E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Polycystic liver disease (PLD) |
Enfermedad hepática poliquística (EHP) |
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E.1.1.1 | Medical condition in easily understood language |
Liver cysts as a consequence of polycystic liver disease |
Quistes en el hígado como consecuencia de la enfermedad poliquística del hígado |
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
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 |
First, to demonstrate whether UDCA-therapy is effective in reducing total liver volume in PLD patients. |
En primer lugar, para demostrar si el AUDC-terapia es efectiva en la reducción de volumen total de hígado en pacientes EHP. |
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E.2.2 | Secondary objectives of the trial |
Second, we want to assess if UDCA modifies quality of life. Finally, we want to assess safety and tolerability |
En segundo lugar, queremos evaluar si el UDCA modifica la calidad de vida. Por último, queremos evaluar la seguridad y tolerabilidad |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
18 ? age ? 80 years/ Polycystic liver disease with underlying diagnosis of (PCLD or ADPKD), defined as ? 20 liver cysts / Total liver volume ? 2500 mL/ Symptomatic defined as ECOG-PS ? 1, and having at least three out of ten PCLD symptoms: (Abdominal pain; Abdominal distension; Abdominal fullness; Dyspnea; Early satiety; Back pain; Nausea/vomiting; Anorexia; Weight loss; Jaundice)/ Informed consent, patients are willing and able to comply with the study drug regimen and all other study requirements. |
18 ? edad ? 80 años / Enfermedad hepática poliquística con diagnóstico subyacente de (PCLD o PQRAD), definida como ? 20 quistes hepáticos / Totalvolumen hepático ? 2.500 ml / Sintomático define como ECOG-PS ? 1, y que tiene al menos tres de cada diez síntomas PCLD: (dolor abdominal; distensión abdominal; plenitud abdominal, disnea, sensación de saciedad precoz, dolor de espalda, náuseas / vómitos, anorexia, pérdida de peso, ictericia) / El consentimiento informado, los pacientes están dispuestos y son capaces de cumplir con el régimen del fármaco del estudio y todos los demás requisitos del estudio. |
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E.4 | Principal exclusion criteria |
Use of oral anticonceptives or estrogen supplementation / Use of UDCA in 3 months before baseline / Females who are pregnant or breast-feeding or patients of reproductive potential not employing an effective method of birth control./ Intervention (aspiration or surgical intervention) within six months before baseline/ Treatment with somatostatin analogues within months before baseline/ Renal dysfunction (MDRD-GFR < 30 ml/min/1.73m2)/ Patients with a kidney transplant/ Hypersensitivity reaction to UDCA or patients with galactose-intolerance, lactase deficiency or glucose-galactose malabsorption/ Acute cholecystitis or frequent biliary colic attacks/ Acute stomach or duodenal ulcers/ Inflammation of small intestine or colon/ Use of drugs that can interact with UDCA, such as colestyramine or aluminium hydroxide/ Enrolment in another clinical trial of an investigational agent while participating in this study/ History or other evidence of severe illness or any other conditions which would make the patient, in the opinion of the investigator, unsuitable for the study/ Mental illness that interferes with the patient ability to comply with the protocol |
El uso de anticonceptives orales o la suplementación de estrógeno / El uso de UDCA en 3 meses antes de la línea de base / Las mujeres que están embarazadas o en periodo de lactancia o en pacientes de la capacidad de reproducción que no utilicen un método anticonceptivo eficaz . / Intervención (aspiración o intervención quirúrgica) en los seis meses antes de la línea de base / El tratamiento con análogos de la somatostatina en cuestión de meses antes de la línea de base / La disfunción renal ( MDRD- TFG < 30 ml/min/1.73m2 ) / Los pacientes con un trasplante de riñón / Reacción de hipersensibilidad a AUDC o en pacientes con intolerancia a la galactosa , deficiencia de lactasa o malabsorción de glucosa-galactosa / La colecistitis aguda o ataques de cólicos biliares frecuentes / Estómago aguda o úlcera duodenal / La inflamación del intestino delgado o del colon / El uso de medicamentos que pueden interactuar con el UDCA , tales como colestiramina o hidróxido de aluminio / La matrícula en otro ensayo clínico de un fármaco en investigación durante su participación en este estudio / Historia u otra evidencia de enfermedad grave o cualquier otra condición que haría que el paciente , en opinión del investigador , no aptos para el estudio / La enfermedad mental que interfiere con la capacidad del paciente para cumplir con el protocolo |
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E.5 End points |
E.5.1 | Primary end point(s) |
The main outcome measure will be the proportional change of total liver volume from baseline to 6 months as determined by CT. |
La medida de resultado principal será el cambio proporcional del volumen total del hígado desde el inicio hasta 6 meses según lo determinado por la TC. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Baseline liver volume will be compared to liver volume at 24 weeks. |
Volumen hepático línea de base se compara con el volumen hepático a las 24 semanas. |
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E.5.2 | Secondary end point(s) |
Change in absolute total liver volume/ Change in symptoms, measured by GI-questionnaire/ Change in quality of life, measured by SF-36 questionnaire/ Proportion of patients having any reduction in total liver volume after 24 weeks/ (Serious )Adverse events that occur in these 24 weeks/ TKV at baseline and after 24 weeks |
Cambio en el volumen total del hígado absoluto / Cambio en los síntomas, medida por GI-cuestionario / Cambio en la calidad de vida, medido por el cuestionario SF-36 / Proporción de pacientes con una reducción en el volumen total del hígado después de 24 semanas / (Graves) Los eventos adversos que ocurren en estos 24 semanas / TKV al inicio del estudio y después de 24 semanas |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
For all secondary time points baseline values will be compared to values at week 24. |
Para todos los puntos de tiempo secundaria valores de línea de base se comparan con los valores en la semana 24. |
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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 | 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 | Yes |
E.8.1.3 | Single blind | No |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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E.8.2.4 | Number of treatment arms in the trial | 3 |
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 | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
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 | 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
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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 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |