Efficacy and Safety of 24 Weeks of Oral Treatment With BIIL 284 BS in Adult and Pediatric Patients

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to determine the effect of 24 weeks of treatment with BIIL 284 BS compared with placebo on pulmonary function and incidence of pulmonary exacerbation in adult and pediatric cystic fibrosis patients.

Official Title
-----------------
A Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of 24 Weeks of Oral Treatment With BIIL 284 BS in Adult (75 mg, 150 mg) and Pediatric (75 mg) Cystic Fibrosis Patients

Conditions
-----------------
Cystic Fibrosis

Intervention / Treatment
-----------------
* Drug: BIIL 283 BS (Amelubent)


Participation Criteria
=================
Eligibility Criteria
-----------------
INCLUSION CRITERIA: Male or female patients >= 6 years pediatric 6-17 years inclusive; adult >= 18 years) Body weight >= 20 kg (determined at Visit 1) Confirmed diagnosis of CF Able to perform acceptable spirometric maneuvers, according to American Thoracic Society standards . FEV1 25-85% predicted Clinically stable The patient or the patient's legally acceptable representative must be able to give informed consent. The patient must be able to swallow the BIIL 284 BS tablets whole. Patients taking a chronic medication must be willing to continue this therapy for the entire duration of the study. EXCLUSION CRITERIA: Patients with a significant history of allergy/hypersensitivity (including medication allergy) which is deemed relevant to the trial as judged by the Investigator. Relevance in this context refers to any increased risk of hypersensitivity reaction to trial medication; there are no specific issues of concern currently identified with respect to use of BIIL 284 BS in allergic patients per se. Patients who have participated in another study with an Investigational drug within one month or 6 half-lives (whichever is greater) preceding the screening visit. Patients with known relevant substance abuse, including alcohol or drug abuse. Female patients who are pregnant or lactating, including females who have a positive serum pregnancy test at screening (pregnancy tests will be performed for all females of child bearing potential). Female patients of child bearing potential who are not using a medically approved form of contraception. Patients who are unable to comply with food requirements prior to dosing. Patients with documented persistent colonization with Burkholderia cepacia. Patients chronically using oral corticosteroids or high-dose ibuprofen. Patients with hemoglobin < 9.0 g/dL; platelets < 100x10 to the 9th power/L; SGOT (ALT) or SGPT (AST) > 2.5 times the upper limit of normal; creatinine > 1.5 times upper limit normal. Clinically significant disease or medical condition other than Cystic Fibrosis or Cystic Fibrosis-related conditions that, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data.

Ages Eligible for Study
-----------------
Minimum Age: 6 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: BIIL 283 BS (Amelubent)|nan|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in post-bronchodilator forced expiratory volume in one second (FEV1) (percent predicted) |  | 28 weeks | 
| Proportion of patients with at least one pulmonary exacerbation during the treatment period as per definition of Fuchs et al |  | 28 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in post-bronchodilator forced vital capacity (FVC) percent predicted |  | 28 weeks | 
| Change from baseline in post-bronchodilator mean forced expiratory flow during the middle half of the FVC (FEF25-75% ) percent predicted |  | 28 weeks | 
| Change from baseline in post-bronchodilator maximal expiratory flow when 50% of FVC remains in lung (MEF50% )percent predicted |  | 28 weeks | 
| Change from baseline in post-bronchodilator maximal expiratory flow when 25% of FVC remains in lung (MEF25%) percent predicted |  | 28 weeks | 
| Change from baseline in post-bronchodilator inspiratory capacity (IC) |  | 28 weeks | 
| Change from baseline in post-bronchodilator slow vital capacity (SVC) |  | 28 weeks | 
| Change from baseline in pre-bronchodilator FEV1% predicted |  | week 12, 24 and 28 | 
| Change from baseline in pre-bronchodilator FVC % predicted |  | week 12, 24 and 28 | 
| Change from baseline in pre-bronchodilator FEF25-75% % predicted |  | week 12, 24 and 28 | 
| Change from baseline in pre-bronchodilator MEF50% % predicted |  | week 12, 24 and 28 | 
| Change from baseline in pre-bronchodilator MEF25%% predicted |  | week 12, 24 and 28 | 
| Proportion of patients with at least one pulmonary exacerbation during the treatment period as described in Rosenfeld et al. |  | 28 weeks | 
| Time to first pulmonary exacerbation |  | 28 weeks | 
| Number of pulmonary exacerbations during the treatment period |  | 28 weeks | 
| Proportion of patients with at least 1 hospitalisation for a pulmonary exacerbation during the treatment period |  | 28 weeks | 
| Time to first hospitalisation for a pulmonary exacerbation |  | 28 weeks | 
| Number of hospitalisations for a pulmonary exacerbation |  | 28 weeks | 
| Number of days in hospital for a pulmonary exacerbation |  | 28 weeks | 
| Proportion of patients with at least one pulmonary exacerbation requiring i.v. antibiotics during the treatment period |  | 28 weeks | 
| Time to first course of i.v. antibiotics for a pulmonary exacerbation |  | 28 weeks | 
| Number of pulmonary exacerbations requiring i.v. antibiotics during the treatment period |  | 28 weeks | 
| Number of days of i.v. antibiotic use for pulmonary exacerbations during the treatment period |  | 28 weeks | 
| Change from baseline in weight |  | 28 weeks | 
| Change from baseline in height (in pediatrics) |  | 28 weeks | 
| Change from baseline in weight for age percentiles |  | 28 weeks | 
| Change from baseline in weight for age percentiles (in pediatrics) |  | 28 weeks | 
| Change from baseline in weight expressed as % ideal body weight (IBW) |  | 28 weeks | 
| Change from baseline in body mass index |  | 28 weeks | 
| Change from baseline in BMI for age percentiles |  | 28 weeks | 
| Change from baseline in blood levels of cytokines, chemokines and other inflammatory mediators |  | 28 weeks | 
| Change in patient's health status as reported by patient |  | 28 weeks | 
| Change in patient's health status as reported by physician |  | 28 weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Cystic Fibrosis, BIIL 284, Boehringer Ingelheim