Aztreonam Lysine for Pseudomonas Infection Eradication Study (ALPINE)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Gilead Sciences
ClinicalTrials.gov Identifier:
NCT01375049
First received: June 15, 2011
Last updated: June 10, 2013
Last verified: June 2013
  Purpose

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 subjects will begin a 28-day course of AZLI 75 mg 3 times daily (TID). After completion of study drug, subjects will be followed up through Day 196 for safety and recurrence of PA.


Condition Intervention Phase
Cystic Fibrosis
Drug: Aztreonam Lysine for Inhalation (AZLI)
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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)

Resource links provided by NLM:


Further study details as provided by Gilead Sciences:

Primary Outcome Measures:
  • Proportion of patients with PA-negative cultures [ Time Frame: Through 6 month follow-up ] [ Designated as safety issue: No ]
    To evaluate the proportion of patients with PA-negative microbiological cultures at all time points during a 6 month monitoring period (through Day 196) after cessation of 28 day course of AZLI.


Enrollment: 105
Study Start Date: August 2011
Study Completion Date: May 2013
Primary Completion Date: May 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Aztreonam Lysine for Inhalation (AZLI)
Aztreonam Lysine for inhalation dosing TID using the investigational eFlow nebulizer (AZLI 75 mg TID)
Drug: Aztreonam Lysine for Inhalation (AZLI)
75 mg three times a day (TID)
Other Name: Cayston

Detailed Description:

This is an open-label, multicenter study in pediatric patients age 3 months to less than 18 years with CF and newly detected PA pulmonary colonization/infection. The primary objective of this study is to evaluate the proportion of patients with PA-negative cultures at all time points during a 6-month monitoring period (through Day 196) after cessation of Aztreonam for Inhalation Solution (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).

  Eligibility

Ages Eligible for Study:   3 Months to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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)
  • 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 require administration of IV antipseudomonal antibiotics, oxygen supplementation, or hospitalization
  • All sexually active female subjects who are 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
  • Male subjects 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
  • Subjects 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 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 subjects 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:

    • AST or 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 female subjects 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 subject 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01375049

  Show 58 Study Locations
Sponsors and Collaborators
Gilead Sciences
Investigators
Study Director: Mark Bresnik, MD Gilead Sciences
  More Information

No publications provided

Responsible Party: Gilead Sciences
ClinicalTrials.gov Identifier: NCT01375049     History of Changes
Other Study ID Numbers: GS-US-205-0162
Study First Received: June 15, 2011
Last Updated: June 10, 2013
Health Authority: United States: Food and Drug Administration
Belgium: Ethics Committee
Germany: Ethics Commission
Germany: Federal Institute for Drugs and Medical Devices
France: Ministry of Health
Spain: Comité Ético de Investigación Clínica
Italy: Ministry of Health

Keywords provided by Gilead Sciences:
Cystic fibrosis
CF
PA
Pseudomonas aeruginosa
AZLI
aztreonam

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pseudomonas Infections
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes
Gram-Negative Bacterial Infections
Bacterial Infections
Aztreonam
Anti-Bacterial Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 22, 2014