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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00737100




Registration number
NCT00737100
Ethics application status
Date submitted
15/08/2008
Date registered
18/08/2008
Date last updated
16/05/2014

Titles & IDs
Public title
Safety and Efficacy of 12-wk Treatment With Two Doses of Tiotropium Respimat in Cystic Fibrosis
Scientific title
A Randomized, Double-blind, Placebo-controlled Parallel Group Study to Investigate the Safety and Efficacy of Two Doses of Tiotropium Bromide (2.5 mcg and 5 mcg) Administered Once Daily Via the Respimat Device for 12 Weeks in Patients With Cystic Fibrosis.
Secondary ID [1] 0 0
2008-001156-43
Secondary ID [2] 0 0
205.339
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - Placebo Respimat
Treatment: Drugs - Tiotropium bromide 5 mcg
Treatment: Drugs - tiotropium bromide-low dose-2.5mcg

Experimental: Tiotropium Respimat 2.5 mcg - patient to receive low dose tiotropium once daily

Experimental: Tiotropium Respimat 5 mcg - patient to receive high dose tiotropium once daily

Placebo Comparator: Placebo Respimat - patient to receive placebo once daily


Treatment: Drugs: Placebo Respimat
patient to receive placebo matching active drug once daily

Treatment: Drugs: Tiotropium bromide 5 mcg
patient to recieve high dose tiotropium once daily

Treatment: Drugs: tiotropium bromide-low dose-2.5mcg
patient to receive low dose tiotropium once daily

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent Predicted FEV1 AUC0-4 Response at the End of Week 12 - Outcome measure description: Change from baseline in percent predicted Forced Expiratory Volume in one second (FEV1) Area Under the Curve from 0 to 4 hours (AUC0-4). Calculated as percent predicted at week 12 minus percent predicted at baseline.
Timepoint [1] 0 0
Baseline, Week 12
Primary outcome [2] 0 0
Percent Predicted FEV1 Trough Response at the End of Week 12 - Outcome measure description: Change from baseline in percent predicted trough Forced Expiratory Volume in one second. Calculated as percent predicted at week 12 minus percent predicted at baseline.
Timepoint [2] 0 0
Baseline, Week 12
Secondary outcome [1] 0 0
Percent Predicted FVC AUC0-4 Response at the End of Week 12 - Change from baseline in percent predicted Forced Vital Capacity (FVC) Area Under the Curve from 0 to 4 hours (AUC0-4). Calculated as percent predicted at week 12 minus percent predicted at baseline.
Timepoint [1] 0 0
Baseline, Week 12
Secondary outcome [2] 0 0
Percent Predicted FVC Trough Response at the End of Week 12 - Change from baseline in percent predicted trough Forced Vital Capacity (FVC). Calculated as percent predicted at week 12 minus percent predicted at baseline.
Timepoint [2] 0 0
Baseline, Week 12
Secondary outcome [3] 0 0
Pre-bronchodilator FEF25-75 Percent Predicted at the End of Week 12 - Forced Expiratory Flow at 25-75% of vital capacity (FEF25-75). Calculated as percent predicted at week 12 minus percent predicted at baseline.
Timepoint [3] 0 0
Baseline, Week 12
Secondary outcome [4] 0 0
Change From Baseline in Residual Volume/Total Lung Capacity (RV/TLC) at the End of Week 12 - Change from baseline in static lung hyperinflation as measured by RV/TLC. Calculated as percent predicted at week 12 minus percent predicted at baseline.
Timepoint [4] 0 0
Baseline, Week 12
Secondary outcome [5] 0 0
Respiratory and Systemic Symptoms Questionnaire (RSSQ) - Outcome measure description: The RSSQ questionnaire is used to determine the presence or absence of an exacerbation during the recall period.
Timepoint [5] 0 0
12 weeks
Secondary outcome [6] 0 0
Change From Baseline in CFQ Scores - Adult Group - The Cystic Fibrosis questionnaire (CFQ) is a disease-specific instrument that measures health-related quality of life (HRQOL) for adults with CF. This validation questionnaire consists of 50 items on generic and disease-specific scales. The scores range from 0 to 100, with higher scores indicating better health.
Timepoint [6] 0 0
12 weeks
Secondary outcome [7] 0 0
Change From Baseline in CFQ Scores - Adolescents Group - The Cystic Fibrosis questionnaire (CFQ) is a disease-specific instrument that measures health-related quality of life (HRQOL) for adolescents (age 6-13) with CF. This validation questionnaire consists of 50 items on generic and disease-specific scales. The scores range from 0 to 100, with higher scores indicating better health.
Timepoint [7] 0 0
12 weeks
Secondary outcome [8] 0 0
Change From Baseline in CFQ Scores - Parent Questionnaire - The Cystic Fibrosis questionnaire (CFQ) is a disease-specific instrument that measures health-related quality of life (HRQOL) for adolescents with CF - parent questionnaire. This validation questionnaire consists of 50 items on generic and disease-specific scales. The scores range from 0 to 100, with higher scores indicating better health.
Timepoint [8] 0 0
12 weeks
Secondary outcome [9] 0 0
Amount of Tiotropium Eliminated in Urine From 0 to 4 Hours at Steady State (Ae0-4,ss) - Ae0-4,ss represents the amount of tiotropium that is eliminated in urine from time 0 to 4 hours at steady state
Timepoint [9] 0 0
pre-dose, and 5 minutes (min), 20 min, 1 hour (h), and 2 h post-dose
Secondary outcome [10] 0 0
Maximum Measured Concentration at Steady State (Cmax,ss) - Cmax,ss represents the maximum measured concentration of tiotropium in plasma at steady state.
Timepoint [10] 0 0
pre-dose, and 5 minutes (min), 20 min, 1 hour (h), and 2 h post-dose
Secondary outcome [11] 0 0
Time From Dosing to the Maximum Concentration (Tmax,ss) - Tmax,ss represents the time from dosing to the maximum concentration of tiotropium in plasma
Timepoint [11] 0 0
pre-dose, and 5 minutes (min), 20 min, 1 hour (h), and 2 h post-dose
Secondary outcome [12] 0 0
Clinical Relevant Abnormalities for Vital Signs and Laboratory Evaluation - Clinical Relevant Abnormalities for Vital Signs and Laboratory evaluation. Any new or clinically relevant worsening of baseline conditions was reported as Adverse Event.
Timepoint [12] 0 0
From first drug administration until 30 days after last drug administration (up to 121 days)

