Hypertriglyceridemia, FamilialPhase 1NCT05860569
Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis
A Phase 1 study testing GC304 in people with Hypertriglyceridemia, Familial, run by GeneCradle Inc at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Hypertriglyceridemia, Familial
- What is being tested?
- GC304
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteChina
- Study dates
- 20 Sep 2024to Dec 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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;
Show all 26Show fewer
- 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 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%;
- 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% 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College
Beijing, Beijing Municipality
Qi Liu, 86-15628951937, liuq@bj-genecradle.com
Shuyang Zhang, MD
About this study
Official title: A Multi-center, Open Label, Multi-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Tolerance of Gene Therapy Drug GC304 in the Treatment of Primary Hypertriglyceridemia Patients With History of Acute Pancreatitis
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.
Read the detailed description
The purpose of this trial is to evaluate safety and tolerance of gene therapy drug GC304 in primary hypertriglyceridemic patients who have loss of function mutations in GPIHBP1 or LPL genes, with previous onset of acute pancreatitis.
Open-label, dose-escalation clinical trial of GC304 will be conducted in China. GC304 will be administrated intravenously. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up on various time points.
- Conditions
- Hypertriglyceridemia, Familial
- Being tested
- GC304
- Medical subject headings
- Hyperlipoproteinemia Type IV
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GC304Genetic
Self-complementary adeno-associated virus serotype 5 (AAV5) carrying a codon-optimized LPL coding sequence(coLPL) driven by a liver-specific promoter (LP)
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 3.0x10^12 vg/kg of GC304 delivered one-time intravenously (n=3) Receives: Genetic: GC304 |
| Cohort 2 | Experimental | 1.0x10^13 vg/ kg of GC304 delivered one-time i intravenously (n=3) Receives: Genetic: GC304 |
| Cohort 3 | Experimental | 3.0x10^13 vg/ kg of GC304 delivered one-time i intravenously (n=3) Receives: Genetic: GC304 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])
Frequency of treatment-related adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
Measured: 12 weeks
The incidence of dose limited tolerance (DLT)
Measured: 12 weeks
Secondary outcomes (5)
Changes of plasma triglyceride levels from baseline
Measured: 12 weeks
The proportion of patients who stop using lipid-lowering medications;
Measured: 12 weeks
Copy numbers of viral vector DNA [Shedding of viral vectors];
Measured: 12 weeks
Titers of antibody against viral vector
Measured: 12 weeks
Titers of antibody against LPL (lipoprotein lipase) protein
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2023
- Study start20 Sep 2024
- Primary completionDec 2026 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypertriglyceridemia, FamilialStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 7 participants (estimated)
Sponsor and investigators
- Lead sponsor
- GeneCradle Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05860569
- Sponsor's protocol ID
- JL-GC304-01
- Status last verified
- Jul 2025
- First submitted
- 23 Apr 2023
- Last updated
- 3 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open Label, Multi-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Tolerance of Gene Therapy Drug GC304 in the Treatment of Primary Hypertriglyceridemia Patients With History of Acute Pancreatitis
- ClinicalTrials.gov ID
- NCT05860569
- Acronym / sponsor ID
- JL-GC304-01
- Conditions
- Hypertriglyceridemia, Familial
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 7 participants (estimated)
- Lead sponsor
- GeneCradle IncIndustry (pharma, biotech, devices)
- Study start
- 20 Sep 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 16 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The purpose of this trial is to evaluate safety and tolerance of gene therapy drug GC304 in primary hypertriglyceridemic patients who have loss of function mutations in GPIHBP1 or LPL genes, with previous onset of acute pancreatitis.
Open-label, dose-escalation clinical trial of GC304 will be conducted in China. GC304 will be administrated intravenously. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up on various time points.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Genetic: GC3043.0x10^12 vg/kg of GC304 delivered one-time intravenously (n=3) |
| Cohort 2 | Experimental | Genetic: GC3041.0x10^13 vg/ kg of GC304 delivered one-time i intravenously (n=3) |
| Cohort 3 | Experimental | Genetic: GC3043.0x10^13 vg/ kg of GC304 delivered one-time i intravenously (n=3) |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (26 listed)
- 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:
- The results of the laboratory examination at screening meet either of the following:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GC304Self-complementary adeno-associated virus serotype 5 (AAV5) carrying a codon-optimized LPL coding sequence(coLPL) driven by a liver-specific promoter (LP) | Genetic | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])
Time frame: 12 weeks
Frequency of treatment-related adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
The incidence of dose limited tolerance (DLT)
Time frame: 12 weeks
5 secondary outcomes
Changes of plasma triglyceride levels from baseline
Time frame: 12 weeks
The proportion of patients who stop using lipid-lowering medications;
Time frame: 12 weeks
Copy numbers of viral vector DNA [Shedding of viral vectors];
Time frame: 12 weeks
Titers of antibody against viral vector
Time frame: 12 weeks
Titers of antibody against LPL (lipoprotein lipase) protein
Time frame: 12 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2025.
- Individual participant dataSharing planned: No
Related trials for Hypertriglyceridemia, Familial
RecruitingPhase 1
Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia
Hypercholesterolemia, Hypertriglyceridemia
RecruitingPhase 1
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
Cardiovascular, Metabolic Disease
RecruitingEarly Phase 1
CS-121 APOC3 Base Editing in FCS
Familial Chylomicronemia Syndrome (FCS)
Active, not recruitingPhase 1/2
Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects
Hypertriglyceridemia, Familial Chylomicronemia Syndrome
Active, not recruitingObservational study
Stockholm hyperTRIglyceridemia REGister (STRIREG) Study
Familial Hypertriglyceridemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GC304 in people with Hypertriglyceridemia, Familial, run by GeneCradle Inc at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact GeneCradle, Inc. China (86-13501380583, ind@bj-genecradle.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]) (time frame: 12 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jul 2025.