Program to Give the Drug Aztreonam Lysine for Inhalation to Patients with Cystic Fibrosis and infection by the Bacteria Pseudomonas aeruginosa Who Have Limited Treatment Options and are at Risk for Disease Progressio

• Conditions: Adult and paediatric cystic fibrosis (CF) patients with pulmonary Pseudomonas aeruginosa (PA) infection.; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2015-000396-26-Outside-EU/EEA
• Lead Sponsor: Gilead Sciences, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-02-02
• Last Update Posted: 16 February 2015

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 603

Inclusion Criteria

> 6years of age
Patient has CF as diagnosed by one of the following:
- Documented sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis test, or
-Two well characterized genetic mutations in the CFTR gene, or
- Abnormal nasal potential difference with accompanying symptoms characteristic of CF
PA present in expectorated sputum or throat swab culture within 2 months prior to consent
Patient must be able to provide written informed consent/assent prior to any study related procedure; parent/guardian must be able to give written informed consent as necessary prior to any study related procedure
At high risk for disease progression as defined by one of the following patient populations:
-First Cohort Criteria:
Those who are waitlisted or eligible for lung transplant based on FEV1 criteria.
Patients who have a level of lung function impairment consistent with lung transplantation criteria, but who are ineligible for transplantation for other reasons, can enroll in this program.
Or
Completed participation in CP-Al-006 (through Visit 20). Patients who withdraw from CP-Al-006 prior to completing all courses of AZLI and all study visits will not be eligible for this protocol.
- Second Cohort Criteria:
Meets first cohort criteria; or
FEV1 < 40 % predicted at the time of consent
- Third Cohort Criteria:
Meets first or second cohort criteria; or
FEV1 < 50 % predicted at the time of consent
Are the trial subjects under 18? yes
Number of subjects for this age range: 108
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 495
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients with any serious or active medical or psychiatric illness that, in the opinion of the investigator, would interfere with patient treatment, assessment or compliance with the protocol or dosing requirements
Patients with hypersensitivity to any of the components of the drug product
Currently enrolled in another clinical trial
Pregnant or lactating females

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of this program is to provide expanded access to aztreonam lysine for inhalation 75 mng prior to its<br>commercial availability to patients with cystic fibrosis and chronic P. aeruginosa airway infection who have limited treatment options and are at risk for disease progression.;Secondary Objective: Not applicable;Primary end point(s): Not applicable: Expanded Access Program;Timepoint(s) of evaluation of this end point: Not applicable

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable