E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Bile acid malabsorption / bile acid diarrhoea |
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E.1.1.1 | Medical condition in easily understood language |
Bile acid diarrhoea is a chronic dissease causing watery diarrhoea, due to bile acids escaping fromt he small intestine to the large intestine, causing chronic watery diarrhoea. |
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E.1.1.2 | Therapeutic area | Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Diagnosis [E01] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10069703 |
E.1.2 | Term | Bile acid malabsorption |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066557 |
E.1.2 | Term | Chronic diarrhoea |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
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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 |
Validation of stimulated ∆FGF19 for diagnosis of BAD in patients referred to SeHCAT |
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E.2.2 | Secondary objectives of the trial |
correlation of ∆FGF19 to fasting 7α-hydroxy-4-cholesten-3-on (C4). Correlation of all the above to diarheoa. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
referral to SeHCAT for investigation of chronic watery diarrhoea |
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E.4 | Principal exclusion criteria |
Treatment with sequestrants or constipants/laxatives within one week before the SeHCAT. Pregnancy. Small bowel resection. Untreated active small bowel disease such as Crohns’ disease. Age < 18 years or > 80 years. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Negative predictive value of stimulated ∆FGF19 >2.5pg/mL for detecting BAD defined by SeHCAT < 10%. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
after inclusion is completed. |
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E.5.2 | Secondary end point(s) |
• Positive predictive value of stimulated ∆FGF19 >2.5pg/mL for detecting BAD defined by SeHCAT < 10%. • Negative and positive predictive value of fasting C4 >5.5ng/mL for detecting BAD defined by SeHCAT < 10% • ROC curve analysis for stimulated ∆FGF19 >2.5pg/mL for detecting BAD defined by SeHCAT < 5%. • ROC curve analysis for fasting C4 >5.5ng/mL for detecting BAD as compared to SeHCAT < 5% • Correlation between value of stimulated ∆FGF19 and diarrhoea (Bristol scale and Hjortswang criteria) • Correlation between value of fasting C4 and diarrhoea (Bristol scale and Hjortswang criteria) • Correlation between diarrhoea with positive Hjortswang criteria and SHS and HRQOL. • Median stimulated ∆FGF19 and fasting C4 in SeHCAT stratum: 0-5%, >5-10%, >10-15%, > 15%.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
after inclusion is completed and through the data analysis |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
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 | 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
| 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 | 6 |
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
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |