DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more

Aztreonam Lysine for Pseudomonas Infection Eradication Study

Information source: Gilead Sciences
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Cystic Fibrosis

Intervention: Aztreonam for Inhalation Solution (AZLI) (Drug)

Phase: Phase 2

Status: Completed

Sponsored by: Gilead Sciences

Official(s) and/or principal investigator(s):
Mark Bresnik, MD, Study Director, Affiliation: Gilead Sciences


This is an open-label, multi-center study in pediatric patients age 3 months to less than 18 years with cystic fibrosis (CF) and newly detected Pseudomonas aeruginosa (PA) pulmonary colonization/infection. All eligible participants will be treated with a 28-day course of Aztreonam for Inhalation Solution (AZLI) 75 mg 3 times daily. After completion of study drug, subjects will be followed up through Day 196 for safety and recurrence of PA. The primary objective is to evaluate the proportion of participants with PA-negative cultures at all time points during a 6-month monitoring period (through Day 196) after cessation of AZLI treatment. Microbiological cultures will be obtained at Baseline, Day 28 (end of AZLI treatment), Day 56 (1 month after completing AZLI treatment), Day 112 (3 months after completing AZLI treatment), and Day 196 (6 months after completing AZLI treatment).

Clinical Details

Official title: Open-Label Phase 2 Trial to Evaluate the Safety and Efficacy of Aztreonam 75 mg Powder and Solvent for Nebuliser Solution/Aztreonam for Inhalation Solution (AZLI) in Pediatric Patients With Cystic Fibrosis (CF) and New Onset Lower Respiratory Tract Culture Positive for Pseudomonas Aeruginosa (PA)

Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome:

Percentage of Participants With PA-negative Cultures at All Time Points After Cessation of Active Treatment (Evaluable Analysis Set)

Percentage of Participants With PA-negative Cultures at All Time Points After Cessation of Active Treatment (Sensitivity Analysis Set)

Secondary outcome:

Change From Baseline in FEV1% Predicted

Change From Baseline in CFQ-R RSS Score

Percentage of Participants With PA-negative Cultures

Use of Additional (Non-study) Antipseudomonal Antibiotics

Change From Baseline in Weight

Change From Baseline in Height

Change From Baseline in Body Mass Index (BMI)

Pharmacokinetics (PK) Peak and Trough Plasma Concentrations of Aztreonam


Minimum age: 3 Months. Maximum age: 17 Years. Gender(s): Both.


Inclusion Criteria:

- Males or females age 3 months to less than 18 years

- 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 test OR

- Two well-characterized, disease-causing genetic mutations in the CF

transmembrane conductance regulator (CFTR) gene AND

- One or more clinical features consistent with CF

- Documented new onset of positive lower respiratory tract culture (e. g., throat swab,

sputum, or BAL) for PA within 30 days of study entry (prior to screening visit) 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 1 second (FEV1) ≥ 80% predicted at screening visit

(subjects ≥ 6 years of age)

- 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.

- All sexually active females who were of childbearing potential must agree to use a

highly effective method of contraception during heterosexual intercourse throughout the study. Females utilizing hormonal contraceptives as a birth control method must have used the same method for at least 3 months prior to study drug dosing.

- Males must agree to use barrier contraception (condom with spermicide) during

heterosexual intercourse from screening through to study completion and for 90 days from the last dose of study investigational medicinal product

- Participants and/or parent/guardian must be able to give written informed consent

prior to any study related procedure Exclusion Criteria:

- Use of IV or inhaled antipseudomonal antibiotics within 2 years of study entry

(screening visit)

- Use of oral antipseudomonal antibiotics within 30 days of study entry (screening


- History of sputum or throat swab culture yielding Burkholderia spp. within 2 years

prior to screening visit

- History of local or systemic hypersensitivity to monobactam antibiotics

- History of intolerance to inhaled short acting beta 2 agonists

- History of lung transplantation

- History of AZLI (or Cayston®) administration

- Administration of any investigational drug or device within 28 days prior to

screening visit or within 6 half-lives of the investigational drug (whichever is longer)

- Current use of oral corticosteroids in doses exceeding the equivalent of 10 mg

prednisone per day or 20 mg prednisone every other day

- Current requirement for daily continuous oxygen supplementation or requirement of

more than 2 L/minute at night

- 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 antimicrobial, bronchodilator (BD), corticosteroid, dornase alfa, or

hypertonic saline medications within 7 days prior to screening visit; for participants 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 visit

- 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

- Pregnant or lactating females; a negative urine pregnancy test is required for all

females of childbearing potential (unless surgically sterile), and confirmatory serum pregnancy test in the event of an initial positive urine test result

- Any serious or active medical or psychiatric illness (including drug or alcohol

abuse), which in the opinion of the investigator, would interfere with treatment, assessment, or compliance with the protocol

- Presence of a condition or abnormality that would compromise the patient's safety or

the quality of study data, in the opinion of the investigator

Locations and Contacts

Medizinische Universität Innsbruck Abt. für Kinder- und Jugendheilkunde, Pädiatrie III (Zystische Fibrose), Innsbruck 6020, Austria

Hôpital Universitaire des Enfants Reine Fabiola Brussels, Brussels 1020, Belgium

Paediatrics, University Hospital Brussels (UZB), Brussels 1090, Belgium

Pediatric Respiratory Department, Ghent University Hospital, Ghent 9000, Belgium

Pediatric Pulmonology, Dept Pediatrics University Hospital Gasthuisberg, Leuven 3000, Belgium

CHU de Boredaux Hôpital des Enfants - Pellegrin CEDRE, Bordeaux 33076, France

CRCM mixte / CHU ESTAING, Clermont Ferrand 63003, France

CHI de Créteil Departement pediatrie, Creteil 94000, France

Centre hospitalier Robert Bissons CRCM - service pédiatrie, Lisieux 14100, France

Hopital Robert Debre, Paris, France

Service pédiatrie II Hôpital Necker Enfants Malades, Paris 75743, France

Centre de Ressources et de Compétences sur la Mucoviscidose ( CRCM), Roscoff, France, Roscoff 29684, France

Charité Campus Virchow Klinikum, Universitätsmedizin Berlin, Klinik für Pädiatrie mit Schwerpunkt Pneumologie/Immunolgie Prof. Wahn, Berlin 13353, Germany

Klinik fur Kinder- und Jugendmedizinim St Josef-Hopsital, Bochum, Germany

Universitätsklinikum Essen, Zentrum für Kinderheilkunde - Abteilung Allg. Kinderheilkunde/Neuropaediatrie, Erlangen 91054, Germany

Universitaetsklinikum Bonn-Zentrum fuer Kinderheikunde, Essen, Germany

Christiane Herzog CF-Center, Goethe University Hospital, Frankfurt 60590, Germany

Universitätsklinikum Gießen und Marburg GmbH, Giessen 35392, Germany

University Children's Hospital, Tubingen 72076, Germany

Azienda Ospedaliero-Universitaria di Catania, Dipartimento di Pediatria, UO Broncopneumologia Pediatrica, Catania 95123, Italy

Cystic Fibrosis Centre Paediatric Department, A. Meyer Children Hospital Florence, Florence 50139, Italy

Universita' Federico II di Napoli, Napoli 80131, Italy

Fondazione IRCCS, Ospedale Pediatrico, Bambino Gesu' di Roma, Rome 00165, Italy

Centro Fibrosi Cistica di Verona, Azienda Ospedaliera Universitaria Integrata di Verona, Verona 37126, Italy

Division of Respiratory Medicine and Allergology, Department of Pediatrics, Erasmus MC-Sophia Children's Hospital, Rotterdam, The Netherlands, Rotterdam 3015 GJ, Netherlands

Longziekten Universitair Medisch (PEDIATRIC), Ultrecht, Utrecht 3584 EA, Netherlands

ISPL Centrum Medyczne, Bialystok 15-003, Poland

Specjalistyczny Zespół Opieki Zdrowotnej nad Matką i Dzieckiem, Poradnia Leczenia Mukowiscydozy, Gdansk 80-308, Poland

Instytut Gruźlicy i Chorób Płuc, Klinki Pneumologii i Mukowiscydozy, Rabka Zdroj 34-700, Poland

Instytut Matki i Dziecka Klinika Pediatrii, Warszawa 01-211, Poland

Hospital Vall D' Hebron Pediatric Pneunmonology and Cystic Fibrosis Clinic, Barcelona 08035, Spain

Hospital Infantil La Paz, Madrid, Spain

Hospital infantil Universitario Niño Jesus, Servicio de Neumología Pediatrica, Madrid 28009, Spain

Hospital Ramon y Cajal, Madrid 28049, Spain

Hospital Materno-Infantil Carlos Haya, Servicio de Neumología Pediatrica, Malaga 29011, Spain

Phoenix Children's Hospital, Phoenix, Arizona 85016, United States

Nemours Children's Clinic- Jacksonville, Jacksonville, Florida 32207, United States

Nemours Childrens Clinic Orlando, Orlando, Florida 32801, United States

Children's Memorial Hospital, Chicago, Illinois 60614, United States

Riley Hospital for Children, Indianapolis, Indiana 46202, United States

Children's Hospital Boston, Boston, Massachusetts 02115, United States

University of Michigan, Ann Arbor, Michigan 48109, United States

Children's Mercy Hospital and Clinics, Kansas City, Missouri 64108, United States

Saint Louis University, St. Louis, Missouri 63104, United States

Cohen Children's Medical Center of NY, Great Neck, New York 11021, United States

SUNY Upstate Medical University, Syracuse, New York 13210, United States

UNC Chapel Hill, Chapel Hill, North Carolina 27599, United States

Akron Children's Hospital, Akron, Ohio 44308, United States

Cincinnati Children's Hospital, Cincinnati, Ohio 45229, United States

Nationwide Children's Hospital, Columbus, Ohio 43205, United States

Toledo Children's Hospital CF Research Center, Toledo, Ohio 43606, United States

PennState Milton S. Hershey Medical Center, Hershey, Pennsylvania 17033, United States

St. Christopher's Hospital for Children, Philadelphia, Pennsylvania 19134, United States

Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania 15224, United States

Vanderbilt Children's Hospital, Nashville, Tennessee 37232, United States

Baylor College of Medicine, Houston, Texas 77030, United States

University of Utah, Salt Lake City, Utah 84108, United States

Children's Hospital of Wisconsin, Milwaukee, Wisconsin 53201, United States

Additional Information

Starting date: August 2011
Last updated: July 7, 2014

Page last updated: August 20, 2015

-- advertisement -- The American Red Cross
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2017