NGGT006 Gene Therapy for Homozygous 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
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.
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) ≥2 × upper limit of normal (ULN) and/or aspartate aminotransferase (AST) ≥2 × 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
Treatments tested in this trial
- NGGT006
Treatment groups
12 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov