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

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

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.

Study Overview

• Status: Terminated
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: BIIL 283 BS (Amelubent)

• Study Type: Interventional
• Enrollment: 420
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Tucson, Arizona, United States, 85724-5073
      • University of Arizona
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • University of Arkansas for Medical Sciences
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital of Los Angeles
    • Palo Alto, California, United States, 94304-5786
      • Stanford University Medical Center
    • San Diego, California, United States, 92128-4284
      • Children's Hospital & Health Center
    • San Francisco, California, United States, 94143-0106
      • University of California at San Francisco
  • Colorado
    • Denver, Colorado, United States, 80262
      • University of Colorado
  • Florida
    • Orlando, Florida, United States, 32801
      • The Nemours Children's Clinic
    • St. Petersburg, Florida, United States, 33701
      • Pediatric Pulmonary Associates, PA
  • Illinois
    • Chicago, Illinois, United States, 60614
      • Children's Memorial Hospital
    • Maywood, Illinois, United States, 60153-3333
      • Loyola University Medical Center
  • Indiana
    • Indianapolis, Indiana, United States, 46202-5225
      • Riley Hospital
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals and Clinics
  • Kentucky
    • Lexington, Kentucky, United States, 40536-0284
      • University of Kentucky
  • Louisiana
    • New Orleans, Louisiana, United States, 70112
      • Tulane University
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
    • Boston, Massachusetts, United States, 02115
      • Children's Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109-0212
      • University of Michigan Health System
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota
  • Missouri
    • St. Louis, Missouri, United States, 63110
      • Washington University-St. Louis Children's Hospital
  • Nebraska
    • Omaha, Nebraska, United States, 68198-5190
      • University of Nebraska
  • New York
    • Albany, New York, United States, 12208
      • Albany Medical College
    • Rochester, New York, United States, 14642
      • University of Rochester
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599-7220
      • University of North Carolina
  • Ohio
    • Akron, Ohio, United States, 44308-1062
      • Children's Hospital Medical Center of Akron
    • Cincinnati, Ohio, United States, 45229-3039
      • Children's Hospital Medical Center
    • Cleveland, Ohio, United States, 44106
      • Rainbow Babies & Children's Hospital
    • Columbus, Ohio, United States, 45205
      • Columbus Children's Hospital
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19134
      • St. Christopher's Hospital for Children
    • Philadelphia, Pennsylvania, United States, 19129
      • MCP Hospital
    • Pittsburgh, Pennsylvania, United States, 15213
      • Children's Hospital of Pittsburgh
  • Tennessee
    • Nashville, Tennessee, United States, 37232-2586
      • Vanderbilt Children's Hospital
  • Texas
    • Dallas, Texas, United States, 75235
      • Children's Memorial Center of Dallas
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital
  • Wisconsin
    • Madison, Wisconsin, United States, 53792
      • University of Wisconsin Hospitals & Clinics

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Boehringer Ingelheim

Study record dates

Study Major Dates

• Study Start: May 1, 2003
• Primary Completion (Actual): July 1, 2004

Study Registration Dates

• First Submitted: May 13, 2003
• First Submitted That Met QC Criteria: May 13, 2003
• First Posted (Estimate): May 14, 2003

Study Record Updates

• Last Update Posted (Estimate): October 31, 2013
• Last Update Submitted That Met QC Criteria: October 30, 2013
• Last Verified: October 1, 2013

More Information

Terms related to this study

• Keywords: Cystic Fibrosis; BIIL 284; Boehringer Ingelheim
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: BI 543.45