Search clinical trials for: Homozygous Sitosterolemia

Total 132 results

Organon and Co

Completed

Sitosterolemia Extension Study (0653-004)(COMPLETED)

Heart Disease | Lipid Metabolism, Inborn Errors
Organon and Co
Bayer

Completed

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)

Hypercholesterolemia | Familial Hypercholesterolemia | Homozygous Sitosterolemia
Organon and Co

Completed

Sitosterolemia Extension Study (0653-003)(COMPLETED)

Heart Disease | Lipid Metabolism, Inborn Errors
Organon and Co
Bayer

Completed

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)

Hypercholesterolemia | Familial Hypercholesterolemia | Homozygous Sitosterolemia
National Heart, Lung, and Blood Institute (NHLBI)

Completed

SCH-58235 (Ezetimibe) to Treat Homozygous Sitosterolemia

Lipoidosis

United States
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Higher-Dose Ezetimibe to Treat Homozygous Sitosterolemia

Heart Diseases | Metabolism, Inborn Errors

United States
InSilico Medicine Hong Kong Limited

Not yet recruiting

Study of ISM3412 in Participants With Locally Advanced/Metastatic Solid Tumors

Locally Advanced/Metastatic Solid Tumors
Amgen

Recruiting

A Study Evaluating AMG 193 in Combination With Other Therapies in Participants With Advanced Gastrointestinal, Biliary Tract, or Pancreatic Cancers With Homozygous Methylthioadenosine Phosphorylase (MTAP)-Deletion

Advanced Gastrointestinal, Biliary Tract, and Pancreatic Cancers

United States
Amgen

Recruiting

AMG 193 Alone or in Combination With Other Therapies in Subjects With Advanced Thoracic Tumors With Homozygous MTAP-deletion (Master Protocol)

Non-small Cell Lung Cancer | Thoracic Tumors

United States
First Affiliated Hospital Xi'an Jiaotong University

Recruiting

NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

China
Regeneron Pharmaceuticals

Not yet recruiting

A Trial to Learn if Receiving ALN-PNP siRNA is Safe and Well Tolerated, and How it Works in Adult Participants With Nonalcoholic Fatty Liver Disease (NAFLD) and a Genetic Risk Factor

Nonalcoholic Steatohepatitis (NASH) | Nonalcoholic Fatty Liver Disease (NAFLD) | Genetic Risk Factor
Beijing Suncadia Pharmaceuticals Co., Ltd

Not yet recruiting

Evaluate the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia
Novartis Pharmaceuticals

Recruiting

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies (V-PEDS-OLE)

Heterozygous or Homozygous Familial Hypercholesterolemia

Netherlands, Israel, Hungary, Italy, Germany, Spain, United States, France, Norway, Russian Federation, South Africa, Turkey, United Kingdom, Canada, Switzerland, Brazil, Lebanon, Slovenia, Poland, Taiwan, Malaysia
Shenzhen People's Hospital

Recruiting

Evaluate the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial Hypercholesterolemia

Lipid Metabolism Disorders | Familial Hypercholesterolemia - Homozygous | Proprotein Convertase Subtilisin/Kexin 9

China
Instituto de Investigación Sanitaria Aragón
Hospital Clínico Universitario Lozano Blesa; FEIRACO LACTEOS SL

Recruiting

Effects of UNICLA-A2 Dairy Products on Patients at High-risk of Colorectal Cancer Development

Colorectal Polyp

Spain
University of Pennsylvania
University of Amsterdam; University of Cape Town; University of Witwatersrand...

Recruiting

HoFH, the International Clinical Collaborators Registry (HICC)

Homozygous Familial Hypercholesterolemia

United States, Netherlands, South Africa
Institut Investigacio Sanitaria Pere Virgili

Recruiting

Early Detection of Familial Hypercholesterolemia in Children (DECOPIN)

Familial Hypercholesterolemia | Familial Hypercholesterolemia - Homozygous | Familial Hypercholesterolemia - Heterozygous

Spain
Amryt Pharma

Recruiting

LOWER: Lomitapide Observational Worldwide Evaluation Registry (LOWER)

Homozygous Familial Hypercholesterolemia

Italy, United States, Argentina, Canada, France, Greece, Netherlands, United Kingdom
Icahn School of Medicine at Mount Sinai

Recruiting

Longitudinal Study of the Porphyrias

Acute Porphyrias | Cutaneous Porphyrias

United States
The Rogosin Institute

Recruiting

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

Homozygous Familial Hypercholesterolemia

United States
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Cardiovascular Evaluation of Patients With High Cholesterol and Normal Volunteers

Homozygous Familial Hypercholesterolemic

United States
Arrowhead Pharmaceuticals

Active, not recruiting

Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH) (Gateway)

Homozygous Familial Hypercholesterolemia

Australia, Canada, United States, South Africa
H. Lee Moffitt Cancer Center and Research Institute
Novartis Pharmaceuticals

Active, not recruiting

Study of Trametinib + Ceritinib in Patients With Unresectable Melanoma

Melanoma | Unresectable Melanoma | Advanced Melanoma

United States
Regeneron Pharmaceuticals

Completed

Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

United States, Netherlands, South Africa, Australia, Austria, Canada, Czechia, France, Greece, Italy, Japan, Ukraine
Novartis Pharmaceuticals

Active, not recruiting

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia (ORION-13)

