Clinical trials: Heterozygous Familial Hypercholesterolemia (HeFH)
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
Heterozygous Familial Hypercholesterolemia (HeFH)
QLC7401 in Participants With Heterozygous Familial Hypercholesterolemia (HeFH)
This Phase III clinical trial aims to assess the efficacy and safety of QLC7401 Treatment in adults with Heterozygous Familial Hypercholesterolemia on a stable lipid-lowering therapy.
- Trial name
- NCT07450378
- Testing
- QLC7401 Injection
- Volunteers
- 120 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Hypercholesterolemia, Familial
Carotid IMT (Intima Media Thickening) Study (0524A-041)(TERMINATED)
This is a 105-week clinical study in patients with heterozygous familial hypercholesterolemia on intensive Low Density Lipoprotein-cholesterol (LDL-C) lowering therapy intended to assess the affects of MK0524A on carotid intima…
- Trial name
- ACHIEVE (NCT00384293)
- Testing
- Comparator: niacin (+) laropiprant (MK0524A)
- Volunteers
- 937 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Hyperlipidemias
A Study Evaluating the Safety and Efficacy of Bempedoic Acid Plus Ezetimibe Fixed-Dose Combination Compared to Bempedoic Acid, Ezetimibe, and Placebo in Patients Treated With Maximally Tolerated Statin Therapy
The purpose of this study is to determine if Bempedoic Acid (BA) + Ezetimibe (EZE) in a fixed-dose combination (FDC) is effective and safe versus its individual components and placebo in patients with elevated LDL cholesterol…
- Trial name
- NCT03337308
- Testing
- Bempedoic Acid + Ezetimibe Fixed-Dose Combination, Bempedoic Acid, Ezetimibe
- Where
- 5 sites in United States
- Volunteers
- 382 people
- Sponsor
- Esperion Therapeutics, Inc.
Familial Hypercholesterolemia
Implitapide in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) on Maximal Concurrent Lipid-Lowering Therapy
The purpose of this study is to determine if implitapide, used in conjunction with other lipid-lowering therapies, is safe and effective when compared to placebo in lowering low-density lipoprotein cholesterol (LDL-C) in patients…
- Trial name
- NCT00079859
- Testing
- Implitapide
- Where
- 6 sites in United States, Netherlands and 1 more
- Volunteers
- 120 people
- Sponsor
- Medical Research Laboratories International
Dyslipidemias, Hypercholesterolemia
Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies
The study is a placebo-controlled, double-blind, randomized, phase 3 study in participants with heterozygous familial hypercholesterolemia (HeFH) and/or atherosclerotic cardiovascular disease (ASCVD) or multiple ASCVD risk…
- Conditions
- Dyslipidemias, Hypercholesterolemia, Familial Hypercholesterolemia, ASCVD
- Trial name
- TANDEM (NCT06005597)
- Testing
- Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe
- Where
- 45 sites in United States
- Volunteers
- 407 people
- Sponsor
- NewAmsterdam Pharma
Heterozygous Familial Hypercholesterolemia
Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders
A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.
- Trial name
- HAUSER-RCT (NCT02392559)
- Testing
- Evolocumab
- Where
- 66 sites in United States, Australia and 24 more
- Volunteers
- 158 people
- Sponsor
- Amgen
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH)
The ORIGIN-FH Study
The goal of this clinical trial is to identify different types of Familial Hypercholesterolemia (FH) in infants and newborns. Participants will: undergo a cheek swab for genetic testing (parents only) have 5 blood samples…
- Conditions
- Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Familial Hypercholesterolemia
- Trial name
- ORIGIN-FH (NCT07470723)
- Testing
- Screening for FH
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- University of Wisconsin, Madison
Heterozygous Familial Hypercholesterolaemia
Efficacy and Safety Study of Eprotirome in HeFH Patients Who Are on Optimal Standard of Care
Eprotirome is a liver selective thyroid hormone that can reduce several independent risk factors for cardiovascular disease, while an euthyroid state is preserved in the extrahepatic tissue.