Eligibility
Key inclusion criteria
Inclusion criteria:

1. Male or female patients

2. Diagnosis of Cystic Fibrosis (positive sweat chloride test or two identifiable
mutations)

3. Pre-bronchodilator FEV1 greater/equal 25% of predicted values
Minimum age
No limit
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:

1. Significant history of allergy/hypersensitivity

2. Hypersensitivity to study drug

3. Participation in another trial

4. Female patients who are pregnant or lactating

5. Female patients of childbearing potential

6. Patients who have started a new medication for CF within 4 weeks of screening

7. Patients with known substance abuse

8. Clinically significant disease other than CF

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,WA
Recruitment hospital [1] 0 0
205.339.100 Boehringer Ingelheim Investigational Site - Westmead
Recruitment hospital [2] 0 0
205.339.101 Boehringer Ingelheim Investigational Site - Westmead
Recruitment hospital [3] 0 0
205.339.103 Boehringer Ingelheim Investigational Site - Adelaide
Recruitment hospital [4] 0 0
205.339.104 Boehringer Ingelheim Investigational Site - Subiaco
Recruitment postcode(s) [1] 0 0
- Westmead
Recruitment postcode(s) [2] 0 0
- Adelaide
Recruitment postcode(s) [3] 0 0
- Subiaco
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Iowa
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
New Hampshire
Country [8] 0 0
United States of America
State/province [8] 0 0
New Jersey
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Oklahoma
Country [12] 0 0
United States of America
State/province [12] 0 0
South Carolina
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Utah
Country [15] 0 0
United States of America
State/province [15] 0 0
Vermont
Country [16] 0 0
United States of America
State/province [16] 0 0
Virginia
Country [17] 0 0
United States of America
State/province [17] 0 0
Wisconsin
Country [18] 0 0
Belgium
State/province [18] 0 0
Bruxelles
Country [19] 0 0
Belgium
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Jette
Country [20] 0 0
Belgium
State/province [20] 0 0
Leuven
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France
State/province [21] 0 0
Amiens
Country [22] 0 0
France
State/province [22] 0 0
Angers
Country [23] 0 0
France
State/province [23] 0 0
BRON Cedex
Country [24] 0 0
France
State/province [24] 0 0
Lille Cedex
Country [25] 0 0
France
State/province [25] 0 0
Lille
Country [26] 0 0
France
State/province [26] 0 0
Lisieux
Country [27] 0 0
France
State/province [27] 0 0
Montpellier
Country [28] 0 0
France
State/province [28] 0 0
Nantes
Country [29] 0 0
France
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Paris Cedex 14
Country [30] 0 0
France
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Paris
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France
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Rennes
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France
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Roscoff Cedex
Country [33] 0 0
France
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Rouen cedex
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France
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Vandoeuvre les Nancy
Country [35] 0 0
France
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Vannes
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Germany
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Erlangen
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Germany
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Frankfurt/Main
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Germany
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Frankfurt
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Germany
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Freiburg
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Germany
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Gerlingen
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Germany
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Hamburg
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Germany
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Hannover
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Germany
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Heidelberg
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Germany
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München
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Germany
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Tübingen
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Italy
State/province [46] 0 0
Ancona
Country [47] 0 0
Italy
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Firenze
Country [48] 0 0
Italy
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Genova
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Netherlands
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Groesbeek
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Netherlands
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Rotterdam
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New Zealand
State/province [51] 0 0
Grafton / Auckland
Country [52] 0 0
New Zealand
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Hamilton
Country [53] 0 0
Portugal
State/province [53] 0 0
Lisboa
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Portugal
State/province [54] 0 0
Porto
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Russian Federation
State/province [55] 0 0
Moscow
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Russian Federation
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Rostov-on-Don
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Russian Federation
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St. Petersburg
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Russian Federation
State/province [58] 0 0
Voronezh
Country [59] 0 0
Russian Federation
State/province [59] 0 0
Yaroslavl
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Belfast
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United Kingdom
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Birmingham
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United Kingdom
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Boston
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United Kingdom
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Leeds
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United Kingdom
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Lincoln
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United Kingdom
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Liverpool
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United Kingdom
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Nottingham
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United Kingdom
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Oxford
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United Kingdom
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Plymouth
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United Kingdom
State/province [69] 0 0
Sheffield
Country [70] 0 0
United Kingdom
State/province [70] 0 0
Wolverhampton

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Boehringer Ingelheim
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study evaluates the effects of 12-week treatment with two doses of tiotropium bromide
(2.5 mcg q.d. and 5 mcg q.d.) compared to placebo administered via the Respimat device on
lung function in patients with Cystic Fibrosis. The selection of the optimal dose will be
based on bronchodilator efficacy, safety evaluations and pharmacokinetic evaluations
Trial website
https://clinicaltrials.gov/show/NCT00737100
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Boehringer Ingelheim
Address 0 0
Boehringer Ingelheim
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications