Ignite Creation Date:
2025-12-24 @ 11:14 PM
Ignite Modification Date:
2025-12-25 @ 8:49 PM
Study NCT ID:
NCT05860569
Status:
RECRUITING
Last Update Posted:
2025-07-03
First Post:
2023-04-23
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis
Sponsor:
GeneCradle Inc
Organization:
Study Overview
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
Status:
RECRUITING
Status Verified Date:
2025-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
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 Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: