E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Paediatric patient who present a cystic fibrosis |
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MedDRA Classification |
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 primary objective is to assess the biological efficacy of Vedrop in 30 paediatric patients with cystic fibrosis over a mid term exposure period of time, with assessments after 3 and 6 months of treatment. Patients will be monitored after 3 and 6 months of treatment, on their clinical status and on their tocopherolemia and lipidic status. |
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E.2.2 | Secondary objectives of the trial |
The secondary aim of the present study is to monitor the safety of Vedrop in these cystic fibrosis patients treated over this 6-month period. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patients should be less than or equal to 15 years old, i.e. up to their 16th birthday; - With written informed consent of both parents and, if considered receivable by the investigator, of the patient;
- Cystic fibrosis (CF) will be ascertained by: either by a sweat chloride value ≥ 60mEq/L (at least two tests are required);
or by a patient's genotype (if already available in the patient's medical file) with two identifiable mutations consistent with cystic fibrosis;
and confirmed by a comprehensive clinical assessment (detailed medical history and a complete physical examination) and laboratory investigations (haematological and blood chemistry tests, urinalysis), the results of which are within the normal range or clinically acceptable for this category of patients; - Patients with an exocrine pancreatic insufficiency defined by at least one functional pancreatic test. Pancreatic insufficiency could be defined by the existence of an obvious steatorrhea quantified by: - a fat loss equal to or more than 4 g/day and /or - a fat absorption below 93% in 3 day faecal fat balance and /or - a faecal elastase below 100 µg/g faeces.
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E.4 | Principal exclusion criteria |
- Who have a medical history which, in the judgement of the investigator, puts them 'at risk' or is likely to modify their handling of the study drug; - Who have a too poor clinical status to cope with a clinical investigation; - History of allergy or hypersensitivity reaction to the study drug or one of its constituents; - Absence of written informed consent by either parents or child; - Renal failure; - Patients who might be during the protocol duration or are already on a waiting list for lung or liver transplant; - Who present a meconial ileus with intestinal resection.
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E.5 End points |
E.5.1 | Primary end point(s) |
The end points are the biological efficacy and safety variables: -The patient clinical and biological status; -The patient tocopherolemia and lipidic status; -The patient incidence of treatment-emergent adverse events (TEAEs); -The patient incidence of serious adverse events (SAEs).
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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 | No |
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 | 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
| 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 | Information not present in EudraCT |
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 | |