Implitapide in Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering Therapy

Study Overview
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Brief Summary
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The purpose of this study is to determine if implitapide, used in conjunction with other lipid-lowering therapies, is safe and effective when compared to placebo in lowering low-density lipoprotein cholesterol (LDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).

Official Title
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Safety and Efficacy Study of Implitapide Compared With Placebo in Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering Therapy

Conditions
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Familial Hypercholesterolemia

Intervention / Treatment
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* Drug: Implitapide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: In order to participate in this study, patients must meet all of the following inclusion criteria: be between 8 and 70 years old with a diagnosis of HoFH; be stable on and maintain concomitant therapy with hypolipidemic drugs or treatments; have an appropriate calculated, fasting LDL-C levels and an appropriate triglyceride (TG) level; be male or nonpregnant, nonlactating female; give informed consent; and meet body weight and height requirements. Exclusion Criteria: In order to participate in this study, patients must not meet any of the following exclusion criteria: recent myocardial infarction, percutaneous transluminal coronary intervention, coronary artery bypass graft surgery, or cerebrovascular accident; uncontrolled hypothyroidism or other uncontrolled endocrine disease; known, clinically significant eye abnormalities (e.g., cataracts); appropriate serum creatinine phosphokinase levels; history of liver disease or liver enzyme levels above appropriate levels; alkaline phosphatase above appropriate levels; liver cirrhosis and severe liver steatosis; clinically significant infection, malignancy, or psychosis; use of oral anticoagulants or digoxin, unless the dose has been stable for 4 weeks; participation in any other investigational study, including device or observational studies, within 30 days; lactating or have a positive serum pregnancy test; history of or current drug or alcohol abuse; or unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the investigator.

Ages Eligible for Study
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Minimum Age: 8 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Implitapide|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Homozygous Familial Hypercholesterolemia (HoFH)