A Clinical Trial to Evaluate the Effects of the drug Aztreonam Lysine for Inhalation in Patients with Cystic Fibrosis, Mild Lung Disease, and infection with the bacteria Pseudomonas aeruginosa (AIR-CF4)

• Register: EUCTR
• Last refreshed on: 16 February 2015
• Main ID: EUCTR2015-000395-97-Outside-EU/EEA
• Date of registration: 02/02/2015
• Prospective Registration: Yes
• Primary sponsor: Gilead Sciences, Inc.
• Public title: A Clinical Trial to Evaluate the Effects of the drug Aztreonam Lysine for Inhalation in Patients with Cystic Fibrosis, Mild Lung Disease, and infection with the bacteria Pseudomonas aeruginosa (AIR-CF4)
• Scientific title: A Double-Blind, Multicenter, Multinational, Randomized, Placebo-Controlled Trial Evaluating Aztreonam Lysine for Inhalation in Patients with Cystic Fibrosis, Mild Lung Disease, and P. aeruginosa (AIR-CF4)
• Target sample size: 157
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-000395-97
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Australia, Canada, United States

Contacts

• Name: Clinical Trials Information
• Affiliation: Gilead Sciences International Ltd

• Name: Clinical Trials Information
• Affiliation: Gilead Sciences International Ltd

Key inclusion & exclusion criteria

Inclusion criteria:
1. Patients = 6 years of age.
2. Documentation of CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
a. Sweat chloride = 60 mEq/L by quantitative pilocarpine iontophoresis test (QPIT);
b. Two well-characterized mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene; or
c. Abnormal nasal potential difference.
3. PA present in expectorated sputum or throat swab culture at Visit 1 OR documented PA in 2 expectorated sputum or throat swab cultures within the 12 months prior to Visit 1. (One of the previous PA positive cultures must be no more than 3 months prior to Visit 1.)
4. FEV1 > 75% predicted at Visit 1.
5. Patient must exhibit two or more of the following chronic and/or intermittent CF symptoms, for a minimum of 28 days prior to randomization and with no worsening of symptoms within 7 days prior to randomization:
a. Chest congestion
b. Daily cough
c. Productive cough
d. Wheezing
e. Trouble breathing
f. Nocturnal wakening due to coughing
6. Patients (and parent/guardian as required) must be able to provide written informed consent/assent prior to any study related procedures.
7. Females of childbearing potential must have a negative urine pregnancy test at Visit 1.
8. Ability to perform reproducible pulmonary function tests.
9. In the opinion of the Investigator, the patient does not require immediate antipseudomonal antibiotic intervention to treat an impending exacerbation, and the patient’s condition is stable enough to enroll in the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 89
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 68
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Administration of any investigational drug or device within 28 days prior to Visit 1 or within 6 half-lives of the investigational drug (whichever is longer).
2. Administration of any IV, oral, or inhaled antipseudomonal antibiotic within 28 days prior to Visit 1.
3. Known local or systemic hypersensitivity to monobactam antibiotics.
4. Inability to tolerate short-acting bronchodilator use at least three times daily.
5. Changes in or initiation of chronic azithromycin treatment within 28 days prior to Visit 1.
6. Changes in or initiation of chronic hypertonic saline treatment within 28 days prior to Visit 1.
7. Changes in or initiation of dornase alpha within 28 days prior to Visit 1.
8. Changes in antimicrobial, bronchodilator (BD), or corticosteroid medications within 7 days prior to Visit 1.
9. Changes in physiotherapy technique or schedule within 7 days prior to Visit 1.
10. History of lung transplantation.
11. History of participation (enrollment) in any prior clinical trials with AZLI.
12. A chest radiograph at Visit 1 (or within the previous 180 days of Visit 1), with abnormalities indicating a significant acute finding (e.g., lobar infiltrate and atelectasis, pneumothorax, or pleural effusion). A chest radiograph obtained and interpreted between Visits 1 and 2 is also acceptable for determining eligibility.
13. Positive urine pregnancy test at Visit 1; all women of childbearing potential will be tested.
14. Females of childbearing potential who are lactating or are not (in the opinion of the investigator) practicing an acceptable method of birth control; female patients who utilize hormonal contraceptives as their birth control method must have used the same method
for at least 3 months before study dosing.
15. Patient is being assessed at Visit 1 by the investigator for an acute change in respiratory symptoms.
16. Any serious or active medical or psychiatric illness, which in the opinion of the investigator, would interfere with patient treatment, assessment, or compliance with the protocol.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Cystic Fibrosis with Mild Lung Disease and P. aeruginosa (AIR-CF4)

Intervention(s)

Trade Name: Cayston
Aztreonam 75 mg powder and solvent for nebuliser solution
Pharmaceutical Form: Powder and solvent for nebuliser solution
INN or Proposed INN: AZTREONAM
CAS Number: 78110-38-0
Current Sponsor code: AZLI
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 75-
Pharmaceutical form of the placebo: Powder and solvent for nebuliser solution
Route of administration of the placebo: Inhalation use

Primary Outcome(s)

Primary end point(s): The primary endpoint is change from baseline at Day 28 in clinical symptoms as assessed by the respiratory symptoms domain of the CFQ-R.

Main Objective: The primary objective of this study is to assess the safety and efficacy of a 28-day course of AZLI in patients with CF, mild lung disease (FEV1 > 75% predicted), and PA.

Secondary Objective: Not applicable

Timepoint(s) of evaluation of this end point: Day 28

Secondary Outcome(s)

Secondary end point(s): • Change from baseline in clinical symptoms as assessed by the respiratory symptoms
domain of the CFQ-R at Days 14 and 42.
• Change from baseline in clinical symptoms as assessed by the non-respiratory domains of
the CFQ-R at Day 28.
• Use of additional (non-protocol specified) antipseudomonal antibiotics during the course
of the study.
• Proportion of patients hospitalized and the number of days patients were hospitalized
during the study.
• Change from baseline in PA colony-forming units (CFUs) in sputum at Day 28.
• Relative change from baseline FEV1 percent of predicted at Day 28.

Timepoint(s) of evaluation of this end point: Days 14, 28 and 42

Secondary ID(s)

64,402

GS-US-205-0117

Source(s) of Monetary Support

Gilead Sciences, Inc.

Gilead Sciences Pty Ltd

Gilead Sciences Canada, Inc.

Secondary Sponsor(s)

Gilead Sciences Canada, Inc.

Gilead Sciences Pty Ltd