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

NCT ID: NCT01404234

Last Updated: 2014-05-29

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 61 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-12-31
• Study Completion Date: 2013-04-30

Brief Summary

This was an open-label, multicenter study in children ≤ 12 years of age with cystic fibrosis (CF) and chronic Pseudomonas aeruginosa (PA) infection in the lower airways using three 28-day courses of Aztreonam for Inhalation Solution (AZLI) 75 mg three times daily, each followed by 28 days off AZLI. The total treatment duration was to be 6 months.

Conditions

Cystic Fibrosis; Pseudomonas Aeruginosa

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Open-label AZLI

Participants received three 28-day courses of AZLI, each followed by 28 days off-treatment.

Group Type: EXPERIMENTAL

AZLI

Intervention Type: DRUG

AZLI 75 mg was administered 3 times daily via the investigational nebulizer.

Interventions

AZLI

AZLI 75 mg was administered 3 times daily via the investigational nebulizer.

Intervention Type: DRUG

Other Intervention Names

Cayston®

Eligibility 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

• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Gilead Sciences

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mark Bresnik, M.D.

Role: STUDY_DIRECTOR

Gilead Sciences

Locations

The Children's Hospital - Denver

Aurora, Colorado, United States

Nemours Children's Clinic - Jacksonville

Jacksonville, Florida, United States

Children's Memorial Hospital

Chicago, Illinois, United States

Riley Hospital for Children

Indianapolis, Indiana, United States

Children's Hospital Boston

Boston, Massachusetts, United States

Children's Mercy Hospital & Clinics

Kansas City, Missouri, United States

SUNY Upstate Medical University

Syracuse, New York, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Baylor College of Medicine

Houston, Texas, United States

University of Utah

Salt Lake City, Utah, United States

C.H.U de Bordeaux

Bordeaux, , France

Centre Hospitalier Robert Bissons

Lisieux, , France

Hopital Necker Enfants Malades

Paris, , France

Charite Campus Virchow Klinikum

Berlin, , Germany

Universitatsklinik St. Josef-Hospital

Bochum, , Germany

Kinder und Jugendklinik, Abteilung Lungen Bronchialheikunde

Erlandgen, , Germany

Universitatsklinikum Essen

Essen, , Germany

J.W. Goethe University Hopsital

Frankfurt, , Germany

Azienda Ospedaliero Universitaria - Policlinico di Catania

Catania, , Italy

A. Meyer Children Hospital Florence

Florence, , Italy

Azienda Ospedaliera Instituti Ospitalieri di Verona

Verona, , Italy

Specjalistyczny Zespot Opieki Zdrowotnej nad Matka i Dzieckiem

Gdansk, , Poland

Instytut Gruzlicy I Chorob Pluc

Rabka-Zdrój, , Poland

Instytut Matki i Dziecka

Warsaw, , Poland

Pediatric Pneunmonology and Cystic Fibrosis Clinic

Barcelona, , Spain

Hospital Infantil La Paz

Madrid, , Spain

Hospital Infantil Universitario Nino Jesus

Madrid, , Spain

Hospital Ramon y Cajal

Madrid, , Spain

Hosp. Mat-Inf. Carlos Haya

Málaga, , Spain

Countries

United States; France; Germany; Italy; Poland; Spain

Other Identifiers

GS-US-205-0160

Identifier Type: -

Identifier Source: org_study_id