Aztreonam Lysine for Pseudomonas Infection Eradication Study

Completed · Phase 2

Conditions studied: Cystic Fibrosis

In brief

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).

Key facts

Study ID

NCT01375049

Run by

Gilead Sciences

People needed

105

Starts

2011-08-01

Expected to finish

2013-05-01

Last updated by the study team

2014-07-17

Who can join

Age: 0 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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 visit)
• 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

Where it is running

• Nemours Children's Clinic- Jacksonville — Jacksonville, Florida, United States
• Nemours Childrens Clinic Orlando — Orlando, 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
• University of Michigan — Ann Arbor, Michigan, United States
• Children's Mercy Hospital and Clinics — Kansas City, Missouri, United States
• Saint Louis University — St Louis, Missouri, United States
• Cohen Children's Medical Center of NY — Great Neck, New York, United States
• SUNY Upstate Medical University — Syracuse, New York, United States
• UNC Chapel Hill — Chapel Hill, North Carolina, United States
• Akron Children's Hospital — Akron, Ohio, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Toledo Children's Hospital CF Research Center — Toledo, Ohio, United States
• PennState Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States
• St. Christopher's Hospital for Children — Philadelphia, Pennsylvania, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Vanderbilt Children's Hospital — Nashville, Tennessee, United States
• Baylor College of Medicine — Houston, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• Children's Hospital of Wisconsin — Milwaukee, Wisconsin, United States
• Medizinische Universität Innsbruck Abt. für Kinder- und Jugendheilkunde, Pädiatrie III (Zystische Fibrose) — Innsbruck, Austria
• Hôpital Universitaire des Enfants Reine Fabiola Brussels — Brussels, Belgium
• Phoenix Children's Hospital — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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