E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
familial hypercholesterolemia |
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E.1.1.1 | Medical condition in easily understood language |
familial hypercholesterolemia is characterised by elevated levels of cholesterol in plasma |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cardiovascular Diseases [C14] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The objective is to develope a mathematical model describing the lipoproteins of low density (LDL) in healthy probands and patients with familial hypercholesterinemia based on clinical data |
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E.2.2 | Secondary objectives of the trial |
Secondary objectives are to characterize the influence of statin on LDL metabolism and to compare healthy probands and patients with familial hypercholesterinemia. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
inclusion criteria for FH: • Age 18-75 years • written consent • positive Simon Broome criteria (possible and definitely) • no lipid-lowering therapy in the 6 weeks before inclusion inclusion criteria control subjects: • Age 18-75 years • written consent • Patients without disorder of LDL metabolism, proven by< 200 mg/dl cholesteryl, < 150 mg/dl triglycerides and <160 mg/dl LDL-cholesteryl in fasting plasma • no relevant internistic deseases and no lipidlowering therapy in the 6 weeks before inclusion • no lipid-lowering medication 6 weeks before the first study-associated blood withdrawl and until the second visit. |
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E.4 | Principal exclusion criteria |
1. Active Liver-disease or impairment of liver function with GOT and/or GPT > 2 x value of the Upper Limit of Normal= (ULN) 2. mid-level or severe renal insufficiency 3. TSH not im reference range 4. uncontrolled aterial hypertension: Diastolic RR 105 mmHg and/or systolic RR 160 mmHg 5. lipid-lowering therapy within the last 6 weeks before the screening 6. alcohol abuse, smoking or other drug abuse 7. blood donation within the last 6 weeks before the screening 8. Patients with a adverse acute disease 9. HbA1c > 6.5% 10. Other important striking internistic diseases, which may alter the lipidmetabolism |
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E.5 End points |
E.5.1 | Primary end point(s) |
Apolipoprotein B (ApoB), triglyceride (TG), free cholesteryl (FC) and cholesteryl ester (CE) distribution in the LDL and their subfractions |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
activity of the following enzyms: Lecithin—cholesterol acyltransferase (LCAT), Cholesterylester transfer protein (CETP), Phospholipid transfer protein (PLTP), Lipoprotein-associated phospholipase A2 (LP-PLA2) concentration of the lipids: TG, CE, FC and phospholipids (PL) in all lipoprotein-subfractions concentration of the apolipoproteins: Apolipoprotein-A1, Apolipoprotein-A2, Apolipoprotein B-100, Apolipoprotein-E, Apolipoprotein-CII, Apolipoprotein-CIII and Lipoprotein Lp(a) in all lipoprotein subfractions, HDL functionality |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Evaluate data to create a mathematical model |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The trial ends after the last visit of the last subject |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |