Safety of AZLI in Children With Cystic Fibrosis (CF) and Chronic Pseudomonas Aeruginosa in the Lower Airways

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01404234
• Date of registration: 26/07/2011
• Prospective Registration: Yes
• Primary sponsor: Gilead Sciences
• Public title: Safety of AZLI in Children With Cystic Fibrosis (CF) and Chronic Pseudomonas Aeruginosa in the Lower Airways PALS
• Scientific title: Open-Label Phase 3 Trial to Evaluate the Safety of Aztreonam 75 mg Powder and Solvent for Nebuliser Solution/Aztreonam for Inhalation Solution (AZLI) in Children With Cystic Fibrosis (CF) and Chronic Pseudomonas Aeruginosa (PA) in the Lower Airways
• Date of first enrolment: December 2011
• Target sample size: 61
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01404234
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

France, Germany, Italy, Poland, Spain, United States

Contacts

• Name: Mark Bresnik, M.D.
• Affiliation: Gilead Sciences

Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosis of CF as determined by the 1997 CF Consensus Conference criteria:

- Documented sweat chloride = 60 mEq/L by quantitative pilocarpine iontophoresis
test OR

- Abnormal nasal transepithelial potential difference (NPD) test OR

- A genotype with 2 identifiable mutations consistent with CF AND

- One or more clinical features consistent with CF.

- Documented positive lower respiratory tract culture for PA at the screening visit plus
two documented positive lower respiratory tract cultures for PA within 12 months prior
to study entry (must have been a minimum 3 months apart.)

- Clinically stable with no evidence of significant respiratory symptoms or, if obtained
for clinical evaluation, no chest radiograph findings at screening that would have
required administration of IV antipseudomonal antibiotics, oxygen supplementation, or
hospitalization.

Exclusion Criteria:

- Use of IV or inhaled antipseudomonal antibiotics within 14 days of study entry

- Presence of a condition or abnormality that would have compromised the participant's
safety or the quality of study data, in the opinion of the investigator

- History of sputum or throat swab culture yielding Burkholderia spp. within 2 years
prior to screening visit

- History of hypersensitivity/adverse reaction to aztreonam

- History of hypersensitivity/adverse reaction to beta-agonists

- History of lung transplantation

- Administration of any investigational drug or device within 30 days prior to screening
visit or within 6 half-lives of the investigational drug (whichever was longer)

- Hospitalization for pulmonary-related illness within 28 days prior to screening visit

- Changes in or initiation of chronic azithromycin treatment within 28 days prior to
screening visit

- Changes in or initiation of hypertonic saline treatment within 7 days prior to
screening visit; for subjects on a stable regimen of hypertonic saline (28 days on/28
days off), beginning or ending a cycle of hypertonic saline was allowed

- Changes in antimicrobial, bronchodilator (BD), corticosteroid or dornase alfa
medications within 7 days prior to screening visit;

- Changes in physiotherapy technique or schedule within 7 days prior to screening visit

- Abnormal renal or hepatic function results at most recent test within the previous 90
days



Age minimum: N/A
Age maximum: 12 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Pseudomonas Aeruginosa

Intervention(s)

Drug: AZLI

Primary Outcome(s)

Percentage of Participants Who Discontinued Study Drug Due to Safety or Tolerability Reasons [Time Frame: Baseline to Day 168]

Secondary Outcome(s)

Adverse Event Rates Adjusted for Study Duration [Time Frame: Baseline to Day 168]

Change in Pseudomonas Aeruginosa (PA) Sputum Density [Time Frame: Baseline to Day 28, 84, and 140]

Percentage of Participants Hospitalized at Least Once Due to a Respiratory Event [Time Frame: Baseline to Day 168]

Percentage of Participants With Study-drug Induced Bronchospasm [Time Frame: Pretreatment at Baseline to 30 minutes following treatment]

Time to Pulmonary Exacerbation [Time Frame: Baseline to Day 168]

Change From Baseline in FEV1 % Predicted in Subjects Aged = 6 Years [Time Frame: Baseline to Day 28, 84, and 140]

Percentage of Participants Who Used Additional (Non-study) Antipseudomonal Antibiotics [Time Frame: Baseline to Day 168]

Number of Days Participants Were Hospitalized Due to a Respiratory Event [Time Frame: Baseline to Day 168]

Percentage of Participants With Pulmonary Exacerbations [Time Frame: Baseline to Day 168]

Change From Baseline in CFQ-R Respiratory Symptoms Scale (RSS) Score in Subjects Aged = 6 Years [Time Frame: Baseline to Day 28, 84, and 140]

Secondary ID(s)

GS-US-205-0160

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 01/05/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01404234