E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary biliary cirrhosis (PBC) Primary sclerosing cholangitis (PSC) Secondary sclerosing cholangitis (SSC) |
Cirrosis biliar primaria (PBC) Colangitis esclerosante primaria (PSC) Colangitis esclerosante secundaria (SSC) |
|
E.1.1.1 | Medical condition in easily understood language |
a subset of liver diseases that are accompanied by itch |
picazón en un subconjunto de enfermedades hepáticas |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
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 |
to evaluate the effect of bezafibrate on itch in cholestatic patients |
evaluar el efecto del bezafibrato en prúrito en pacientes colestáticos |
|
E.2.2 | Secondary objectives of the trial |
- to test the effect of bezafibrate on serum autotaxin activity in cholestatic patients - to screen for hepatotoxic and other side effects (rhabdomyolysis, modifications of the lipid profile, nephrotoxic effects) of bezafibrate in cholestatic liver diseases - to determine the effect of bezafibrate on fatigue and quality of life scores in cholestatic patients |
- probar el efecto del bezafibrato en la actividad sérica de la autotaxina en pacientes colestáticos - para detectar efectos hepatotóxicos y otros efectos secundarios (rabdomiolisis, modificaciones del perfil lipídico, efectos nefrotóxicos) del bezafibrato en las enfermedades hepáticas colestáticas - Determinar el efecto del bezafibrato en las puntuaciones de fatiga y calidad de vida en pacientes colestáticos |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- age > 18 years - understanding of Dutch, German, English, Spanish or Italian language - Diagnosis of PBC, PSC or SSC as defined by EASL clinical practice guidelines of cholestasis 2009 - itch without primary dermatologic abnormalities and with an intensity score of 5 of higher on a scale from 0 to 10, scored twice within the week before start of the treatment |
- edad> 18 años - comprensión del neerlandés, alemán, inglés, español o italiano - Diagnóstico de PBC, PSC o SSC tal y como se define en las guías de práctica clínica de la EASL de la colestasis 2009 - picazón sin anomalías dermatológicas primarias y con una puntuación de intensidad de 5 en una escala de 0 a 10, la puntuación recogida dos veces en la semana anterior al inicio del tratamiento |
|
E.4 | Principal exclusion criteria |
- concomitant antipruritic therapy - pregnancy - cholestasis due to obstruction that requires invasive desobstructive treatment within the time scope of the study (5 weeks), such as ERCP or surgery - use of opiates |
- tratamiento antiprurítico concomitante - embarazo - colestasis debida a obstrucción que requiere tratamiento invasivo desobstrutivo dentro del tiempo de estudio (5 semanas), como CPRE o cirugía - uso de opiáceos |
|
E.5 End points |
E.5.1 | Primary end point(s) |
proportion of patients reaching a 50% or more reduction in itch intensity VAS score after 21 days of treatment |
proporción de pacientes que alcanzan una reducción del 50% o más en la puntuación de VAS de intensidad de picazón después de 21 días de tratamiento |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
- 5D itch score questionnaire - serum autotaxin activity serum liver, kidney function and lipid profile parameters (AST, ALT, ALP, gGT, albumin, creatinin, ureum, CK, VLDL, LDL, HDL cholesterol, triglycerides) - number of adverse and serious adverse events during and after treatment -fatigue and quality of life |
- Cuestionario de puntuación 5D - Actividad sérica de la autotaxina - Función hepática y renal y los parámetros del perfil lipídico (AST, ALT, ALP, gGT, albúmina, creatinina, ureum, CK, VLDL, LDL, colesterol HDL, triglicéridos) - número de efectos secundarios adversos y graves durante y después del tratamiento -fatiga y calidad de vida |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
day 0, 21, 35 |
Dia 0, 21, 35 |
|
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 | No |
E.6.5 | Efficacy | Yes |
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 | 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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | 13 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 13 |
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |