Gene Therapy Infusion Tested in Homozygous Familial Hypercholesterolemia Patients
Tests NGGT006, a gene therapy infusion, in people with homozygous familial hypercholesterolemia.
Plain-language title generated from the registry record
Registry title: NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia
Key facts
- Where
- Xi'an, ChinaSee the site
- Phase
- Early phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 12 and 55 years old?
Source: NCT06125847, who can take part
Where in China
- First Affiliated Hospital of Xian Jiaotong UniversityXi'an, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- First Affiliated Hospital Xi'an Jiaotong University
- Trial code
- NCT06125847
- Record updated
- 3 September 2026
- Original description
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).Read full original descriptionShow less
A Clinical Study for the Safety and Efficacy of Intravenous Infusion of NGGT006 in Treatment of Homozygous Familial Hypercholesterolemia With LDLR Mutations
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).
Inclusion: 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; Had been on stable medication for ≥30 days if receiving lipid-lowering therapy (or ≥60 days if receiving alirocumab or evolocumab) prior to screening and not scheduled for addition of new drugs or dose adjustments during the study; Agreed to follow a low-fat diet and comply with all study procedures, including dietary requirements, study visits, fasting blood draws, and the study treatment regimen; Agreed to maintain a similar exercise volume and intensity to baseline during the study period; Agreed to maintain good lifestyle habits; No history of alcohol abuse or alcohol dependence (diagnosed as F10 in ICD-10 code); No sexual activity for 14 days prior to administration and negative serum pregnancy test in female participants; Participants of childbearing potential agreed to use highly effective contraception for at least 365 days from administration of NGGT006; No plan of stent implantation within 3 months.
Exclusion: Previous gene therapy treatment; Positive for hepatitis B surface antigen, hepatitis C, human immunodeficiency virus (HIV) or syphilis test; Uncontrolled myocardial infarction or heart failure, or had surgery plan within 1 year; Acute or chronic kidney failure; Had contraindications to glucocorticoid (e.g., epilepsy, severe schizophrenia, active peptic ulcer); With malignant tumors; Liver fibrosis or liver cancer; Hypersensitivity to any excipient of the AAV product (including trehalose) or cortisone or immunosuppressants (sirolimus, rituximab, tacrolimus); History of stent implantation within 1 month or myocardial infarction within 3 months; Any other condition that may not be appropriate for the study in the opinion of the Investigator; Clinically significant abnormalities in liver function test: alanine aminotransferase (ALT) >3 × upper limit of normal (ULN) and/or aspartate aminotransferase (AST) >3 × ULN; Diabetes diagnosed within 3 months or with poor control (HbA1c >9%); Hemoglobin (Hb) <120g/L (male), Hb <110 (female); Platelet test results deemed clinically significantly abnormal by the investigator; History or laboratory tests suggestive of thrombosis; Baseline blood pressure >160/100 mmHg (1 repeat measurement is allowed); Life expectancy less than 1 year; Participation in any other clinical trial within 3 months; Breastfeeding females.