Safety and Efficacy of 2 Treatment Regimens of Aztreonam for Inhalation Solution in Children With Cystic Fibrosis and New Onset Pseudomonas Aeruginosa Infection

• Status: Completed
• Trial end date: 2021-09-23
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of this study is to evaluate the safety and efficacy of a 14-day course versus a 28-day course of aztreonam for inhalation solution (AZLI) in participants with new onset Pseudomonas aeruginosa respiratory tract infection.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Gilead Sciences
• Treatments: Aztreonam

Criteria

Key Inclusion Criteria:

• Diagnosis of Cystic Fibrosis (CF) as determined by the 2008 CF Consensus Conference criteria: Sweat chloride level ≥ 60 milliequivalents per liter (mEq/L) by quantitative pilocarpine iontophoresis; or a genotype with 2 identifiable mutations consistent with CF; 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)

• Forced expiratory volume in one second (FEV1) ≥ 80% predicted (for subjects ≥ 6 years of age who can reliably perform spirometry assessments)

• Clinically stable with no evidence of acute significant respiratory symptoms that would require administration of IV antipseudomonal antibiotics, oxygen supplementation, or hospitalization

Key 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 of intolerance to inhaled short acting β2 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.

• Significant changes (per investigators discretion) 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 Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 5 times upper limit of normal (ULN), or 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

• Respiratory cultures performed within 24 months prior to screening that are positive for ANY Burkholderia spp. or Non-tuberculous Mycobacteria (NTM)

Note: Other protocol defined Inclusion/Exclusion criteria may apply