Ignite Creation Date:
2024-05-06 @ 8:13 PM
Last Modification Date:
2024-10-26 @ 3:22 PM
Study NCT ID:
NCT06293729
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-04-17
First Post:
2024-02-27
Brief Title:
Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients
Sponsor:
Suzhou Municipal Hospital
Organization:
Suzhou Municipal Hospital
Study Overview
Official Title:
A Clinical Study for the Safety and Efficacy of Intravenous Infusion of NGGT006 in Treatment of Refractory Hypercholesterolemia
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-04
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:
This is an early phase 1 open-label single-center dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT006 in patients with refractory Hypercholesterolemia diagnosed by gene testing for familial hypercholesterolemia NGGT006 uses adeno-associated virus AAV as a vector carrying a liver specific promoter and codon optimized human LDLR gene driving the expression of LDLR protein with normal function and promoting the clearance of low-density lipoprotein cholesterol LDL-C
Detailed Description:
Familial Hypercholesterolemia caused by common genetic mutations can be divided into heterozygous hypercholesterolemia HeFH Heterozygous Familial Hypercholesterolemia and homozygous hypercholesterolemia HoFH Homozygous Familial Hypercholesterolemia Refractory hypercholesterolemia was defined as an LDL-C level of 70 mgdL or greater or 100 mgdL or greater for patients with or without clinical ASCVD respectively A large proportion of patients with familial hypercholesterolemia belong to refractory hypercholesterolemia This is an early phase 1 open-label single-center dose-escalation pilot trial to evaluate the safety and efficacy of a single intravenous infusion of NGGT006 in patients with refractory Hypercholesterolemia diagnosed by gene testing for familial hypercholesterolemia 3-9 subjects will be enrolled and divided into 3 groups according to the principle of dose escalation respectively administered intravenous infusion of NGGT006 at low dose 75e12vgkg medium dose 15e13vgkg and high dose 3e13vgkg All subjects will undergo 52 weeks of treatment observation and further 260 weeks of long-term follow-up
Study Oversight
Has Oversight DMC:
None
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?:
None
Is an FDA AA801 Violation?:
None