ALPINE 2: AZLI for children with CF and PA lung infection
Research type
Research Study
Full title
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/Colonization
IRAS ID
226910
Contact name
Francis Gilchrist
Contact email
Sponsor organisation
Gilead Sciences, Inc.
Eudract number
2016-002749-42
Clinicaltrials.gov Identifier
064402, IND Number
Duration of Study in the UK
3 years, 7 months, 4 days
Research summary
Cystic fibrosis (CF) is an inherited condition which causes the lungs to become clogged with thick sticky mucus. This causes recurrent chest infections and progressive respiratory failure. Pseudomonas aeruginosa (PA) is the most important cause of chest infections as chronic infection causes increased symptoms and shortened life expectancy. Successful treatment of early PA infection (eradication) prevents chronic infection and its associated morbidity and mortality. Inhaled antibiotics are established as a method of eradicating PA infection but current eradication plans have a significant failure rate.
Aztreonam (AZLI) is approved for the treatment of CF patients with PA infection. The purpose of this study is to evaluate how safe and effective a 14 day course of AZLI is compared to the standard treatment of 28 days for new onset PA infection. Patients aged 3 months to less than 18 years with a diagnosis of CF and new onset PA infection may be eligible for the study.
Participants will be randomly assigned to receive one of the following:
- AZLI administered three times a day for 28 days
- AZLI administered three times a day for 14 days followed by placebo to match three times a day for 14 days.Participants will complete 28 days of dosing. They will then be followed up for a maximum duration of 112 weeks. Participants will be followed through Week 8 for safety and recurrence of PA, and through Week 112 for PA recurrence and additional comparative analyses. Participants with PA recurrence after study drug treatment will be re-treated at the study doctor’s discretion.
Procedures involved include physical exams, vital signs, blood tests, respiratory sample collection, spirometry and clinical observations. This study is sponsored by Gilead Sciences Inc. Approximately 140 participants will take part globally with 51 patients from 15 hospitals in the UK.
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
17/WM/0308
Date of REC Opinion
9 Oct 2017
REC opinion
Further Information Favourable Opinion