At a glance
ClinicalIndex Comparison Record- ✓Age 12-55 years (first patient ≥18 years)
- ✓Confirmed homozygous familial hypercholesterolemia with two mutant LDLR alleles
- ✓Untreated LDL-C ≥10 mmol/L (386 mg/dL) or treated LDL-C ≥7 mmol/L (270 mg/dL) with cutaneous or tendon xanthoma before age 18
- ✓Stable lipid-lowering medication for ≥30 days (or ≥60 days for alirocumab/evolocumab)
- ✕Positive for hepatitis B, hepatitis C, HIV, or syphilis
- ✕ALT ≥2× ULN and/or AST ≥2× ULN
- ✕Baseline blood pressure ≥160/100 mmHg
- ✕Uncontrolled myocardial infarction, heart failure, or surgery plan within 1 year
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
NCT06125847Early Ph 1RecruitingUpdate OverdueUpdated 18mo ago · Completion was 19mo agoA Clinical Study for the Safety and Efficacy of Intravenous Infusion of NGGT006 in Treatment of Homozygous Familial Hypercholesterolemia With LDLR Mutations
In Brief
A Early Phase 1 clinical trial evaluating NGGT006 for Homozygous Familial Hypercholesterolemia. Currently recruiting, targeting 12 participants across 1 site.
Signals
Detailed 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).
Study Details
Timeline
Interventions
Single intravenous infusion of NGGT006 at dose 1 (7.5e12vg/kg), dose 2 (1.5e13vg/kg) , dose 3 (3e13vg/kg) and dose 4 (4e13vg/kg).