Clinical trial NCT01818544: Safety and Efficacy of Oral BAY85-8501 in Patients With Non-CF (Cystic Fibrosis) Bronchiectasis
Official title: A Phase IIa, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety and Efficacy of 28 Day Oral Administration of BAY85-8501 in Patients With Non-Cystic Fibrosis Bronchiectasis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Bronchiectasis
Keywords: Elastase Inhibition
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2013-03-26
Enrollment: 94
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to assess the safety and tolerability of 28 day oral administration of BAY85-8501 versus placebo in subjects with non-CF Bronchiectasis (BE).

The secondary objectives are to examine the effect of BAY85-8501 on pulmonary function, biomarkers of inflammation and tissue damage, and the impact on overall health and perceived well-being and to evaluate the pharmacokinetics of BAY85-8501.


Eligibility criteria: Inclusion Criteria:

* Proven and documented diagnosis of non-CF (cystic fibrosis) idiopathic or post-infectious BE (bronchiectasis) by computed tomography (CT) scan \[conventional high resolution CT is considered the standard\], including 2 or more lobes and dilated airways compatible with BE at initial diagnosis
* Stable pulmonary status as indicated by the forced, expired volume in 1 second (FEV1) percent predicted ≥30% and \<90% (post-bronchodilator)
* Stable (i.e., no dose change) regimen of standard BE treatment administered at least for 4 weeks prior to screening
* Cough on most days

Exclusion Criteria:

* Forced, expired volume in 1 second \<30% or ≥90% predicted (post-bronchodilator)
* Recent significant hemoptysis (≥300 mL or requiring blood transfusion) in the preceding 4 weeks before screening (and during the screening period)
* Known cystic fibrosis and/or documented chronic bronchial asthma
* Active allergic bronchopulmonary aspergillosis (ABPA)
* Diagnosis of common variable immunodeficiency (CVID)
* Systemic or inhaled antibiotic treatment within 4 weeks prior to screening
* Treatment of an exacerbation within 4 weeks prior to screening
* Systemic corticosteroids at \>10 mg/day prednisolone equivalent for \>2 weeks within 4 weeks prior to screening

Primary outcomes:
measure: Number Of Subjects Who Need To Discontinue Study Medication Due To Findings In Physical Examination
timeFrame: From start of study treatment up to follow up visit (28 days after last dose)
measure: Change From Baseline in Systolic Blood Pressure At Days 7, 14, 21, 28, 56
timeFrame: Baseline (Day 1), Day 7, 14, 21, 28, 56
measure: Change From Baseline in Diastolic Blood Pressure At Days 7, 14, 21, 28, 56
timeFrame: Baseline (Day 1), Day 7, 14, 21, 28, 56
measure: Change From Baseline in Heart Rate At Days 7, 14, 21, 28, 56
timeFrame: Baseline (Day 1), Day 7, 14, 21, 28, 56
measure: Number of Subjects With new Abnormal (Pathologic) Electrocardiogram (ECG) Findings From Baseline to Day 28
timeFrame: Baseline (Day 1), Day 7, 14, 21, 28
measure: Number of Subjects who Show Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Total Bilirubin (TB) Abnormalities in Their Safety Lab Assessment
timeFrame: Baseline (Day 1), Day 7, 14, 21, 28, 56
measure: Number of Subjects With Drug Related Adverse Events as a Measure of Safety And Tolerability
timeFrame: Baseline (Day 1), Day 7, 14, 21, 28, 56
Secondary outcomes:
measure: Change From Baseline in Pulmonary Function Test Forced Expired Volume in 1 Second (FEV1) At Days 7, 14, 21, 28, 56
timeFrame: Baseline (Day 1), Day 7, 14, 21, 28, 56
measure: Change From Baseline in Pulmonary Function Test Forced Vital Capacity (FVC) at Days 7, 14, 21, 28, 56
timeFrame: Baseline (Day 1), Day 7, 14, 21, 28, 56
measure: Change From Baseline in Pulmonary Function Test Forced Expiratory Flow Over the Middle Half of Subject's Forced Vital Capacity (FVC) (FEF2575) at Days 7, 14, 21, 28, 56
timeFrame: Baseline (Day 1), Day 7, 14, 21, 28, 56
measure: Change From Baseline in Total Score on St. George's Respiratory Questionnaire (SGRQ) at Day 28 and 56
timeFrame: Baseline (Day 1), Day 28 and 56
measure: Change From Baseline in 24 Hours Sputum Weight at Day 28
timeFrame: Baseline (Day 1), Day 28
measure: Change From Baseline of Biomarkers in Sputum at Days 14, 28, 56: Alpha1 Antitrypsin Human Neutrophil Elastase (A1AHNE) Complex, Interleukin-8(IL-8)
timeFrame: Baseline (Day 1), Day 14, 28 and 56
measure: Change From Baseline of Biomarkers in Sputum at Days 14, 28, 56: Neutrophil cell Count
timeFrame: Baseline (Day 1), Day 14, 28 and 56
measure: Change From Baseline of Human Neutrophil Elastase (NE) Activity in Sputum at Days 14, 28, 56
timeFrame: Baseline (Day 1), Day 14, 28 and 56
measure: Change From Baseline of Human Neutrophil Elastase (NE) Concentration in Sputum at Days 14, 28, 56
timeFrame: Baseline (Day 1), Day 14, 28 and 56
measure: Change From Baseline of Biomarkers in Blood at Days 14, 28, 56: C-reactive Protein
timeFrame: Baseline (Day 1), Day 14, 28 and 56
measure: Change From Baseline of Biomarkers in Blood at Days 14, 28, 56: Interleukin-8 (IL8)
timeFrame: Baseline (Day 1), Day 14, 28 and 56
measure: Change From Baseline of Biomarkers in Blood at Days 14, 28, 56: Neutrophil cell Count
timeFrame: Baseline (Day 1), Day 14, 28 and 56
measure: Change From Baseline of Biomarkers in Urine At Days 14, 28, 56: Creatinine
timeFrame: Baseline (Day 1), Day 14, 28 and 56
measure: Change From Baseline of Biomarkers in Urine at Days 14, 28, 56: Desmosine
timeFrame: Baseline (Day 1), Day 14, 28 and 56
measure: Change From Baseline of Biomarkers in Urine at Days 14, 28, 56: Normalized Desmosine Value to Creatinine
timeFrame: Baseline (Day 1), Day 14, 28 and 56
Other outcomes:
Locations:
city: Nice
country: France
city: Perpignan
country: France
city: Donaustauf
country: Germany
state: Bayern
city: Rüdersdorf
country: Germany
state: Brandenburg
city: Hannover
country: Germany
state: Niedersachsen
city: Leipzig
country: Germany
state: Sachsen
city: Großhansdorf
country: Germany
state: Schleswig-Holstein
city: Kiel
country: Germany
state: Schleswig-Holstein
city: Lübeck
country: Germany
state: Schleswig-Holstein
city: Jena
country: Germany
state: Thüringen
city: Hamburg
country: Germany
city: Orbassano
country: Italy
state: Torino
city: Genova
country: Italy
city: Milano
country: Italy
city: Napoli
country: Italy
city: Padova
country: Italy
city: Siena
country: Italy
city: Badalona
country: Spain
state: Barcelona
city: Salt
country: Spain
state: Girona
city: Majadahonda
country: Spain
state: Madrid
city: Madrid
country: Spain
city: Southampton
country: United Kingdom
state: Hampshire
city: Cottingham
country: United Kingdom
state: Humberside
city: Leicester
country: United Kingdom
state: Leicestershire
city: Liverpool
country: United Kingdom
state: Merseyside
city: Sheffield
country: United Kingdom
state: South Yorkshire
city: Dundee
country: United Kingdom
state: Tayside
city: Newcastle Upon Tyne
country: United Kingdom
state: Tyne and Wear
city: South Shields
country: United Kingdom
state: Tyne and Wear
city: Wolverhampton
country: United Kingdom
state: West Midlands
city: Bradford
country: United Kingdom
state: West Yorkshire
city: Glasgow
country: United Kingdom
city: London
country: United Kingdom
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References: