STUDY OF THE LONG TERM BIOLOGICAL EFFICACY AND TOLERABILITY OF VEDROP (VITAMIN E-TPGS FORMULATION) AFTER DAILY ORAL ADMINISTRATION IN 30 PAEDIATRIC PATIENTS WITH CYSTIC FIBROSIS - 1ORP2

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2007-001007-38-FR
• Date of registration: 26/03/2007
• Prospective Registration: Yes
• Primary sponsor: ORPHAN
• Public title: STUDY OF THE LONG TERM BIOLOGICAL EFFICACY AND TOLERABILITY OF VEDROP (VITAMIN E-TPGS FORMULATION) AFTER DAILY ORAL ADMINISTRATION IN 30 PAEDIATRIC PATIENTS WITH CYSTIC FIBROSIS - 1ORP2
• Scientific title: STUDY OF THE LONG TERM BIOLOGICAL EFFICACY AND TOLERABILITY OF VEDROP (VITAMIN E-TPGS FORMULATION) AFTER DAILY ORAL ADMINISTRATION IN 30 PAEDIATRIC PATIENTS WITH CYSTIC FIBROSIS - 1ORP2
• Date of first enrolment: 25/04/2007
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-001007-38
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

France

Key inclusion & exclusion 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:
- 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.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Paediatric patient who present a cystic fibrosis

Intervention(s)

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
Concentration type: equal
Concentration number: 50-

Primary Outcome(s)

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.

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).

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.

Secondary ID(s)

VITAMIN E VEDROP/06/LONGTERM/FR