Familial Hypercholesterolemia - Homozygous

Greece, Lebanon, Turkey, France, Canada, Malaysia, Netherlands, United States
Novartis Pharmaceuticals

Completed

Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C (ORION-8)

Elevated Cholesterol | Homozygous Familial Hypercholesterolemia | Heterozygous Familial Hypercholesterolemia | ASCVD

United States, Canada, Czechia, Denmark, Germany, Hungary, Netherlands, Poland, South Africa, Spain, Sweden, Ukraine, United Kingdom
Amgen

Completed

Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) (HAUSER-OLE)

Familial Hypercholesterolemia

Canada, Italy, Norway, Belgium, United States, Turkey, Australia, Austria, South Africa, Poland, Switzerland, Spain, Portugal, Slovenia, Brazil, Russian Federation, Hungary, United Kingdom, Colombia, Czechia, Netherlands, Greece, Ma...
Vertex Pharmaceuticals Incorporated

Completed

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

Cystic Fibrosis

United States, United Kingdom, Belgium, Netherlands, France, Denmark, Israel, New Zealand, Australia, Ireland, Sweden, Canada, Germany, Poland, Switzerland, Italy, Austria, Hungary, Greece, Norway
Bayer
Merck Sharp & Dohme LLC

Terminated

Early Signs of Efficacy Study With Riociguat in Adult Homozygous Delta F508 Cystic Fibrosis Patients

Cystic Fibrosis

Germany, United States, France, Canada, Belgium, Netherlands, United Kingdom
Vertex Pharmaceuticals Incorporated

Completed

Long-term Safety of Lumacaftor/Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

Cystic Fibrosis

United States, Canada
Daniel Gaudet
Ultragenyx Pharmaceutical Inc

Active, not recruiting

Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

Canada
Innovent Biologics (Suzhou) Co. Ltd.

Completed

A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

China
REGENXBIO Inc.
National Heart, Lung, and Blood Institute (NHLBI)

Terminated

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

Homozygous Familial Hypercholesterolemia (HoFH)

United States, Canada, Italy, Netherlands
Regeneron Pharmaceuticals

Completed

Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

United States, Australia, Austria, Netherlands, Taiwan, Ukraine
Amryt Pharma

Active, not recruiting

Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)

Homozygous Familial Hypercholesterolaemia (HoFH)

Germany, Israel, Italy, Saudi Arabia, Spain, Tunisia
LIB Therapeutics LLC

Completed

Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH

Homozygous Familial Hypercholesterolemia

United States, Israel, Norway, South Africa, Turkey, India
Invenux, LLC
State University of New York - Downstate Medical Center

Active, not recruiting

Dose-Escalation Study of SCD-101 in Sickle Cell Disease

Sickle Cell Disease | Sickle-Beta Zero Thalassemia

United States
REGENXBIO Inc.

Active, not recruiting

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

Homozygous Familial Hypercholesterolemia (HoFH)

United States, Canada, Netherlands
Akeso
AD Pharmaceuticals Co., Ltd.

Completed

A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH

Homozygous Familial Hypercholesterolemia

China
Centre Leon Berard

Completed

Evaluation of ABEMACICLIB Monotherapy in Patients With Locally Advanced/Metastatic Head and Neck Cancer After Failure of Platinum and Cetuximab or Anti-EGFR-based Therapy and Harboring an Homozygous Deletion of CDKN2A, and/or an Amplification of CCND1 and/or of CDK6 (ABORL)

Head and Neck Cancer | Metastatic Cancer | Advanced Cancer

France
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Homozygous Familial Hypercholesterolemia (BioHoFH) (BioHoFH)

Lipoprotein Lipase Deficiency | Inborn Error of Lipid Metabolism | Corneal Arcus

Germany, India, Sri Lanka
Novartis Pharmaceuticals

Completed

A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH) (ORION-5)

Homozygous Familial Hypercholesterolemia

Hong Kong, Israel, Russian Federation, Serbia, South Africa, Taiwan, Turkey, Ukraine
Vertex Pharmaceuticals Incorporated

Completed

Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

Cystic Fibrosis

United States, Canada
Amgen

Completed

Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders (TAUSSIG)

Severe Familial Hypercholesterolemia

Italy, Czechia, United States, Israel, New Zealand, South Africa, France, Spain, Australia, Belgium, Canada, Netherlands, Hong Kong, Brazil, United Kingdom, Greece, Japan, Lebanon
Vertex Pharmaceuticals Incorporated

Completed

A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

Cystic Fibrosis

Germany
Assistance Publique - Hôpitaux de Paris

Completed

Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. (PREDICT-CF)

Cystic Fibrosis | Homozygous F508del Mutation

France
Imara, Inc.

Completed

A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)

Sickle Cell Disease

United States, United Kingdom
Organon and Co

Terminated

Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED)

Lipid Metabolism, Inborn Errors | Hypercholesterolemia, Familial
Organon and Co

Completed

Safety and Tolerability Study of Ezetimibe (SCH 058235/MK-0653) Plus Atorvastatin or Simvastatin in Homozygous Familial Hypercholesterolemia (P01417/MK-0653-019)

Hypercholesterolemia
Organon and Co

Completed

An Investigational Drug Study to Lower Non-Cholesterol Sterol Levels Associated With Sitosterolemia (0653-062)(COMPLETED)

Heart Disease | Lipid Metabolism, Inborn Errors

Search clinical trials for: Homozygous Sitosterolemia

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