Evaluation of Lung Clearance Index in Cystic Fibrosis (CF) Patients, Infected With P.Aeruginosa (ELIXIR)

Novartis

Status and phase: Terminated; Phase 4

Conditions: Chronic Lung Infection With Pseudomonas Aeruginosa in Cystic Fibrosis

Treatments: Drug: TIS or TIP

Study type: Interventional

Funder types: Industry

Identifiers

NCT02248922

CTBM100CDE02

Details and patient eligibility

About

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.

Full 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.

Enrollment: 17 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Diagnostic

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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17 participants in 1 patient group

Tobramycin ALL

Experimental group

Description:

300mg nebulized Tobramycin (Tobramycin inhalation solution(TIS)) or TOBI Podhaler (Tobramycin inhalation powder(TIP), equivalent dry powder)twice a day (BID) 28days on / 28 days off

Treatment:

Drug: TIS or TIP