Clinical trials: Homozygous Familial Hypercholesterolemia
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.
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
Hypercholesterolemia, Familial
Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
The aim of this study is to assess the safety and efficacy of varying doses of ISIS 301012 as add-on therapy in subjects with Homozygous Familial Hypercholesterolemia
- Trial name
- NCT00280995
- Testing
- ISIS 301012
- Where
- 6 sites in United States, Netherlands
- Volunteers
- 12 people
- Sponsor
- Kastle Therapeutics, LLC
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 (HoFH)
Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients
This study is designed to help identify patients with HoFH due to mutations in the LDLR as confirmed by genotyping.
- Trial name
- NCT04148001
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- REGENXBIO Inc.
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, Familial Hypercholesterolemia - Heterozygous
Early Detection of Familial Hypercholesterolemia in Children
Heterozigous FH is an underdiagnosed disease in the paediatric population. Its early detection, would allow us to initiate lifestyle therapeutical changes and early pharmacological therapy if necessary.
- Conditions
- Familial Hypercholesterolemia, Familial Hypercholesterolemia - Heterozygous, Familial Hypercholesterolemia - Homozygous
- Trial name
- DECOPIN (NCT04370899)
- Testing
- lifestyle assessment
- Where
- 2 sites in Spain
- Volunteers
- 400 people
- Sponsor
- Institut Investigacio Sanitaria Pere Virgili
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
Homozygous Familial Hypercholesterolemia
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to <18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and…
- Trial name
- SPRUCE (NCT07473843)
- Testing
- Zodasiran
- Where
- 3 sites in United States, Denmark and 1 more
- Volunteers
- 10 people
- Sponsor
- Arrowhead Pharmaceuticals
Familial Hypercholesterolemia - Homozygous
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to <12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein…
- Trial name
- ORION-19 (NCT06597006)
- Testing
- Inclisiran
- Where
- 19 sites in United States, Austria and 9 more
- Volunteers
- 9 people
- Sponsor
- Novartis Pharmaceuticals
Hyperlipoproteinemia Type II, Homozygous Familial Hypercholesterolemia
Efficacy and Tolerability of Anacetrapib Added to Ongoing Lipid-Lowering Therapy in Adult Participants With Homozygous Familial Hypercholesterolemia (HoFH) (MK-0859-042)
This study will evaluate the safety and effect of anacetrapib on low-density lipoprotein-cholesterol (LDL-C) when added to ongoing lipid-lowering therapy.
- Trial name
- NCT01841684
- Testing
- Anacetrapib
- Volunteers
- 2 people
- Sponsor
- Merck Sharp & Dohme 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
Homozygous Familial Hypercholesterolemia
Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia
This is an open-label study designed to evaluate the long-term safety and efficacy of evinacumab, a fully human ANGPTL3 antibody, in patients with homozygous familial hypercholesterolemia (HoFH), in a real-life setting in Canada.
- Trial name
- NCT05611528
- Testing
- Evinacumab
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- Daniel Gaudet
Hypercholesterolemia
An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Primary Objective: To evaluate the efficacy of alirocumab (75 or 150 milligrams [mg] depending on body weight [BW]), administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels at Week 12 of treatment…
- Trial name
- NCT03510715
- Testing
- Alirocumab SAR236553 (REGN727), Atorvastatin, Simvastatin
- Where
- 10 sites in Brazil, Canada and 8 more
- Volunteers
- 18 people
- Sponsor
- Sanofi
Homozygous Familial Hypercholesterolemia
Study of cardiovAscular Contrasted Phenotypes in Patients With FamIliaI hypercholesteRolemia
The main objective of SAFIR is to identify the atherosclerotic genetic factors in these patients, which will identify new therapeutic targets for the treatment of CV and Familial Hypercholesterolemia diseases.
- Trial name
- SAFIR (NCT03234127)
- Testing
- Whole Genome Sequencing
- Where
- 9 sites in France
- Volunteers
- 562 people
- Sponsor
- Nantes University Hospital
Homozygous Familial Hypercholesterolemia
The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository
This repository will establish for the first time a system to carefully assess and monitor over time the general health and the amount of cholesterol in the arteries of U.S.
- Trial name
- NCT01109368
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- The Rogosin Institute
Familial Hypercholesterolemia
A Study to Determine the Safety, Tolerability, and Efficacy of SHR-1209 in Patients With Familial Hypercholesterolemia
The study is being conducted to evaluate the efficacy, safety and tolerability of SHR-1209 in subjects with familial hypercholesterolemia.
- Trial name
- NCT04455581
- Testing
- SHR-1209
- Volunteers
- 8 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Hypercholesterolemia
Safety and Tolerability Study of Ezetimibe (SCH 058235/MK-0653) Plus Atorvastatin or Simvastatin in Homozygous Familial Hypercholesterolemia (P01417/MK-0653-019)
The primary purpose of this study is to evaluate the long-term safety and tolerability of ezetimibe (SCH 058235/MK-0653) 10 mg dosed daily and co-administered with either atorvastatin or simvastatin for up to 24 months in…
- Trial name
- NCT03885921
- Testing
- Ezetimibe, Atorvastatin, Simvastatin
- Volunteers
- 44 people
- Sponsor
- Organon and Co
Hypercholesterolemia
Efficacy of Lapaquistat Acetate Co-Administered With Current Lipid-Lowering Treatment on Blood Cholesterol Levels in Subjects With Homozygous Familial Hypercholesterolemia
The purpose of the study is to determine the efficacy of lapaquistat acetate, once daily (QD), to lower cholesterol in subjects with homozygous familial hypercholesterolemia undergoing lipid-lowering treatment.
- Trial name
- NCT00263081
- Testing
- Lapaquistat acetate and current lipid-lowering treatment, Current lipid-lowering treatment
- Where
- 10 sites in United States, Canada and 4 more
- Volunteers
- 44 people
- Sponsor
- Takeda
Homozygous Familial Hypercholesterolemia
HoFH, the International Clinical Collaborators Registry
Homozygous familial hypercholesterolemia (HoFH), a rare inherited disorder caused by bi-allelic mutations in the LDL Receptor pathway, is characterized by extremely elevated levels of low-density lipoprotein cholesterol (LDL-C)…
- Trial name
- HICC (NCT04815005)
- Testing
- Demographics, diagnosis type, genotype, lipid profile, treatment allocation, country of residence.
- Where
- 4 sites in United States, Netherlands and 1 more
- Volunteers
- 1,000 people
- Sponsor
- University of Pennsylvania