E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Short Bowel Syndrome with intestinal failure (SBS IF) |
Korttarmssyndrom med intestinal svigt |
|
E.1.1.1 | Medical condition in easily understood language |
Short Bowel Syndrome with intestinal failure (SBS IF) |
Korttarmssyndrom med intestinal svigt |
|
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 | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10022683 |
E.1.2 | Term | Intestinal malabsorption |
E.1.2 | System Organ Class | 100000004856 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10036430 |
E.1.2 | Term | Post surgical malabsorption NOS |
E.1.2 | System Organ Class | 100000004856 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10042822 |
E.1.2 | Term | Syndrome malabsorption |
E.1.2 | System Organ Class | 100000004856 |
|
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 |
The objective of the trial is to assessthe therapeutic effect of the di-peptidyl-peptidase 4 inhibitor, Sitagliptin (Januvia®) for SBS patients with colon in function. The efficacy is assessed through the ability of sitagliptin (Januvia®) to increase intestinal absorption, reduce diarrhea and the need for hyperphagia. |
Projektet har til formål at undersøge effekten af di-peptidyl-peptidase 4 hæmmeren, Sitagliptin (Januvia®), på patienter med korttarmssyndrom og colon i funktion. Der fokuseres på sitagliptins (Januvia®) evne til at øge intestinal absorption, reducere patientens diarré og behov for hyperfagi. |
|
E.2.2 | Secondary objectives of the trial |
To test possible biomarkers for determination of intestinal adaptation, p-citrulline, p-paracetamol absorptions test are performed. |
For at teste mulige biomarkører til bestemmelse af intestinal tilpasning udføres p-citrulline, p-paracetamol absorptionstest. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients with intestinal failure and short bowel syndrome and colon in continuation and function and fecal output ≥ 1,5 kg Age 18 to 90 years |
Patienter med korttarmssyndrom og tarmsvigt med colon i funktion/kontinuitet samt fæces output ≥ 1,5 kg. Alder ≥ 18 år og ≤ 90 år
|
|
E.4 | Principal exclusion criteria |
Pregnant, disabled and patients with short bowel syndrome and stomi. |
Gravid, handicappede og patienter med korttarmssyndrom og stomi. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Faecal weight/diarrhea |
Fæces vægt/diare |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Before and at the end of 8 weeks of treatment with Januvia |
Inden og efter 8 ugers behandling med januvia |
|
E.5.2 | Secondary end point(s) |
Change in: urinary volume oral intake parenteral volume support Fluid composite effect Body weight Body composition (DEXA) Mesenteric blood flow |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Before and at the end of 8 weeks of treatment with Januvia |
|
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 | Yes |
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 | No |
E.8.1.1 | Randomised | No |
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 | 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 | 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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | |