NCT06125847 NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia
| NCT ID | NCT06125847 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | First Affiliated Hospital Xi'an Jiaotong University |
| Condition | Homozygous Familial Hypercholesterolemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 21 participants |
| Start Date | 2023-10-29 |
| Primary Completion | 2027-03-30 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 21 participants in total. It began in 2023-10-29 with a primary completion date of 2027-03-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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).
Eligibility Criteria
Inclusion Criteria: * 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 Criteria: * 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; * Diabetes diagnosed within 3 months or with poor control (HbA1c \>9%); * Acute or chronic kidney failure; * 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; * Had contraindications to glucocorticoid (e.g., epilepsy, severe schizophrenia, active peptic ulcer); * Life expectancy less than 1 year; * With malignant tumors; * Liver fibrosis or liver cancer; * Previous gene therapy treatment; * Hypersensitivity to any excipient of the AAV product (including trehalose) or cortisone or immunosuppressants (sirolimus, rituximab, tacrolimus); * Participation in any other clinical trial within 3 months; * History of stent implantation within 1 month or myocardial infarction within 3 months; * Breastfeeding females; * Any other condition that may not be appropriate for the study in the opinion of the Investigator.
Contact & Investigator
Zuyi Yuan, M.D.
STUDY CHAIR
First Affiliated Hospital of Xian Jiaotong University
Frequently Asked Questions
Who can join the NCT06125847 clinical trial?
This trial is open to participants of all sexes, aged 12 Years or older, up to 55 Years, studying Homozygous Familial Hypercholesterolemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06125847 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06125847 currently recruiting?
Yes, NCT06125847 is actively recruiting participants. Contact the research team at zhengtao900305@163.com for enrollment information.
Where is the NCT06125847 trial being conducted?
This trial is being conducted at Xi'an, China.
Who is sponsoring the NCT06125847 clinical trial?
NCT06125847 is sponsored by First Affiliated Hospital Xi'an Jiaotong University. The principal investigator is Zuyi Yuan, M.D. at First Affiliated Hospital of Xian Jiaotong University. The trial plans to enroll 21 participants.