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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 19 March 2012
Main ID:  EUCTR2006-006693-24-FR
Date of registration: 16/05/2007
Prospective Registration: Yes
Primary sponsor: AOP Orphan Pharmaceuticals AG
Public title: A Randomized, Double-Blind, Placebo-Controlled, Dose-finding Study to Evaluate the Efficacy and Safety of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults with Cystic Fibrosis
Scientific title: A Randomized, Double-Blind, Placebo-Controlled, Dose-finding Study to Evaluate the Efficacy and Safety of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults with Cystic Fibrosis
Date of first enrolment: 23/08/2007
Target sample size: 160
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2006-006693-24
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no  
Phase: 
Countries of recruitment
Austria Czech Republic France Germany Hungary Italy Spain Sweden
Contacts
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Key inclusion & exclusion criteria
Inclusion criteria:
1. Male or female subjects of 12 years of age or older.
2. Body mass index equal or above -2 SDS (Standard Deviation Score-According to CDC standards) for 12-19 year old patients and =18.5 kg/m2 for adults 20 years old and older.
3. Have a confirmed diagnosis of cystic fibrosis: positive sweat chloride value ³ 60 mEq/l by quantitative pilocarpine iontophoresis (on at least 2 occasions, if no genotype was determined) and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype.
Measurement of nasal potential difference could be considered as an additional diagnostic tool, if results of sweat chloride test and genotype tests are not unambiguous. Nasal potential difference test can not replace any of the above tests and should not be interpreted in isolation.
4. Have Screening FEV1 between 50% and 85% of predicted.
5. Have oxygen saturation level measured by pulse oximetry (SpO2) > 90 % on room air.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Show bronchial hyperresponsiveness as known from previous visits e.g. the subject needs additional puffs of salbutamol (or other beta-2-agonists) more than twice daily. This also applies to subjects who have shown bronchoconstriction to previously administered inhalation therapies or a decrease of FEV1 = 20% during or after administration of placebo at the screening visit.
2. Have shown any evidence for unstable lung function, e.g. have had more than 15% FEV1 variation (difference between highest and lowest measured value in % of highest value measured) in the last 3 months before treatment starts (i.e. visit 2).
3. Have a pulmonary disease such as pneumonia, tuberculosis, or lung cancer.
4. Have had an acute upper respiratory tract infection within the last 2 weeks.
5. Have had an acute lower respiratory tract infection (requiring antibiotics or hospitalization) within the last 4 weeks.
6. Have had an pulmonary exacerbation within the last 4 weeks.
7. Have had any changes from routine maintenance therapy within the last 4 weeks.
8. Have any scheduled changes to inhaled antibiotics regimen during the course of the study.
9. Receive or are planned to receive any treatment via “on-off” regimen (e.g. Tobramycin – TOBI®)
10. Have received the last dose of any “on-off” treatment within the last 6 weeks.
11. Have any clinically significant liver, renal, cardiac (as defined by QTc >450 msec (or 0.450 seconds) for males or QTc> 470 msec (or 0.470 seconds) for females), neurological, or hematological disease.
12. Have allergic bronchopulmonary aspergillosis or colonization with Burkholderia cepacia.
13. Have poorly controlled diabetes mellitus.
14. Have smoked more than 3 cigarettes per day within the last 12 months.
15. Have a history of alcohol (> 40g/day) or drug abuse.
16. Have participated in an investigational drug study (including Moli1901) within the last 8 weeks.
17. Are women of child bearing potential and refuse to use effective contraception or are pregnant or lactating.
18. Are unwilling to perform appropriate safe contraception for at least 6 months after investigational product administration.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Cystic fibrosis is the most common fatal inherited disease in the Caucasian population, affecting about 4 in 10.000 children. In Cystic Fibrosis chloride transport accross the respiratory epithelium is deficient, so the mucus contains less water and its viscosity is abnormally increased. Moli 1901 corrects the abnormal transport of chloride thereby reducing the formation of mucus plugs and improving clearance.
Intervention(s)

Product Name: Moli 1901 (2622U90, duramycin)
Product Code: Moli 1901
Pharmaceutical Form: Inhalation vapour, solution
Pharmaceutical form of the placebo: Inhalation vapour, solution
Route of administration of the placebo: Inhalation use

Primary Outcome(s)
Main Objective: Study objectives are to establish minimum effective dose (MED), optimal dose, and maximum safe dose (MSD) in this dose-finding study with three different dosage schedules :
1. 2,5 mg/day (O.5 mg/ml;5ml) Moli 1901, daily
2. 2,5 mg/day (O.5 mg/ml;5ml) Moli 1901, every other day
3. 2,5 mg/day (O.5 mg/ml;5ml) Moli 1901, twice a week
Additionally, the tolerability of Moli 1901 shall be investigated.
Primary efficacy criterion (confirmatory evaluation) : the change in the percentage of the predicted FEV1 (forced expiratory volume in the first second) value.
Primary end point(s): Change in the percentage of the predicted FEV1 (Forced Expiratory Volume in first second) value.
Secondary Objective: Co-primary criterion (supportive evaluation) : the ensemble of two subject reported clinical scores, which are physical dimensions of the self-report, disease-specific quality of life test, the cystic fibrosis questionnaire in the revised version.
Secondary efficacy criteria :
The change in the percentage of the predicted FVC (Forced vital Capacity)
The change in the percentage of the predicted FEF25-75 (Forced Expiratory Flow 25-75% of FVC)
The change in the absolute values of FEV1 (Forced Expiratory Volume in first second)
Number of pulmanory exacerbations
Number of hospitalization days due to pulmonary exacerbation
Dose (cumulative dose) and duration of therapy needed during study participation to treat bronchial obstruction, infection or inflammation (i.e antibiotics, mucolytics, anti-inflammatory drugs)
Time to first pulmonary exacerbation
Change in oxigen saturation as measured with pulse oxymetry
Quality of Life Questionnaire (cystic fibrosis questionnaire-revised).
Secondary Outcome(s)
Secondary ID(s)
2006-006693-24-AT
Moli1901-010B
Source(s) of Monetary Support
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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