Paediatric patient who present a cystic fibrosis
Conditions
Interventions
Product Name: VEDROP
Product Code: VITAMIN E - TGPS
Pharmaceutical Form: Oral solution
INN or Proposed INN: d-a-tocopheryl PEG 1000 succinate
Concentration unit: mg/ml milligram(s)/millilitre
Concentr
Sponsors
ORPHAN
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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. ;Secondary Objective: 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.;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). | — |
Countries
France
Outcome results
None listed