cystic fibrosis Mucoviscidosis
Conditions
Interventions
There is a 50% chance to receive AZLI for 28 days and a 50% chance to receive
AZLI for 14 days and placebo for another 14 days.
Sponsors
Gilead Sciences
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: * 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
Exclusion criteria
Exclusion criteria: * 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the safety and efficacy of a 14-day course vs a 28-day course of AZLI 75 mg three times a day (TID) in subjects with new onset PA respiratory tract colonization/infection as determined by PA eradication over a 28-day post-treatment follow-up period. | — |
Secondary
| Measure | Time frame |
|---|---|
| * To evaluate the time from primary eradication to PA recurrence over a 108-week post-treatment follow-up period * To compare the efficacy of AZLI 75 mg TID for 14 days vs historical pooled tobramycin nebulizer solution (TNS) two times a day (BID) for 28 days as determined by PA eradication over a 28-day post-treatment follow-up period * To evaluate the time to PA recurrence for a sub-group of subjects matching the population in the TNS ELITE Study over a 108-week post-treatment follow-up period | — |
Countries
Netherlands
Outcome results
None listed