Clinical trials: Hypercholesterolemia, Autosomal Dominant
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.
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.
Homozygous Familial Hypercholesterolemia
Evaluate the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
This is a single-arm, open-label study to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by SHR-1918 in patients with homozygous familial hypercholesterolemia (HoFH).
- Trial name
- NCT06009393
- Testing
- SHR-1918
- Where
- China
- Volunteers
- 26 people
- Sponsor
- Beijing Suncadia Pharmaceuticals Co., Ltd
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
Heterozygous Familial Hypercholesterolemia
A 52 Week Study To Assess The Use Of Bococizumab (PF-04950615; RN316) In Subjects With Heterozygous Familial Hypercholesterolemia
This is a multicenter, randomized study in subjects with heterozygous familial hypercholesterolemia receiving highly effective statins to assess the safety, efficacy and tolerability of Bococizumab (PF-04950615; RN316) to lower…
- Trial name
- SPIRE-FH (NCT01968980)
- Testing
- Bococizumab (PF-04950615;RN316)
- Where
- 88 sites in United States, Bulgaria and 9 more
- Volunteers
- 370 people
- Sponsor
- Pfizer
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.
Homozygous Familial Hypercholesterolemia
Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor
The primary objective of this study is to evaluate the safety and tolerability of 4 doses of lomitapide (AEGR-733; BMS-201038) given as an initial low dose and then escalated through an additional 3 dose levels over a 16-week…
- Trial name
- NCT01556906
- Testing
- Lomitapide
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Aegerion Pharmaceuticals, Inc.
Familial Hypercholesterolemia
Atorvastatin Three Year Pediatric Study
The purpose of this study is to characterize three year descriptive growth and development (ie, height, weight, body mass index, Tanner Stage) and efficacy of cholesterol reduction in pediatric subjects with Heterozygous Familial…
- Trial name
- NCT00827606
- Testing
- atorvastatin
- Where
- 40 sites in United States, Belgium and 13 more
- Volunteers
- 272 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
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
Study to Assess the Efficacy and Safety of LIB003 in HeFH Patients on Oral Lipid Therapy Needing Further LDL-C Reduction
This study is to assess LDL-C reductions at Week 24 and the mean of Weeks 22 and 24 with monthly Q4W (≤31 days) dosing of LIB003 300 mg administered subcutaneously (SC) compared to placebo in patients 18 years or older with…
- Trial name
- LIBerate-FH (NCT04797104)
- Testing
- lerodalcibep
- Where
- 7 sites in United States, Israel and 3 more
- Volunteers
- 478 people
- Sponsor
- LIB Therapeutics LLC
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 Familial Hypercholesterolemia (HeFH)
Long-Term Efficacy and Safety of Lerodalcibep in Children and Adolescents With Familial Hypercholesterolemia
The goal is to assess the long term efficacy (LDL cholesterol reduction) and safety over 3 years of lerodalcibep (Lerochol) SC 300 mg QM administered by auto-injector (AI)/pre-filled pen (PFP) in male and female pediatric…
- Trial name
- NCT07775339
- Testing
- Lerodalcibep
- Where
- 5 sites in India, Israel and 3 more
- Volunteers
- 150 people
- Sponsor
- LIB Therapeutics LLC
Dyslipidemias, High Cholesterol
Randomized Study to Evaluate the Effect of Obicetrapib on Top of Maximum Tolerated Lipid-Modifying Therapies
This study will be a placebo-controlled, double-blind, randomized, phase 3 study in participants with underlying heterozygous familial hypercholesterolemia (HeFH) and/or ASCVD to evaluate the efficacy, safety, and tolerability of…
- Conditions
- Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia
- Trial name
- BROADWAY (NCT05142722)
- Testing
- Obicetrapib
- Where
- 181 sites in United States, China and 6 more
- Volunteers
- 2,530 people
- Sponsor
- NewAmsterdam Pharma
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
Severe Familial Hypercholesterolemia
Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders
A study to assess the long term safety and tolerability of evolocumab (AMG 145) in adolescents and adults with severe familial hypercholesterolemia.
- Trial name
- TAUSSIG (NCT01624142)
- Testing
- Evolocumab
- Where
- 44 sites in United States, Australia and 16 more
- Volunteers
- 300 people
- Sponsor
- Amgen
Heterozygous Familial Hypercholesterolemia (HeFH)
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
A phase 1/2, two-part trial consisting of an open label dose-escalation part and a randomized, double blind, placebo-controlled part to investigate safety, tolerability, pharmacokinetics, and efficacy with EDIT-401 gene editing…
- Trial name
- NCT07894887
- Testing
- Drug: EDIT-401
- Where
- 2 sites in Australia
- Volunteers
- 56 people
- Sponsor
- Editas Medicine, Inc.
Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia
Patterns of Inclisiran Use in the Real World: An Analysis of US Databases
This was a retrospective observational cohort study of patients who initiated inclisiran, alirocumab, or evolocumab in a real-world setting in the United States.
- Trial name
- NCT07023445
- Where
- United States
- Volunteers
- 37,688 people
- Sponsor
- Novartis Pharmaceuticals
Homozygous Familial Hypercholesterolemia (HoFH)
A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
This first-in-human study is intended to evaluate the safety and preliminary effectiveness of AAV (Adeno-associated virus)-based liver-directed gene therapy in the treatment of adults with Homozygous Familial Hypercholesterolemia…
- Trial name
- NCT02651675
- Testing
- AAV directed hLDLR gene therapy
- Where
- 9 sites in United States, Canada and 2 more
- Volunteers
- 9 people
- Sponsor
- REGENXBIO Inc.
Non-Familial Hypercholesterolemia and Mixed Hyperlipidemia
IBI306 Monotherapy in Non-Familial Hypercholesterolemia and Mixed Hyperlipidemia
This is a multi-center, randomized, double-blind, placebo-controlled phase III clinical study evaluating the efficacy and safety of IBI306 monotherapy in Chinese Paricipants with non-familial hypercholesterolemia and mixed…
- Trial name
- NCT07473960
- Testing
- IBI306
- Where
- China
- Volunteers
- 198 people
- Sponsor
- Innovent Biologics (Suzhou) Co. Ltd.
Homozygous Familial Hypercholesterolemia
A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
This study was a Phase III,A two-part (double-blind placebo-controlled/open-label) multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in subjects with homozygous familial hypercholesterolemia (HoFH).
- Trial name
- ORION-5 (NCT03851705)
- Testing
- Inclisiran Sodium for injection
- Where
- 13 sites in Hong Kong, Israel and 6 more
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals