Health TrialsbyHealthPanel

Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients

ConditionsRefractory HypercholesterolemiaFamilial Hypercholesterolemia
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

This trial does not accept healthy volunteers

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

Design model

Single group

Treatments tested in this trial

  • NGGT006

    Drug

    Single intravenous infusion of NGGT006 at low dose (7.5e12vg/kg), medium dose (1.5e13vg/kg) and high dose (3e13vg/kg).

Treatment groups

9 Participants
are divided into 1 treatment group
Group A: NGGT006Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of treatment-related adverse events (AE) and serious adverse events (SAE)

Incidence of AE and SAE, as assessed by physical examinations, clinical laboratory parameters and adverse event reporting

Time frame
52 weeks
2

Absolute change and percent change in LDL-C

Change in LDL-C concentration from baseline to week 52

Time frame
52 weeks

Secondary outcomes

1

Absolute change and percent change in non-high density lipoprotein cholesterol

Change in non-HDL-C from baseline to week 52

Time frame
52 weeks
2

Absolute change and percent change in apolipoprotein B

Change in ApoB from baseline to week 52

Time frame
52 weeks
3

Absolute change and percent change in total cholesterol

Change in TC from baseline to week 52

Time frame
52 weeks
4

Absolute change and percent change in HDL-C

Change in HDL-C from baseline to week 52

Time frame
52 weeks

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Suzhou Municipal Hospital

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: