E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic Fibrosis-Related Pancreatic Insufficiency and Fat Malabsorption |
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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 |
The objectives of this study are to evaluate preliminary safety, palatability and efficacy of pancrelipase microtablets to improve steatorrhoea in infants and toddlers with cystic fibrosis (CF). |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1. Male and/or female infants/toddlers 6 to 24 months of age. 2. Diagnosis of CF confirmed by genetic testing including chromosomal analysis and/or clinical criteria including abnormal results of pilocarpine iontophoresis sweat testing consistent with diagnosis of CF. 3. Historical documentation of abnormal coefficient of fat absorption (COA) 4. Subject’s parent or legal guardian must sign informed consent. 5. Stable patient requiring enzyme therapy for management of steatorrhoea in subjects with defined diagnosis as set forth by criteria 2 and 3 6. Historical documentation of abnormal coefficient of fat absorption (COA) or presence of <15 micrograms fecal elastase/gram stool consistent with diagnosis of severe pancreatic insufficiency. |
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E.4 | Principal exclusion criteria |
1. Stable antibiotic therapy for small bowel overgrowth or chronic pulmonary infection. 2. Hypersensitivity to porcine products. 3. Use of prokinetics including metoclopramide or cisapride. 4. Concurrent nasogastric tube feeding for supplemental enteral nutrition. 5. Concomitant steroid therapy. 6. Exacerbation of chronic lung infections. 7. Use of concomitant H2 blockers or proton pump inhibitors as concomitant therapy. 8. Use of herbal supplements. 9. Clinically significant vomiting, malnutrition, or severe dehydration. 10. Severe constipation and intestinal resection. 11. Uncorrected electrolyte disorders (such as hypokalaemia, hypocalcaemia, hypomagnesaemia). 12. Known chromosome abnormality or congenital anomalies of the gastrointestinal tract, heart or liver; including gastrointestinal tract abnormalities. 13. Clinically significant disease that could interfere with the adequate assessment of the study drug. 14. HIV infection. 15. History of drug sensitivity to any of the study medication ingredients; 16. Subject in any other investigational drug study within the previous 30 days of consent. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoints will be:
• the change in coefficient of fecal fat absorption (COA) measured from baseline to the end of study period. • assessment of daily palatability using a 4-point scale. • assessment of percentage of CO2 expired by a 13C-medium chain triglyceride breath test as a measure of exogenous lipase activity and as a surrogate marker of lipase activity and pharmacodynamic effect of pancreatic enzyme therapy. |
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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 | 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 | Yes |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.6.13.1 | Other scope of the trial description |
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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 | Yes |
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 | Yes |
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.8.5 | The trial involves multiple Member States | Yes |
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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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
|
End of study = Visit 3 (last patient out) |
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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 | |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 3 |