Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis
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ClinicalTrials.gov Identifier: NCT05860569 |
Recruitment Status :
Not yet recruiting
First Posted : May 16, 2023
Last Update Posted : February 23, 2024
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Tracking Information | |||||
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First Submitted Date ICMJE | April 23, 2023 | ||||
First Posted Date ICMJE | May 16, 2023 | ||||
Last Update Posted Date | February 23, 2024 | ||||
Estimated Study Start Date ICMJE | December 2024 | ||||
Estimated Primary Completion Date | December 2025 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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
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Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE | Same as current | ||||
Current Other Pre-specified Outcome Measures |
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Original Other Pre-specified Outcome Measures | Same as current | ||||
Descriptive Information | |||||
Brief Title ICMJE | Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis | ||||
Official Title ICMJE | 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 | ||||
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. | ||||
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. |
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Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 | ||||
Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Sequential Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Hypertriglyceridemia, Familial | ||||
Intervention ICMJE | Genetic: GC304
Self-complementary adeno-associated virus serotype 5 (AAV5) carrying a codon-optimized LPL coding sequence(coLPL) driven by a liver-specific promoter (LP)
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Study Arms ICMJE |
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Not yet recruiting | ||||
Estimated Enrollment ICMJE |
12 | ||||
Original Estimated Enrollment ICMJE | Same as current | ||||
Estimated Study Completion Date ICMJE | December 2028 | ||||
Estimated Primary Completion Date | December 2025 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years to 60 Years (Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE |
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Listed Location Countries ICMJE | China | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT05860569 | ||||
Other Study ID Numbers ICMJE | JL-GC304-01 | ||||
Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | GeneCradle Inc | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | GeneCradle Inc | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE | Not Provided | ||||
PRS Account | GeneCradle Inc | ||||
Verification Date | February 2024 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |