Clinical trial NCT02248922: Evaluation of Lung Clearance Index in Cystic Fibrosis (CF) Patients, Infected With P.Aeruginosa
Official title: An 8 Week Open-label Interventional Multicenter Study to Evaluate the Lung Clearance Index as Endpoint for Clinical Trials in Cystic Fibrosis Patients ≥ 6 Years of Age, Chronically Infected With Pseudomonas Aeruginosa
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Chronic Lung Infection With Pseudomonas Aeruginosa in Cystic Fibrosis
Keywords: Cystic Fibrosis, Pseudomonas aeruginosa, Lung Clearance Index, Multiple Breath Washout, Inhaled Tobramycin, Inhaled Antibiotics.
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2014-09-25
Enrollment: 17
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

Brief summary: The purpose of the study was to evaluate lung clearance index (LCI) by a standardized procedure in a well characterized study setting and to assess feasibility of LCI as a more sensitive method than forced expiratory volume at 1 second (FEV1) to measure effectiveness of antibiotic therapy in patients with CF aged 6 years and older with mild to moderate lung disease.

Detailed description: This study was terminated prematurely. The reason for trial termination was challenge with enrollment and patient recruitment. A significant decrease in the eligible patient population had been identified as main driver.

Eligibility criteria: Inclusion Criteria:

* Confirmed diagnosis of CF
* Patients with elevated LCI of ≥ 7.5 at screening
* Patients with FEV1 of ≥ 50% predicted at screening
* Use of inhaled Tobramycin in a 28 days on / off regimen in the past 3 months before screening
* chronic lung Infection with Pseudomonas aeruginosa

Exclusion Criteria:

* Patients who are regularly receiving more than one class of inhaled anti-pseudomonal antibiotic
* Patients who have used oral or intravenous anti-pseudomonal antibiotics within 28 days prior to on-phase of study drug
* Pregnant or nursing (lactating) women
* Change in dose, formulation or strength of the study drug in the past treatment cycle before screening
* History of hearing loss or chronic tinnitus
* Infection with Burkholderia cenocepacia complex

Other protocol-defined inclusion/exclusion criteria may apply

Primary outcomes:
measure: Change From Baseline in Lung Clearance Index (LCI) After 4 Weeks Following Onset of Study
timeFrame: Baseline, week 4
Secondary outcomes:
measure: Change From Baseline of Forced Expiratory Volume at 1 Second (FEV1) After 4 Weeks Following Onset of Study
timeFrame: Baseline, week 4
measure: Change From Baseline of Colony-forming Units (CFU) After 4 Weeks Following Onset of Study
timeFrame: Baseline, week 4
measure: Change From Baseline in Lung Clearance Index (LCI) After 1 Week
timeFrame: Baseline, week 1
measure: Change of Lung Clearance Index (LCI) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)
timeFrame: week 4, week 8
measure: Change of Forced Expiratory Volume at 1 Second(FEV1) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)
timeFrame: week 4, week 8
measure: Change of Colony-forming Units (CFU) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)
timeFrame: week 4, week 8
Other outcomes:
Locations:
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Erlangen
country: Germany
facility: Novartis Investigative Site
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Frankfurt
country: Germany
facility: Novartis Investigative Site
city: Germering
country: Germany
facility: Novartis Investigative Site
city: Jena
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharma GmbH, Nürnberg, Germany
name: Jörg Ziegler, PhD
role: STUDY_DIRECTOR
References:
citation: Sutharsan S, Naehrig S, Mellies U, Sieder C, Ziegler J. An 8 week open-label interventional multicenter study to explore the lung clearance index as endpoint for clinical trials in cystic fibrosis patients >/=8 years of age, chronically infected with Pseudomonas aeruginosa. BMC Pulm Med. 2020 Jun 12;20(1):167. doi: 10.1186/s12890-020-01201-y.