Clinical trials: Homozygous Familial Hypercholesterolemia (HoFH)
- Condition Homozygous Familial Hypercholesterolemia (HoFH)
- Funder type Industry (pharma, biotech, devices)
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.
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 (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.
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 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)
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501
This long-term observational study is designed to follow subjects who, during another Clinical Study, received gene therapy treatment used to treat their Homozygous Familial Hypercholesterolemia (HoFH) disease.
- Trial name
- NCT04080050
- Testing
- Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
- Where
- 6 sites in United States, Canada and 1 more
- Volunteers
- 8 people
- Sponsor
- REGENXBIO 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
Hypercholesteremia
Efficacy and Safety of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid-Lowering Therapy (COBALT-1)
The purpose of this study was to assess the efficacy, safety, and tolerability of multiple doses of Gemcabene in patients with HoFH on stable, lipid-lowering therapy.
- Trial name
- NCT02722408
- Testing
- Gemcabene
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 8 people
- Sponsor
- NeuroBo Pharmaceuticals Inc.
Homozygous Familial Hypercholesterolemia
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
The primary objectives of the study are: To evaluate the long-term safety and tolerability of evinacumab in patients with Homozygous Familial Hypercholesterolemia (HoFH) To evaluate the long-term safety and tolerability of…
- Trial name
- NCT03409744
- Testing
- evinacumab
- Where
- 38 sites in United States, Australia and 10 more
- Volunteers
- 116 people
- Sponsor
- Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia
A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
The purpose of this study is to assess the safety, tolerability, and efficacy of ALN-PCSSC in participants with homozygous familial hypercholesterolemia.
- Trial name
- ORION-2 (NCT02963311)
- Testing
- ALN-PCSSC, Standard of Care
- Where
- 3 sites in United States, Netherlands and 1 more
- Volunteers
- 9 people
- Sponsor
- The Medicines Company
Homozygous Familial Hypercholesterolemia
Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
This is an open-label, single-arm study to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by REGN1500 in patients with homozygous familial hypercholesterolemia (HoFH).
- Trial name
- NCT02265952
- Testing
- REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 9 people
- Sponsor
- Regeneron Pharmaceuticals
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
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)
A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 in Chinese HoFH Patients
Significant reduction in ANGPLT3 levels is expected to reduce plasma LDL-C and TG levels and may lead to the reduction of risk of coronary heart disease and cardiovascular events associated with hypertriglyceridemia and LDL-C in…
- Trial name
- NCT06712771
- Testing
- VSA003
- Where
- China
- Volunteers
- 46 people
- Sponsor
- Visirna Therapeutics HK Limited
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
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
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 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.
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.
Hypercholesterolemia, Familial, Lipid Metabolism, Inborn Errors
Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED)
The purpose of this study is to provide an investigational drug to patients with a specific type of hypercholesterolemia (high cholesterol) or sitosterolemia (unusually high absorption of non-cholesterol sterols) in a treatment…
- Trial name
- NCT00092833
- Testing
- Comparator: ezetimibe
- Volunteers
- 49 people
- Sponsor
- Organon and Co