Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonizatio
- Conditions
- cystic fibrosisMucoviscidosis1003868610004018
- Registration Number
- NL-OMON50497
- Lead Sponsor
- Gilead Sciences
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 4
* Male or female aged 3 months to less than 18 years
* Diagnosis of CF as determined by the 2008 CF Consensus Conference criteria:
o Sweat chloride level * 60 mEq/L by quantitative pilocarpine iontophoresis;
o or a genotype with 2 identifiable mutations consistent with CF;
o or an abnormal nasal transepithelial potential difference (NPD), and 1 or
more clinical features consistent with CF
* Documented new onset of positive respiratory tract culture for PA within 30
days of Screening defined as either first lifetime documented PA positive
culture, or PA recovered after at least a 2-year history of PA-negative
respiratory cultures (at least 2 cultures per year)
* FEV1 * 80% predicted (for subjects * 6 years of age who can reliably perform
spirometry assessments)
* Clinically stable with no evidence of either acute significant respiratory
symptoms that would require administration of IV antipseudomonal antibiotics,
oxygen supplementation, or hospitalization
* Use of IV or inhaled antipseudomonal antibiotics within 2 years of Screening
* Use of oral antipseudomonal antibiotics for a respiratory event within 30
days of study entry (Screening visit)
* History or intolerance to inhaled short acting *2 agonists
* History of lung transplantation
* Current requirement for daily continuous oxygen supplementation or
requirement of more than 2 L/minute at night
* Hospitalization for a respiratory event within 30 days prior to Screening
* Changes in bronchodilator, corticosteroid, dornase alfa, or hypertonic saline
medications within 7 days prior to Screening; for subjects on a stable regimen
of hypertonic saline (28 days on/28 days off), beginning or ending a cycle of
hypertonic saline is allowed
* Changes in physiotherapy technique or schedule within 7 days prior to
Screening
* Abnormal renal or hepatic function results at most recent test within the
previous 12 months, defined as
o AST or ALT >5 times upper limit of normal (ULN), or
o Serum creatinine > 2 times ULN for age
* Presence of a condition or abnormality that would compromise the subject*s
safety or the quality of the study data, in the opinion of the Investigator
* Known hypersensitivity to aztreonam, its metabolites, or formulation
excipients in AZLI
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>To evaluate the safety and efficacy of a 14-day course vs a 28-day course of<br /><br>AZLI 75 mg three times a day (TID) in subjects with new onset PA respiratory<br /><br>tract colonization/infection as determined by PA eradication over a 28-day<br /><br>post-treatment follow-up period.</p><br>
- Secondary Outcome Measures
Name Time Method <p>* To evaluate the time from primary eradication to PA recurrence over a<br /><br>108-week post-treatment follow-up period<br /><br>* To compare the efficacy of AZLI 75 mg TID for 14 days vs historical pooled<br /><br>tobramycin nebulizer solution (TNS) two times a day (BID) for 28 days as<br /><br>determined by PA eradication over a 28-day post-treatment follow-up period<br /><br>* To evaluate the time to PA recurrence for a sub-group of subjects matching<br /><br>the population in the TNS ELITE Study over a 108-week post-treatment follow-up<br /><br>period</p><br>