Clinical trial NCT00060801: Efficacy and Safety of 24 Weeks of Oral Treatment With BIIL 284 BS in Adult and Pediatric 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
BIIL 284
Boehringer Ingelheim
Lead sponsor: Boehringer Ingelheim
Sponsor class: INDUSTRY
First posted: 2003-05-14
Enrollment: 420
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Change from baseline in post-bronchodilator forced expiratory volume in one second (FEV1) (percent predicted)
timeFrame: 28 weeks
measure: Proportion of patients with at least one pulmonary exacerbation during the treatment period as per definition of Fuchs et al
timeFrame: 28 weeks
Secondary outcomes:
measure: Change from baseline in post-bronchodilator forced vital capacity (FVC) percent predicted
timeFrame: 28 weeks
measure: Change from baseline in post-bronchodilator mean forced expiratory flow during the middle half of the FVC (FEF25-75% ) percent predicted
timeFrame: 28 weeks
measure: Change from baseline in post-bronchodilator maximal expiratory flow when 50% of FVC remains in lung (MEF50% )percent predicted
timeFrame: 28 weeks
measure: Change from baseline in post-bronchodilator maximal expiratory flow when 25% of FVC remains in lung (MEF25%) percent predicted
timeFrame: 28 weeks
measure: Change from baseline in post-bronchodilator inspiratory capacity (IC)
timeFrame: 28 weeks
measure: Change from baseline in post-bronchodilator slow vital capacity (SVC)
timeFrame: 28 weeks
measure: Change from baseline in pre-bronchodilator FEV1% predicted
timeFrame: week 12, 24 and 28
measure: Change from baseline in pre-bronchodilator FVC % predicted
timeFrame: week 12, 24 and 28
measure: Change from baseline in pre-bronchodilator FEF25-75% % predicted
timeFrame: week 12, 24 and 28
measure: Change from baseline in pre-bronchodilator MEF50% % predicted
timeFrame: week 12, 24 and 28
measure: Change from baseline in pre-bronchodilator MEF25%% predicted
timeFrame: week 12, 24 and 28
measure: Proportion of patients with at least one pulmonary exacerbation during the treatment period as described in Rosenfeld et al.
timeFrame: 28 weeks
measure: Time to first pulmonary exacerbation
timeFrame: 28 weeks
measure: Number of pulmonary exacerbations during the treatment period
timeFrame: 28 weeks
measure: Proportion of patients with at least 1 hospitalisation for a pulmonary exacerbation during the treatment period
timeFrame: 28 weeks
measure: Time to first hospitalisation for a pulmonary exacerbation
timeFrame: 28 weeks
measure: Number of hospitalisations for a pulmonary exacerbation
timeFrame: 28 weeks
measure: Number of days in hospital for a pulmonary exacerbation
timeFrame: 28 weeks
measure: Proportion of patients with at least one pulmonary exacerbation requiring i.v. antibiotics during the treatment period
timeFrame: 28 weeks
measure: Time to first course of i.v. antibiotics for a pulmonary exacerbation
timeFrame: 28 weeks
measure: Number of pulmonary exacerbations requiring i.v. antibiotics during the treatment period
timeFrame: 28 weeks
measure: Number of days of i.v. antibiotic use for pulmonary exacerbations during the treatment period
timeFrame: 28 weeks
measure: Change from baseline in weight
timeFrame: 28 weeks
measure: Change from baseline in height (in pediatrics)
timeFrame: 28 weeks
measure: Change from baseline in weight for age percentiles
timeFrame: 28 weeks
measure: Change from baseline in weight for age percentiles (in pediatrics)
timeFrame: 28 weeks
measure: Change from baseline in weight expressed as % ideal body weight (IBW)
timeFrame: 28 weeks
measure: Change from baseline in body mass index
timeFrame: 28 weeks
measure: Change from baseline in BMI for age percentiles
timeFrame: 28 weeks
measure: Change from baseline in blood levels of cytokines, chemokines and other inflammatory mediators
timeFrame: 28 weeks
measure: Change in patient's health status as reported by patient
timeFrame: 28 weeks
measure: Change in patient's health status as reported by physician
timeFrame: 28 weeks
Other outcomes:
Locations:
city: Tucson
country: United States
facility: University of Arizona
state: Arizona
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Palo Alto
country: United States
facility: Stanford University Medical Center
state: California
city: San Diego
country: United States
facility: Children's Hospital & Health Center
state: California
city: San Francisco
country: United States
facility: University of California at San Francisco
state: California
city: Denver
country: United States
facility: University of Colorado
state: Colorado
city: Orlando
country: United States
facility: The Nemours Children's Clinic
state: Florida
city: St. Petersburg
country: United States
facility: Pediatric Pulmonary Associates, PA
state: Florida
city: Chicago
country: United States
facility: Children's Memorial Hospital
state: Illinois
city: Maywood
country: United States
facility: Loyola University Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Lexington
country: United States
facility: University of Kentucky
state: Kentucky
city: New Orleans
country: United States
facility: Tulane University
state: Louisiana
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Health System
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: St. Louis
country: United States
facility: Washington University-St. Louis Children's Hospital
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska
state: Nebraska
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Akron
country: United States
facility: Children's Hospital Medical Center of Akron
state: Ohio
city: Cincinnati
country: United States
facility: Children's Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Rainbow Babies & Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: Columbus Children's Hospital
state: Ohio
city: Philadelphia
country: United States
facility: MCP Hospital
state: Pennsylvania
city: Philadelphia
country: United States
facility: St. Christopher's Hospital for Children
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt Children's Hospital
state: Tennessee
city: Dallas
country: United States
facility: Children's Memorial Center of Dallas
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Madison
country: United States
facility: University of Wisconsin Hospitals & Clinics
state: Wisconsin
Study officials:
References: