E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hypercholesterolaemia |
Hipercolesterolemia |
|
E.1.1.1 | Medical condition in easily understood language |
High blood cholesterol level |
Nivel alto de colesterol en sangre |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 15.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10020603 |
E.1.2 | Term | Hypercholesterolaemia |
E.1.2 | System Organ Class | 10027433 - Metabolism and nutrition disorders |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the effect of SAR236553 (REGN727) on low-density lipoprotein cholesterol (LDL-C) levels after 24 weeks of treatment in comparison with placebo |
Demostrar el efecto reductor de los niveles del colesterol transportado por lipoproteínas de baja densidad (C-LDL) de SAR236553 como tratamiento en comparación con el placebo, transcurridas 24 semanas de tratamiento |
|
E.2.2 | Secondary objectives of the trial |
To evaluate the effect of SAR236553 (REGN727) in comparison with placebo on LDL-C at other time points To evaluate the effects of SAR236553 (REGN727) on other lipid parameters To evaluate the safety and tolerability of SAR236553 (REGN727) |
Evaluar el efecto de SAR236553 (REGN727) en comparación con el placebo en el C-LDL, transcurridas 12 semanas tratamiento. Evaluar los efectos de SAR236553 (REGN727) en otros parámetros lipídicos Evaluar la seguridad y la tolerabilidad de SAR236553 (REGN727) |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients with heterozygous familial hypercholesterolemia who are not adequately controlled with their lipid-modifying therapy |
Pacientes con hipercolesterolemia familiar heterocigótica no controlada adecuadamente con su terapia modificadora de los lípidos |
|
E.4 | Principal exclusion criteria |
Age < 18 years or legal age of adulthood, whichever is greater LDL-C < 70 mg/dL (1.81 mmol/L) and with cardiovascular disease LDL-C < 100 mg/dL (2.59 mmol/L) and without cardiovascular disease Fasting serum triglycerides > 400 mg/dL (4.52 mmol/L) Known history of homozygous familial hypercholesterolemia |
- Pacientes con edad inferior a los 18 años o la edad legal adulta, la que sea mayor. - Paciente con C-LDL < 70 mg/dl (< 1,81 mmol/l) y con antecedentes de cardiopatía documentada. - Paciente con C-LDL < 100 mg/dl (< 2,59 mmol/l) y sin antecedentes de cardiopatía documentada. - Nivel sanguíneo de triglicéridos en ayunas > 400 mg/dl (> 4,52 mmol/l). - Antecedentes conocidos de hipercolesterolemia familiar homocigótica. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Percent change in LDL-C |
Cambio porcentual del nivel de C-LDL desde el inicio hasta la Semana 24. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
From baseline to week 24 |
Desde la visita basal hasta la semana 24. |
|
E.5.2 | Secondary end point(s) |
Percent change in LDL-C Percent change in other lipid parameters |
Cambio porcentual del nivel de C-LDL Cambio porcentual de otros parámetros lipídicos |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
From baseline up to week 78 |
Desde la visita basal hasta la semana 78 |
|
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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
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) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 9 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 42 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Austria |
Canada |
Czech Republic |
Denmark |
France |
India |
Israel |
Netherlands |
Norway |
Russian Federation |
South Africa |
Spain |
Sweden |
United Kingdom |
United States |
|
E.8.7 | Trial has a data monitoring committee | Yes |
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
|
LVLS |
Última visita del último paciente |
|
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 | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |