Clinical trials: Familial Hypercholesterolemia - Homozygous
Homozygous Familial Hypercholesterolemia (HoFH)
A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 in Chinese HoFH Patients
Significant reduction in ANGPLT3 levels is expected to reduce plasma LDL-C and TG levels and may lead to the reduction of risk of coronary heart disease and cardiovascular events associated with hypertriglyceridemia and LDL-C in…
- Trial name
- NCT06712771
- Testing
- VSA003
- Where
- China
- Volunteers
- 46 people
- Sponsor
- Visirna Therapeutics HK Limited
Pediatric Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (HTG)
Compassionate Use of Evinacumab
Provide compassionate use of evinacumab
- Conditions
- Pediatric Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (HTG), Dysbetalipoproteinemia (DBL)
- Trial name
- NCT06500598
- Testing
- Evinacumab
- Sponsor
- Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia
LOWER: Lomitapide Observational Worldwide Evaluation Registry
This global product exposure registry is a multicentre, long-term, prospective, observational cohort study (exposure registry), designed to evaluate the long term safety and effectiveness of lomitapide.
- Trial name
- LOWER (NCT02135705)
- Testing
- Lomitapide
- Where
- 71 sites in United States, Argentina and 6 more
- Volunteers
- 260 people
- Sponsor
- Amryt Pharma
Homozygous Familial Hypercholesterolemia
Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)
Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks.
- Trial name
- Gateway (NCT05217667)
- Testing
- ARO-ANG 3 Injection
- Where
- 7 sites in United States, Australia and 2 more
- Volunteers
- 18 people
- Sponsor
- Arrowhead Pharmaceuticals
Homozygous Familial Hypercholesterolemia
A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)
The goal of this trial is to study the effects of AEGR-733 on LDL cholesterol, other lipids as well as measures of safety over the long-term.
- Trial name
- NCT00730236
- Testing
- AEGR-733
- Where
- 10 sites in United States, Canada and 2 more
- Volunteers
- 29 people
- Sponsor
- Aegerion Pharmaceuticals, Inc.
Homozygous Familial Hypercholesterolemia
Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy
This is a Phase 3 single-arm, open-label, international, multi-center clinical trial to evaluate the efficacy and safety of lomitapide in pediatric patients with HoFH who are receiving stable lipid-lowering therapy, including LDL…
- Trial name
- NCT02765841
- Testing
- Lomitapide
- Sponsor
- Aegerion Pharmaceuticals, Inc.
Hyperlipemia
To Evaluate the Efficacy and Safety of JS002 in HoFH Patients
JS002 is a recombinant human anti-PCSK9 monoclonal antibody. This phase II open-label, single-arm study aims to evaluate the efficacy and safety of JS002 in patients with homozygous familial hypercholesterolemia A dose group (450…
- Trial name
- NCT04515927
- Testing
- JS002
- Where
- China
- Volunteers
- 31 people
- Sponsor
- Shanghai Junshi Bioscience Co., Ltd.
Hypercholesterolemia, Familial, Lipid Metabolism, Inborn Errors
Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED)
The purpose of this study is to provide an investigational drug to patients with a specific type of hypercholesterolemia (high cholesterol) or sitosterolemia (unusually high absorption of non-cholesterol sterols) in a treatment…
- Trial name
- NCT00092833
- Testing
- Comparator: ezetimibe
- Volunteers
- 49 people
- Sponsor
- Organon and Co
ASCVD, Elevated Cholesterol
Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C
The purpose of this extension study was to evaluate the efficacy, safety, and tolerability of long-term dosing of Inclisiran. The study was a global multicenter study.
- Conditions
- ASCVD, Elevated Cholesterol, Heterozygous Familial Hypercholesterolemia, Homozygous Familial Hypercholesterolemia
- Trial name
- ORION-8 (NCT03814187)
- Testing
- Inclisiran Sodium
- Where
- 238 sites in United States, Canada and 11 more
- Volunteers
- 3,275 people
- Sponsor
- Novartis Pharmaceuticals
Homozygous Familial Hypercholesterolemic
Cardiovascular Evaluation of Patients With High Cholesterol and Normal Volunteers
Homozygous familial hypercholesterolemia is a rare inherited disease of metabolism. It occurs in less than 1 in 1 million people within the United States.
- Trial name
- NCT00001204
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Familial Hypercholesterolemia
Ezetimibe (SCH 58235) Taken With Either Atorvastatin or Simvastatin in Participants With Familial Hypercholesterolemia (MK-0653-018)
The primary objective of this study is to evaluate the efficacy and the safety of ezetimibe (SCH 58235) co-administered with either atorvastatin or simvastatin in participants with homozygous familial hypercholesterolemia (FH).
