Status:
RECRUITING
NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia
Lead Sponsor:
First Affiliated Hospital Xi'an Jiaotong University
Conditions:
Homozygous Familial Hypercholesterolemia
Eligibility:
All Genders
12-55 years
Phase:
EARLY_PHASE1
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 ...
Detailed Description
Homozygous familial hypercholesterolemia (HoFH) is a rare inherited disorder of lipoprotein metabolism, characterized by extreme elevations in low-density lipoprotein cholesterol (LDL-C) and leading t...
Eligibility Criteria
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.
- 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;
- 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
- 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;
- Diabetes diagnosed within 3 months or with poor control (HbA1c ≥9%);
- Acute or chronic kidney failure;
- Hemoglobin (Hb) ≥120g/L (male), Hb ≥110 (female);
- Abnormal platelet counts or morphology;
- 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 AAV 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.
Key Trial Info
Start Date :
October 29 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 1 2028
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT06125847
Start Date
October 29 2023
End Date
November 1 2028
Last Update
December 9 2024
Active Locations (1)
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1
First Affiliated Hospital of Xian Jiaotong University
Xi'an, Shaanxi, China, 710061