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Tests treatment safety and results for Hypertriglyceridemia, Familial
Official title Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis
ClinicalTrials.gov ID: NCT05860569
What this study is testing
What is GC304?
GC304 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for hypertriglyceridemia, familial.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 60
You may be able to join if
- Diagnosed as primary hypertriglyceridemia poorly managed by regular treatment and dietary control, with episode of acute pancreatitis twice or once...
- 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...
You likely can't join if
- 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:
- unrelieved abdominal pain caused by acute onset of pancreatitis or by other causes；
- disease history of malignancy or currently suffering from any malignant tumor;
See the full eligibility 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...
- 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.
- 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:
- unrelieved abdominal pain caused by acute onset of pancreatitis or by other causes；
- disease history of malignancy or currently suffering from any malignant tumor;
- autoimmune diseases;
- disease history of epilepsy or mental illness (e.g. schizophrenia, depression, mania, anxiety, etc.);
- heart diseases: cardiomyopathy and myocarditis; structural heart diseases; coronary heart disease (acute coronary syndrome, myocardial infarction); pericardial disease; severe arrhythmias (severe tachycardia requiring...
- poorly controlled diabetes (fasting blood glucose ≥11.1mmol/L);
- 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:
- Aspartate transaminase (AST) or alanine transaminase (ALT) \> 2 × upper limit of normals (ULN);
- Total bilirubin \> upper limit of normals (ULN);
- Creatinine \> upper limit of normals (ULN);
- Phosphatase kinase \> 2 × upper limit of normals (ULN);
- Glomerular filtration rate estimate \< 50 mL/min (estimated by the Cockroft-Gault formula);
- Positive hepatitis B surface antigen, positive hepatitis C antibody, positive HIV antibody or positive syphilis spiral antibody before or during screening;
- 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%...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, Beijing Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, Beijing Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.