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Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients

NCT06293729 · Status: NOT_YET_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2024-04-17

No results posted yet for this study

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).

Conditions

  • Refractory Hypercholesterolemia
  • Familial Hypercholesterolemia

Interventions

DRUG

NGGT006

Single intravenous infusion of NGGT006 at low dose (7.5e12vg/kg), medium dose (1.5e13vg/kg) and high dose (3e13vg/kg).

Sponsors & Collaborators

  • Suzhou Municipal Hospital

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-06-01
Primary Completion
2025-03-31
Completion
2029-03-01

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06293729 on ClinicalTrials.gov