Clinical trials: Familial Hypercholesterolemia - Homozygous
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 Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
This multicenter, randomized, placebo-controlled study will evaluate the efficacy and safety of zodasiran subcutaneous (SC) injection in subjects 12 years of age and older with genetically or clinically diagnosed Homozygous…
- Trial name
- NCT07037771
- Testing
- zodasiran Injection
- Where
- 43 sites in United States, Australia and 16 more
- Volunteers
- 70 people
- Sponsor
- Arrowhead Pharmaceuticals
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)
A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia
The purpose of the study is to establish the efficacy, safety and tolerability of rosuvastatin in children and adolescents with homozygous familial hypercholesterolemia.
- Trial name
- HYDRA (NCT02226198)
- Testing
- Rosuvastatin 20mg
- Where
- 9 sites in Belgium, Canada and 5 more
- Volunteers
- 20 people
- Sponsor
- AstraZeneca
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
Homozygous Familial Hypercholesterolemia
Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH
To compare the safety, tolerability and LDL-C response after 24 Weeks of monthly (every 4 weeks [Q4W]) subcutaneous (SC) dosing of LIB003 300 mg with monthly (Q4W) SC dosing of 420 mg evolocumab (Repatha®) in patients with HoFH…
- Trial name
- NCT04034485
- Testing
- lerodalcibep, evolocumab
- Where
- 12 sites in United States, India and 4 more
- Volunteers
- 65 people
- Sponsor
- LIB Therapeutics LLC
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 (HoFH)
A Study to Evaluate the Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia
The purpose of the study is to evaluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia.
- Trial name
- NCT02434497
- Testing
- Rosuvastatin 20mg
- Where
- 6 sites in Belgium, Canada and 4 more
- Volunteers
- 9 people
- Sponsor
- AstraZeneca
Lipoprotein Lipase Deficiency, Inborn Error of Lipid Metabolism
Biomarker for Homozygous Familial Hypercholesterolemia (BioHoFH)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Homozygous familial Hypercholesterolemia from blood
- Conditions
- Lipoprotein Lipase Deficiency, Inborn Error of Lipid Metabolism, Corneal Arcus
- Trial name
- BioHoFH (NCT03198897)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
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.
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
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
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
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
Homozygous Familial Hypercholesterolemia
Evaluate the Long-term Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
A multicenter, single arm, open label phase II clinical study evaluating the long-term efficacy and safety of SHR-1918 in homozygous familial hypercholesterolemia patients
- Trial name
- NCT07133815
- Testing
- SHR-1918
- Where
- China
- Volunteers
- 55 people
- Sponsor
- Beijing Suncadia Pharmaceuticals Co., Ltd
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 Hypercholesterolemia
NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia
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…
- Trial name
- NCT06125847
- Testing
- NGGT006
- Where
- China
- Volunteers
- 21 people
- Sponsor
- First Affiliated Hospital Xi'an Jiaotong University
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
Effects of Lomitapide on Carotid and Aortic Atherosclerosis
Study to assess changes in carotid and aortic atherosclerosis in patients being treated with lomitapide.
- Trial name
- NCT02399852
- Testing
- Lomitapide
- Sponsor
- Aegerion Pharmaceuticals, Inc.
Homozygous Familial Hypercholesterolemia
Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial…
- Trial name
- NCT03399786
- Testing
- evinacumab
- Where
- 30 sites in United States, Australia and 9 more
- Volunteers
- 65 people
- Sponsor
- Regeneron Pharmaceuticals