E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Autosomal Dominant Polycystic Kidney Disease |
Malattia del rene policistico autosomico dominante |
|
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 | 10036046 |
E.1.2 | Term | Polycystic kidney, autosomal dominant |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic 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 |
Short-term (1-year) To compare the effect of long acting somatostatin analogue versus placebo on total kidney volume (TKV) change (delta TKV) as assessed by spiral computed tomography (spiral CT) scan. Long-term (3-years) To compare the effect of long-acting somatostatin analogue vs. placebo on the rate of GFR decline as assessed by serial measurements of the iohexol plasma clearance. |
Breve-termine (1 anno) Paragonare l’effetto dell’analogo della somatostatina long-lasting rispetto al placebo sul cambiamento del volume totale del rene (TKV) valutato con la tomografia computerizzata spirale Lungo-termine (3 anni) Paragonare l’effetto dell’analogo della somatostatina long-lasting rispetto al placebo sulla perdita della funzione renale valutato attraverso misure seriali della clearance plasmatica dello ioexolo. |
|
E.2.2 | Secondary objectives of the trial |
To compare, among the two groups, absolute and relative changes in renal and liver volume parameters (at month 0, 12 and 36) as well as in functional and biochemical parameters by quarterly clinical visits during the 36 month follow-up. |
Paragonare nei due gruppi le variazioni assolute e relative dei parametri del volume del rene e del fegato (ai mesi 0, 12 e 36). |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Age > 18 years - Clinical and ultrasound diagnosis of ADPKD - Estimated GFR between 15 and 40 ml/min/1.73m2 (by the MDRD 4 variable equation) - Written informed consent |
- Eta' > 18 anni - Diagnosi clinica e radiografica di ADPKD - GFR stimato compreso tra 15 e 40 ml/min/1.73m2 - Consenso informato scritto |
|
E.4 | Principal exclusion criteria |
- 24-h Urinary protein excretion rate >3g (suggestive of a concomitant glomerular disease that could benefit of specific therapy) - Symptomatic urinary tract lithiasis or obstruction - Uncontrolled diabetes mellitus (HbA1c >8%) or hypertension (systolic/diastolic BP >180/110 mmHg) - Current urinary tract infection - Symptomatic biliary tract lithiasis - Active cancer - Psychiatric disorders or any condition that might prevent full comprehension of the purposes and risks of the study - Pregnancy, lactation or child bearing potential and ineffective contraception (estrogen therapy in post menopausal women should not be stopped) |
- Escrezione urinaria di proteine >3g/24 ore (che suggerisce la presenza di una malattia glomerulare concomitante che potrebbe beneficiare di una terapia specifica) - Litiasi o ostruzione del canale urinario sintomatiche - Diabete mellito non controllato (HbA1c >8%) o ipertensione (pressione sistolica/diastolica >180/110 mmHg) - Infezione delle vie urinarie in atto - Litiasi sintomatica del tratto biliare - Tumore - Disordini psichiatrici o qualsiasi condizione che possa impedire la completa comprensione da parte del paziente degli obiettivi e dei rischi dello studio - Gravidanza, allattamento, e utilizzo di un sistema contraccettivo non validato scientificamente (nelle donne in post-menopausa la terapia con estrogeni non deve essere interrotta) |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Short-term (1-year) Total kidney volume (TKV) change (delta TKV) as assessed by spiral computed tomography (spiral CT) scan. Long-term (3-years) Rate of GFR decline as assessed by serial measurements of the iohexol plasma clearance. |
Breve termine (1 anno): cambiamenti del volume totale del rene Lungo termine (3 anni): perdita della funzione renale |
|
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) | 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 | 5 |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 48 |
E.8.9.1 | In the Member State concerned days | 0 |