Health TrialsbyHealthPanel

NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

ConditionHomozygous Familial Hypercholesterolemia
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age12-55
SponsorFirst Affiliated Hospital Xi'an Jiaotong University

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Voluntarily sign informed consent form;

Male or female, 12 ≤ age ≤ 55 years (first patient≥ 18 years), diagnosed as homozygous familial hypercholesterolemia with genetic confirmation of two mutant alleles of the LDL receptor (LDLR) gene;

AAV8 neutralizing antibodies can be negative or reduced to negative levels through methods such as plasma exchange or IgG-degrading enzymes.

Untreated LDL-C ≥10 mmol/L (386mg/ dL) or treated LDL-C ≥7 mmol/L (270mg/ dL) together with cutaneous or tendon xanthoma before age 18 years;

Disqualifiers

Positive for hepatitis B surface antigen, hepatitis C, human immunodeficiency virus (HIV) or syphilis test;

Clinically significant abnormalities in liver function test: alanine aminotransferase (ALT) >3 × upper limit of normal (ULN) and/or aspartate aminotransferase (AST) >3 × ULN;

Baseline blood pressure >160/100 mmHg (1 repeat measurement is allowed);

Uncontrolled myocardial infarction or heart failure, or had surgery plan within 1 year;

Trial design

Design model

Sequential

Treatments tested in this trial

  • NGGT006

    Genetic

    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).

Treatment groups

21 Participants
are divided into 1 treatment group
Group A: NGGT006Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of treatment-related adverse events (AE) and serious adverse events (SAE)

Incidence of AE and SAE, as assessed by physical examinations, clinical laboratory parameters and adverse event reporting

Time frame
52 weeks
2

Absolute change and percent change in LDL-C

Change in LDL-C concentration from baseline to week 52

Time frame
52 weeks

Secondary outcomes

1

Absolute change and percent change in apoB

Change in lipid concentrations from baseline to week 52

Time frame
52 weeks
2

Absolute change and percent change in TC

Change in lipid concentrations from baseline to week 52

Time frame
52 weeks
3

Absolute change and percent change in HDL-C

Change in lipid concentrations from baseline to week 52

Time frame
52 weeks
4

Absolute change and percent change in TG

Change in lipid concentrations from baseline to week 52

Time frame
52 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

First Affiliated Hospital Xi'an Jiaotong University

Lead sponsor

Source ClinicalTrials.gov
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