E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Liver cirrhosis |
Levercirrose |
|
E.1.1.1 | Medical condition in easily understood language |
Replacement of normal liver tissue by fibrosis, scar tissue leading to the loss of liver function. |
Vervanging van normale lever weefsel door fibrose, litteken weefsel waardoor de lever zijn functie verliest. |
|
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 | 10024667 |
E.1.2 | Term | Liver 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 |
To evaluate the subsequent effects during 6 months of rhIGF-I treatment on body composition as measured by Dual-energy X-ray absorptiometry (DXA): muscle mass/non-fat-non-mineral mass, fat mass and bone mineral density. |
Het evalueren van de effecten van 6 maanden van rhIGF-I behandeling op de lichaamscompositie gemeten door DXA: spier massa/non-vet-non-mineraal massa, vet massa en bot mineraal densiteit. |
|
E.2.2 | Secondary objectives of the trial |
To assess the effects of stated treatment on (a) the liver function (bilirubin, albumin, total protein, liver enzymes, PT, APTT, antithrombin III, cholinesterase), (b) the respiratory quotient, (c) the resting energy metabolism and (d) the quality of life (Sickness Impact Profile). |
Het evalueren van de effecten van de genoemde behandeling op (a) de lever functie (bilirubine, albumine, totaal eiwit, lever enzymen, PT, APTT, antithrombine III, cholinesterase), (b) het respiratoir quotiënt, (c) het energie metabolisme in rust en (d) de kwaliteit van leven (Sickness Impact Profile). |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
18-70 year old.
Confirmed diagnosis of liver cirrhosis by laparoscopy, liver biopsy and/or clinical, biochemical and imaging studies.
Child-Pugh score 6 or more.
Plasma IGF-I level below the lower 2.5th percentile and adjusted for age (plasma IGF-I -2 SD Z-score).
Willing and able to give a written informed consent to participate in the study, including all study procedures and follow-up visits.
Alcohol abstinence for at least 3 months before entering the study.
Eligible for liver transplantation, and being put on the liver transplant waiting list.
|
18-70 jaar.
levercirrose.
Child Pugh score 6 of meer.
Plasma IGF-I level onder de lager 2.5e percentiel en aangepast op leeftijd (plasma IGF-I < -2 SD Z-score).
Het willen en het kunnen geven van een geinformeerde toestemming.
Alcohol onthouding voor tenminste 3 maanden alvorens entree tot de studie.
Goedgekeurd voor lever transplantatie en op de lijst voor een lever transplantatie. |
|
E.4 | Principal exclusion criteria |
Tense ascites requiring repeated paracenteses (two or more in the preceding year).
Severe peripheral edema.
Hospitalization for gastrointestinal bleeding, spontaneous bacterial peritonitis or other life-threatening complications within 3 months before entering the study.
Episode of encephalopathy requiring protein restriction, or hepatic encephalopathy that precludes participation.
Drug abuse. |
Ernstige ascites waarbij herhaalde paracenteses benodigd waren (2 of meer in de afgelopen jaren).
Ernstige perifere oedeem.
Ziekenhuisopname wegens gastrointestinale bloeding, spontane bacteriële peritonitis of andere levensbedreigend complicaties in de 3 maanden alvorens deelname aan de studie.
Episode van encefalopathie waarbij eiwit restrictie nodig was, of hepatische encefalopathie alvorens deelname.
Drugsmisbruik. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The main study endpoint is when 6 patients have been treated with rhIGF-I for 6 months. |
De studie eindigt wanneer 6 patienten 6 maanden zijn behandeld met rhIGF-I. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
No timepoints. We evaluate whether a patient is the last patient treated. |
Geen tijdpunt. We evalueren of een patiënt de laatste behandelde patiënt is. |
|
E.5.2 | Secondary end point(s) |
No secondary end point. |
Geen secundaire eindpunt. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Not applicable. |
Niet van toepassing. |
|
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 | No |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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 | No |
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 | 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
|
LVLS. |
Laatste bezoek van laatste patiënt. |
|
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 | 9 |
E.8.9.1 | In the Member State concerned days | 0 |