- Trial name
- NCT03884452
- Testing
- Atorvastatin, Simvastatin, Ezetimibe
- Volunteers
- 50 people
- Sponsor
- Organon and Co
Homozygous Familial Hypercholesterolemia
Study to Evaluate the Effects of MBX-8025 in Patients With HoFH
A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH).
- Trial name
- NCT02472535
- Testing
- MBX-8025 50 mg (Dose Escalation Period 1), MBX-8025 50 mg or 100 mg (Dose Escalation Period 2), MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)
- Where
- 5 sites in Canada, France and 2 more
- Volunteers
- 13 people
- Sponsor
- Gilead Sciences
Homozygous Familial Hypercholesterolemia
A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study Evaluating the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
This is a multicenter, randomized, double-blind, placebo-controlled phase III clinical study to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by SHR-1918 in patients with homozygous familial…
- Trial name
- NCT06723652
- Testing
- SHR-1918
- Where
- China
- Volunteers
- 55 people
- Sponsor
- Beijing Suncadia Pharmaceuticals Co., Ltd
Diagnosed With HoFH
Phase II Clinical Trial to Evaluate the Efficacy and Safety of SYH2070 Injection in Participants With Homozygous Familial Hypercholesterolemia
This trial is a multicenter, open-label, phase II clinical study, aiming to evaluate the efficacy and safety of SYH2070 injection in participants with HoFH.
- Trial name
- NCT07591298
- Testing
- SYH2070 injection dose1, SYH2070 injection dose2
- Where
- China
- Volunteers
- 18 people
- Sponsor
- CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
Hypercholesterolemia, Familial
Study To Evaluate The Effect Of Torcetrapib/Atorvastatin In Patients With Genetic High Cholesterol Disorder
The Torcetrapib project was terminated on December 2, 2006 due to safety findings. To evaluate the efficacy and safety of the lipid drug Torcetrapib/atorvastatin in patients with genetically known disorder of extremely high…
- Trial name
- NCT00134511
- Testing
- Torcetrapib/atorvastatin
- Where
- 6 sites in United States, Canada and 1 more
- Volunteers
- 30 people
- Sponsor
- Pfizer
Homozygous Familial Hypercholesterolemia
A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH
AK102 is being developed for the treatment of HoFH. The study will be conducted in 2 parts, part 1 is open label, single arm study to evaluate the safety, tolerability and efficacy of PCSK9 inhibitor AK102, and part 2 is double…
- Trial name
- NCT03933293
- Testing
- AK102, Statins, Ezetimibe
- Where
- 2 sites in China
- Volunteers
- 10 people
- Sponsor
- Akeso
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
To evaluate the safety and efficacy of extended dosing with mipomersen (ISIS 301012) in participants with familial hypercholesterolemia or severe hypercholesterolemia on lipid-lowering therapy who had completed either the…
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases
- Trial name
- NCT00694109
- Testing
- Mipomersen Sodium
- Where
- 33 sites in United States, Brazil and 5 more
- Volunteers
- 144 people
- Sponsor
- Kastle Therapeutics, LLC
Hypercholesterolemia, Familial Hypercholesterolemia
A 12-week Post-marketing, Observational Study to Confirm the Safety and Efficacy of Zetia Alone or in Combination With Other Lipid-lowering Drugs in Japanese Subjects With Hypercholesterolemia (Study P05244)
This study is a non-interventional (observational) study in Japan to confirm the safety and efficacy of Zetia when administered alone or in combination with other lipid-lowering drugs in daily medical practice throughout a…
- Conditions
- Hypercholesterolemia, Familial Hypercholesterolemia, Homozygous Sitosterolemia
- Trial name
- NCT00704444
- Testing
- Ezetimibe, Ezetimibe + other lipid-lowering medication(s)
- Volunteers
- 11,332 people
- Sponsor
- Organon and Co
Familial Hypercholesterolemia
Effects of LDL Apheresis System on the Expression of Genes Involved in Lipoprotein Metabolism and Inflammation in Homozygotes for Familial Hypercholesterolemia
Familial hypercholesterolemia (FH) is an autosomal codominant single-gene disorder caused by mutations in the LDL receptor gene that disrupt the normal clearance of LDL particles from the plasma compartment.
- Trial name
- LA-PBMC (NCT02462655)
- Testing
- Pre-lipid apheresis, Post-lipid apheresis
- Where
- Canada
- Volunteers
- 9 people
- Sponsor
- Laval University
Homozygous Familial Hypercholesterolemia HoFH
Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia
To describe the safety and tolerability of evolocumab in participants with homozygous familial hypercholesterolemia (HoFH) in India. All participants will receive evolocumab over an 8-week period.
- Trial name
- RAMAN (NCT03403374)
- Testing
- evolocumab
- Where
- 12 sites in India
- Volunteers
- 30 people
- Sponsor
- Amgen