Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients
Trial statusNot yet recruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-55
SponsorSuzhou Municipal Hospital
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 patients with refractory Hypercholesterolemia diagnosed by gene testing for familial hypercholesterolemia. NGGT006 uses adeno-associated virus (AAV) as a vector, carrying a liver specific promoter and 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
18 ≤ age ≤ 55 years old;
A patient with a clear diagnosis of refractory hypercholesterolemia and confirmed by genetic testing to be familial hypercholesterolemia;
AAV binding antibody titer ≤1:80 and AAV neutralizing antibody ≤1:5;
18≤BMI (body mass index)≤35;
Disqualifiers
Secondary hyperlipidemia;
Use of other drugs or nutritional products that may affect blood lipids (such as fibrates) within 6 weeks;
Have received low-density lipoprotein apheresis (LDL apheresis) within the past 2 months;
Large weight fluctuations (≥5kg) in the past 2 months;
Trial design
Treatments tested in this trial
- NGGT006
Treatment groups
9 Participants
are divided into 1 treatment groupLocations
Sponsors and collaborators
Source ClinicalTrials.gov