Ignite Creation Date:
2024-05-06 @ 7:46 PM
Last Modification Date:
2024-10-26 @ 3:13 PM
Study NCT ID:
NCT06125847
Status:
RECRUITING
Last Update Posted:
2024-04-02
First Post:
2023-10-30
Brief Title:
NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia
Sponsor:
First Affiliated Hospital Xian Jiaotong University
Organization:
First Affiliated Hospital Xian Jiaotong University
Study Overview
Official Title:
A Clinical Study for the Safety and Efficacy of Intravenous Infusion of NGGT006 in Treatment of Homozygous Familial Hypercholesterolemia With LDLR Mutations
Status:
RECRUITING
Status Verified Date:
2023-11
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 homozygous familial hypercholesterolemia HoFH patients with LDLR mutations NGGT006 is an adeno-associated viral AAV vector carrying 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:
Homozygous familial hypercholesterolemia HoFH is a rare inherited disorder of lipoprotein metabolism characterized by extreme elevations in low-density lipoprotein cholesterol LDL-C and leading to early onset of severe coronary artery disease 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 HoFH patients with LDLR mutations NGGT006 is an adeno-associated viral AAV vector carrying 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 3-12 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