Actively Recruiting
NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia
Led by First Affiliated Hospital Xi'an Jiaotong University · Updated on 2024-12-09
12
Participants Needed
1
Research Sites
261 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Voluntarily sign informed consent form
- Male or female, aged 12 to 55 years (first patient ≥ 18 years)
- Diagnosed with homozygous familial hypercholesterolemia confirmed by two mutant LDLR alleles
- Negative or reduced to negative AAV8 neutralizing antibodies
- Untreated LDL-C ≥ 10 mmol/L or treated LDL-C ≥ 7 mmol/L with cutaneous or tendon xanthoma before age 18
- Stable medication for ≥ 30 days (or ≥ 60 days if on alirocumab or evolocumab) before screening
- Agree to follow a low-fat diet and study procedures
- Agree to maintain similar exercise volume and intensity during the study
- Agree to maintain good lifestyle habits
- No history of alcohol abuse or dependence
- No sexual activity for 14 days before administration and negative pregnancy test for females
- Use highly effective contraception for at least 365 days after NGGT006 administration if of childbearing potential
- No plan for stent implantation within 3 months
You will not qualify if you...
- Positive test for hepatitis B surface antigen, hepatitis C, HIV, or syphilis
- Significant liver function abnormalities (ALT or AST ≥ 2 times upper limit of normal)
- Blood pressure ≥ 160/100 mmHg (one repeat allowed)
- Uncontrolled myocardial infarction or heart failure or planned surgery within 1 year
- Diabetes diagnosed within 3 months or poorly controlled (HbA1c ≥ 9%)
- Acute or chronic kidney failure
- Low hemoglobin (Hb < 120 g/L for males, < 110 g/L for females)
- Abnormal platelet counts or morphology
- History or signs of thrombosis
- Contraindications to glucocorticoids (e.g., epilepsy, severe schizophrenia, active peptic ulcer)
- Life expectancy less than 1 year
- Malignant tumors
- Liver fibrosis or liver cancer
- Previous gene therapy treatment
- Hypersensitivity to AAV, cortisone, or immunosuppressants (sirolimus, rituximab, tacrolimus)
- Participation in another clinical trial within 3 months
- Stent implantation within 1 month or myocardial infarction within 3 months
- Breastfeeding females
- Any other condition deemed inappropriate by the Investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
First Affiliated Hospital of Xian Jiaotong University
Xi'an, Shaanxi, China, 710061
Actively Recruiting
Research Team
T
Tao Zheng, M.D.
CONTACT
G
Ge Gao, M.M.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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