NCT02248922 - Evaluation of Lung Clearance Index in Cystic Fibrosis (CF) Patients, Infected With P.Aeruginosa | Crick

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

NA

Purpose

DIAGNOSTIC

Masking

NONE

Enrollment

17

Conditions

Chronic Lung Infection With Pseudomonas Aeruginosa in Cystic Fibrosis

Interventions

TIS or TIPDRUG

300mg nebulized Tobramycin (Tobramycin inhalation solution(TIS)) or TOBI Podhaler (Tobramycin inhalation powder(TIP), equivalent dry powder)

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

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

Locations (6)

Novartis Investigative Site

Dresden, Germany

Novartis Investigative Site

Erlangen, Germany

Novartis Investigative Site

Essen, Germany

Novartis Investigative Site

Frankfurt, Germany

Outcomes

Primary Outcomes

Change From Baseline in Lung Clearance Index (LCI) After 4 Weeks Following Onset of Study

The Lung Clearance Index (LCI), measured by Multiple Breath Washout of a tracer gas reflects the obstruction of airways in the lung. Wash-out was completed by definition at the time point when the inhaled gas concentration declined to 2.5% of its concentration at baseline. Washout took longer in patients with more severe disease as gas was trapped in narrowed airways (leading to a higher LCI). A LCI of 7.5 and below is normal.

Time frame: Baseline, week 4

Secondary Outcomes

Change From Baseline of Forced Expiratory Volume at 1 Second (FEV1) After 4 Weeks Following Onset of Study

Change of FEV1 (Forced expiry volume in the first second) measured by Spirometry

Time frame: Baseline, week 4

Change From Baseline of Colony-forming Units (CFU) After 4 Weeks Following Onset of Study

Microbacterial density of Pseudomonas aeruginosa in Sputum-Samples in CFU (Colony Forming Units) per gram sputum.

Time frame: Baseline, week 4

Change From Baseline in Lung Clearance Index (LCI) After 1 Week

The Lung Clearance Index (LCI), measured by Multiple Breath Washout of a tracer gas reflects the obstruction of airways in the lung. Wash-out was completed by definition at the time point when the inhaled gas concentration declined to 2.5% of its concentration at baseline. Washout took longer in patients with more severe disease as gas was trapped in narrowed airways (leading to a higher LCI). A LCI of 7.5 and below is normal.

Time frame: Baseline, week 1

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)

The Lung Clearance Index (LCI), measured by Multiple Breath Washout of a tracer gas reflects the obstruction of airways in the lung. Wash-out was completed by definition at the time point when the inhaled gas concentration declined to 2.5% of its concentration at baseline. Washout took longer in patients with more severe disease as gas was trapped in narrowed airways (leading to a higher LCI). A LCI of 7.5 and below is normal.