- Trial name
- AKKA (NCT01410383)
- Testing
- Eprotirome
- Volunteers
- 236 people
- Sponsor
- Karo Bio AB
Heterozygous Familial Hypercholesterolemia (HeFH)
A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)
This study is designed to learn if enlicitide decanoate is safe and effective to treat children and adolescents with heterozygous familial hypercholesterolemia (HeFH) and high amounts of low-density lipoprotein cholesterol…
- Trial name
- NCT07058077
- Testing
- Enlicitide Decanoate
- Where
- 47 sites in United States, Australia and 17 more
- Volunteers
- 153 people
- Sponsor
- Merck Sharp & Dohme LLC
Heterozygous or Homozygous Familial Hypercholesterolemia
Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies
The purpose of this open-label, single arm, multicenter extension study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH who have completed the ORION-16 (CKJX839C12301)…
- Trial name
- V-PEDS-OLE (NCT05682378)
- Testing
- Inclisiran
- Where
- 52 sites in United States, Argentina and 24 more
- Volunteers
- 195 people
- Sponsor
- Novartis Pharmaceuticals
Heterozygous Familial Hypercholesterolemia (HeFH)
"Detection and Follow-up of Coronary Lesions in HeFH (DESTINY-FH Study)"
This multicenter, non-randomized interventional study aims to assess coronary artery disease progression over 5 years in patients with genetically confirmed heterozygous familial hypercholesterolemia (HeFH), using coronary…
- Trial name
- DESTINY-FH (NCT07427472)
- Testing
- Coronary computed tomography angiography
- Where
- 3 sites in France
- Volunteers
- 300 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Familial Hypercholesterolemia - Heterozygous
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol…
- Trial name
- ORION-16 (NCT04652726)
- Testing
- Inclisiran
- Where
- 59 sites in United States, Argentina and 24 more
- Volunteers
- 141 people
- Sponsor
- Novartis Pharmaceuticals
Heterozygous Familial Hypercholesterolemia, Elevated Cholesterol
Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)
This is a Phase III, placebo-controlled, double-blind, randomized study in participants with HeFH and elevated LDL-C to evaluate the efficacy, safety, and tolerability of subcutaneous (SC) injection(s) of inclisiran.
- Trial name
- ORION-9 (NCT03397121)
- Testing
- Inclisiran
- Where
- 45 sites in United States, Canada and 6 more
- Volunteers
- 482 people
- Sponsor
- The Medicines Company
Heterozygous Familial Hypercholesterolemia (HeFH)
A Study to Evaluate the SYH2053 Injection in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)
Heterozygous Familial Hypercholesterolemia (HeFH) is an autosomal dominant disorder characterized by markedly elevated low-density lipoprotein cholesterol (LDL-C) and increased risk of atherosclerotic cardiovascular disease…
- Trial name
- NCT07465263
- Testing
- SYH2053 Injection
- Where
- China
- Volunteers
- 135 people, healthy volunteers welcome
- Sponsor
- CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
Hypercholesterolemia
Effects of Ezetimibe With Simvastatin in the Therapy of Adolescents With HeFH (Study P02579)
This is a randomized, double-blind, controlled, parallel-group, multicenter, Phase-3 study to evaluate the efficacy and safety of ezetimibe with simvastatin taken alone in subjects ages 10-17 years with Heterozygous Familial…
- Trial name
- NCT00129402
- Testing
- ezetimibe with simvastatin, simvastatin
- Volunteers
- 248 people
- Sponsor
- Organon and Co
Familial Hypercholesterolemia - Heterozygous
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to <12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein…
- Trial name
- ORION-20 (NCT06597019)
- Testing
- Inclisiran
- Where
- 66 sites in United States, Argentina and 21 more
- Volunteers
- 60 people
- Sponsor
- Novartis Pharmaceuticals
Hypercholesterolemia, Heterozygous Familial Hypercholesterolemia
Open-Label Extension of Study R727-CL-1003 (NCT01266876) to Evaluate the Long-Term Safety and Efficacy of Alirocumab (REGN727) in Participants With Heterozygous Familial Hypercholesterolemia (HeFH)
The primary objective of the study was to assess the long-term safety and tolerability of alirocumab in patients with heFH who were receiving concomitant treatment with hydroxymethyl glutaryl-coenzyme A (HMG-CoA) reductase…
- Trial name
- NCT01576484
- Testing
- Alirocumab
- Where
- 14 sites in United States, Canada
- Volunteers
- 58 people
- Sponsor
- Regeneron Pharmaceuticals
Cardiovascular, Metabolic Disease
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.
- Conditions
- Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder
- Trial name
- NCT07491172
- Testing
- CTX310
- Where
- 17 sites in United States, Australia and 2 more
- Volunteers
- 90 people
- Sponsor
- CRISPR Therapeutics AG
Familial Hypercholesterolemia
Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)
The main purpose of this study is to describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab when added to standard of care in children 10 to 17 years of age with familial hypercholesterolemia.
- Trial name
- HAUSER-OLE (NCT02624869)
- Testing
- Evolocumab
- Where
- 46 sites in United States, Australia and 21 more
- Volunteers
- 163 people
- Sponsor
- Amgen