E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cirrosis hepática, Hipertensión portal |
|
E.1.1.1 | Medical condition in easily understood language |
Liver Cirrhosis Portal Hypertension |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10020786 |
E.1.2 | Term | Hypertension portal |
E.1.2 | System Organ Class | 10019805 - Hepatobiliary disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10019641 |
E.1.2 | Term | Hepatic cirrhosis |
E.1.2 | System Organ Class | 10019805 - Hepatobiliary 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 |
Evaluar si en pacientes con cirrosis hepática e hipertensión portal clínicamente significativa la administración de sapropterina disminuye el gradiente de presión portal sin ocasionar efectos deletéreos sobre la hemodinámica sistémica. |
|
E.2.2 | Secondary objectives of the trial |
Evaluar tolerabilidad de sapropterina en pacientes con cirrosis hepática; evolución de parámetros de función hepática tras dos semanas de tratamiento; influencia del tratamiento con sapropterina sobre la hemodinámica esplácnica y sistémica en pacientes que realizan tratamiento profiláctico de la hemorragia por varices esofágicas con propranolol/nadolol; cambios en la producción hepática de NO, marcadores de disfunción endotelial y estrés oxidativo tras tratamiento con sapropterina |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Existencia de cirrosis hepática de cualquier etiología demostrada mediante biopsia o por datos clínicos y de imagen. Pacientes de ambos sexos entre 18 y 75 años en quienes esté indicada la práctica de un cateterismo suprahepático. Hipertensión portal clínicamente significativa definida por un GPVH 10 mmHg. |
|
E.4 | Principal exclusion criteria |
Insuficiencia hepática avanzada segun definición protocolo. embarazo o lactancia inicio de beta bloqueantes durante la administración de sapropterina; no son motivo de exclusión si el paciente los recibe desde al menos 6 semanas tratamiento con carvedilol o nitratos; tratamiento porto-sistémico previo; hepatocarcinoma que exceda criterios de Milan; peritonitis bacteriana o infeccion activa en la inclusión; trmbosis o cavernomatosis, insuficiencia respiratoria, cardíaca o renal, hipersensibilidad a sapropterina, antecedentes de convulsiones |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Cambio del gradiente de presión venoso hepático provocado por el tratamiento con sapropterina durante 2 semanas. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
two weeks after treatment |
|
E.5.2 | Secondary end point(s) |
Aparición de efectos adversos graves y/o deterioro de la función hepática. Cambios en el flujo sanguíneo y aclaramiento intrínseco hepáticos. Los cambios en la presión arterial, índice cardíaco, índice de resistencias vasculares sistémicas y pulmonares. Cambios en la producción hepática y sistémica de NO. Cambios de la función hepática medidos por los scores Child-Pugh y MELD. Cambios en los parámetros sanguíneos de disfunción endotelial y de estrés oxidativo. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
two weekns after treatment |
|
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 | 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 | Yes |
E.8.1.4 | Double blind | No |
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 | 2 |
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 | Information not present in EudraCT |
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
|
last visit of last patient |
|
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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |