Clinical trials: Homozygous Familial Hypercholesterolemia
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
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
Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) with alirocumab subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment.
- Trial name
- ODYSSEY HoFH (NCT03156621)
- Testing
- Alirocumab
- Where
- 28 sites in United States, Austria and 11 more
- Volunteers
- 69 people
- Sponsor
- Regeneron Pharmaceuticals
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 Hypercholesterolaemia (HoFH)
Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)
This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable lipid lowering therapy (LLT) (including lipoprotein…
- Trial name
- NCT04681170
- Testing
- Lomitapide
- Where
- 12 sites in Germany, Israel and 4 more
- Volunteers
- 46 people
- Sponsor
- Amryt Pharma
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, HoFH
HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia
Assess the effect on coronary atheroma of serial infusions of autologous selectively delipidated HDL/preβ enriched plasma following use of HDL Therapeutics PDS-2™ System
- Trial name
- NCT03135184
- Testing
- HDL Therapeutics PDS-2 System
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- HDL Therapeutics
Hypercholesterolemia, Familial Hypercholesterolemia
A 52-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 P05245)
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
- NCT00705211
- Testing
- Ezetimibe, Ezetimibe + other lipid-lowering medication(s)
- Volunteers
- 1,794 people
- Sponsor
- Organon and Co
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
Homozygous Familial Hypercholesterolemia
Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
The primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH).
- Trial name
- NCT04233918
- Testing
- Evinacumab
- Where
- 11 sites in United States, Australia and 4 more
- Volunteers
- 20 people
- Sponsor
- Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia
Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects
The available medications used to treat HoFH are targeted at reducing circulating levels of total and LDL-cholesterol. These measures can retard the progression of cardiovascular disease, however, they are unlikely to regress…
- Trial name
- MODE (NCT01412034)
- Testing
- CER-001
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 23 people
- Sponsor
- Cerenis Therapeutics, SA
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
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
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.
Familial Hypercholesterolemia
Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
This was a long term follow on study to assess the continued long term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia.
- Trial name
- NCT00943306
- Testing
- lomitapide
- Where
- 10 sites in United States, Canada and 2 more
- Volunteers
- 19 people
- Sponsor
- Aegerion Pharmaceuticals, Inc.
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
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant
Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia
The purpose of this study is to evaluate the safety and efficacy of extended dosing of mipomersen in patients with familial hypercholesterolemia on lipid-lowering therapy who have completed either the 301012-CS8 (NCT00280995) or…
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases
- Trial name
- NCT00477594
- Testing
- mipomersen sodium
- Where
- 5 sites in United States
- Volunteers
- 21 people
- Sponsor
- Kastle Therapeutics, LLC
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
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
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