; structural heart diseases; coronary heart disease (acute coronary syndrome, myocardial infarction); pericardial disease; severe arrhythmias (severe tachycardia requiring pacemakers, severe rapid arrhythmias, and other arrhythmias beyond the control of medications) ; New York Heart Association (NYHA) classification heart function grading ≥III or Left Ventricular Ejection Fraction (LVEF) ≤50%; 6. poorly controlled diabetes (fasting blood glucose ≥11.1mmol/L); 7. with systolic blood pressure (SBP) \> 150mmHg and/or diastolic blood pressure (DBP) \> 100mmHg after treatment with a stable dose (at least 4 weeks) of antihypertensive drugs; * The results of the laboratory examination at screening meet either of the following: 1. Aspartate transaminase (AST) or alanine transaminase (ALT) \> 2 × upper limit of normals (ULN); 2. Total bilirubin \> upper limit of normals (ULN); 3. Creatinine \> upper limit of normals (ULN); 4. Phosphatase kinase \> 2 × upper limit of normals (ULN); 5. Glomerular filtration rate estimate \< 50 mL/min (estimated by the Cockroft-Gault formula); 6. Positive hepatitis B surface antigen, positive hepatitis C antibody, positive HIV antibody or positive syphilis spiral antibody before or during screening; 7. A positive blood pregnancy test; * AAV5 neutralizing antibody levels above 1:100 * Person who has used a clinical trial drug within 1 month (30 days) prior to screening, or who plans to participate in other clinical trials during the trial period; * Blood loss/donation of more than 400 mL (except for female physiological blood loss) within 3 months (90 days) before screening, and receiving blood transfusion or using blood products; * Person who has undergone major surgery within 3 months (90 days) prior to screening, or who has undergone surgery that could significantly affect the course or safety evaluation of the trial drug; * Alcohol consumption was high in the first 3 months (90 days), i.e. the average alcohol intake was greater than 3 units/day (Male) or 2 units/days (female) (1 unit = 18ml alcohol, such as beer 360 ml with 5% alcohol, 12% wine 150ml, 40% liquor 45ml); or who cannot abstain from drinking during the trial; * Women who are pregnant, pregnant or breastfeeding, or all persons of reproductive age who are unable to take effective contraceptives until 3 months after the completion of the study; * Patients who have poor compliance or who may not be able to complete the test for other reasons, or whom the investigator considers inappropriate to participate in the trial.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05860569 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 60 Years, studying Hypertriglyceridemia, Familial. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05860569 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05860569 currently recruiting?
Yes, NCT05860569 is actively recruiting participants. Contact the research team at ind@bj-genecradle.com for enrollment information.
Where is the NCT05860569 trial being conducted?
This trial is being conducted at Beijing, China.
Who is sponsoring the NCT05860569 clinical trial?
NCT05860569 is sponsored by GeneCradle Inc. The trial plans to enroll 7 participants.