Homozygous Familial Hypercholesterolemia Clinical Trial
Official title:
A Clinical Study for the Safety and Efficacy of Intravenous Infusion of NGGT006 in Treatment of Homozygous Familial Hypercholesterolemia With LDLR Mutations
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).
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 to early onset of severe coronary artery disease. This is an early phase 1, open-label, single-center, dose-escalation, pilot trial to evaluate the safety and efficacy of a single intravenous infusion of NGGT006 in 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). 3-12 subjects will be enrolled and divided into 3 groups according to the principle of dose escalation, respectively administered intravenous infusion of NGGT006 at low dose (7.5e12vg/kg), medium dose (1.5e13vg/kg) and high dose (3e13vg/kg). All subjects will undergo 52 weeks of treatment observation and further 260 weeks of long-term follow-up. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT04031742 -
A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia
|
Phase 2/Phase 3 | |
| Not yet recruiting |
NCT06009393 -
Evaluate the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
|
Phase 2 | |
| Completed |
NCT03156621 -
Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
|
Phase 3 | |
| Completed |
NCT01878604 -
Gene Analysis and Treatment Optimization in Chinese Homozygous Familial Hypercholesterolemia
|
N/A | |
| Completed |
NCT01412034 -
Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects
|
Phase 2 | |
| Withdrawn |
NCT02765841 -
Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy
|
Phase 3 | |
| Completed |
NCT03933293 -
A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH
|
Phase 2 | |
| Completed |
NCT00704535 -
Evaluation of the Safety, Tolerability and Efficacy of Ezetimibe on a Select Population of Filipinos With Hypercholesterolemia (Study P04748)(COMPLETED)
|
||
| Recruiting |
NCT02135705 -
LOWER: Lomitapide Observational Worldwide Evaluation Registry
|
||
| Completed |
NCT03409744 -
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
|
Phase 3 | |
| Completed |
NCT04233918 -
Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
|
Phase 3 | |
| Withdrawn |
NCT02399852 -
Effects of Lomitapide on Carotid and Aortic Atherosclerosis
|
N/A | |
| Terminated |
NCT01841684 -
Efficacy and Tolerability of Anacetrapib Added to Ongoing Lipid-Lowering Therapy in Adult Participants With Homozygous Familial Hypercholesterolemia (HoFH) (MK-0859-042)
|
Phase 3 | |
| Recruiting |
NCT01109368 -
The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository
|
||
| Completed |
NCT01556906 -
Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor
|
Phase 2 | |
| Active, not recruiting |
NCT03135184 -
HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia
|
N/A | |
| Recruiting |
NCT04815005 -
HoFH, the International Clinical Collaborators Registry
|
||
| Completed |
NCT03399786 -
Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
|
Phase 3 | |
| Completed |
NCT03851705 -
A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
|
Phase 3 | |
| Completed |
NCT02265952 -
Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
|
Phase 2 |