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Safety and Efficacy of 2 Treatment Regimens of Aztreonam for Inhalation Solution in Children With Cystic Fibrosis and New Onset Pseudomonas Aeruginosa Infection (ALPINE2)

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ClinicalTrials.gov Identifier: NCT03219164
Recruitment Status : Recruiting
First Posted : July 17, 2017
Last Update Posted : August 14, 2019
Sponsor:
Information provided by (Responsible Party):
Gilead Sciences

Brief Summary:
The primary objective of this study is to evaluate the safety and efficacy of a 14-day course versus a 28-day course of aztreonam for inhalation solution (AZLI) in participants with new onset Pseudomonas aeruginosa respiratory tract infection.

Condition or disease Intervention/treatment Phase
Pseudomonas Aeruginosa Respiratory Tract Infection Cystic Fibrosis Drug: AZLI Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 140 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : November 28, 2017
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: AZLI
AZLI for 28 days
Drug: AZLI
75 mg/ml of aztreonam administered thrice daily via the PARI Altera® Nebulizer System. Participants < 2 years will receive via the SmartMask® Baby, 2 to < 6 years via the SmartMask Kids® and > 6 years via the nebulizer mouthpiece.
Other Name: Cayston®

Experimental: AZLI + Placebo
AZLI for 14 days followed by placebo for 14 days
Drug: AZLI
75 mg/ml of aztreonam administered thrice daily via the PARI Altera® Nebulizer System. Participants < 2 years will receive via the SmartMask® Baby, 2 to < 6 years via the SmartMask Kids® and > 6 years via the nebulizer mouthpiece.
Other Name: Cayston®

Drug: Placebo
Placebo administered thrice daily via the PARI Altera® Nebulizer System. Participants < 2 years will receive via the SmartMask® Baby, 2 to < 6 years via the SmartMask Kids® and > 6 years via the nebulizer mouthpiece.




Primary Outcome Measures :
  1. The Proportion of Participants with PA-Negative Cultures Through 28 Days Posttreatment in the14-Day Treatment Group vs the 28-day Treatment Group [ Time Frame: Up to 28 days Posttreatment ]

Secondary Outcome Measures :
  1. Time from Primary Eradication to PA Recurrence over the 108-Week Posttreatment Follow-Up Period [ Time Frame: Up to Posttreatment Week 108 ]
    The primary eradication is achieved when all cultures at 14 and 28 days post AZLI treatment are PA negative.

  2. Proportion of Participants with PA-Negative Cultures Through 28 Days Posttreatment in the 14-day Treatment Group vs. Historical Pooled Data for PA Eradication at 28 days Posttreatment in Participants Treated with Tobramycin Nebulizer Solution (TNS) [ Time Frame: 28 days Posttreatment ]
    The historical data for the proportion of participants with PA-negative cultures during 28 days post-treatment period will be pooled from the published results from the studies conducted on the participants with new onset of PA infection and similar TNS treatment duration and follow-up.

  3. Time to PA Recurrence over the 108-week Posttreatment Follow-Up Period for a Sub-Group of Participants Matching the Population in the TNS ELITE Study Over a 108-week Posttreatment Follow-Up Period [ Time Frame: Up to Posttreatment Week 108 ]
    TNS Elite study is treatment of early Pseudomonas aeruginosa infection in participants with cystic fibrosis



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Ages Eligible for Study:   3 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Diagnosis of Cystic Fibrosis (CF) as determined by the 2008 CF Consensus Conference criteria: Sweat chloride level ≥ 60 milliequivalents per liter (mEq/L) by quantitative pilocarpine iontophoresis; or a genotype with 2 identifiable mutations consistent with CF; or an abnormal nasal transepithelial potential difference (NPD), and 1 or more clinical features consistent with CF
  • Documented new onset of positive respiratory tract culture for PA within 30 days of screening 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 one second (FEV1) ≥ 80% predicted (for subjects ≥ 6 years of age who can reliably perform spirometry assessments)
  • Clinically stable with no evidence of acute significant respiratory symptoms that would require administration of IV antipseudomonal antibiotics, oxygen supplementation, or hospitalization

Key Exclusion Criteria:

  • Use of IV or inhaled antipseudomonal antibiotics within 2 years of screening
  • Use of oral antipseudomonal antibiotics for a respiratory event within 30 days of study entry (screening visit)
  • History of intolerance to inhaled short acting β2 agonists
  • History of lung transplantation
  • Current requirement for daily continuous oxygen supplementation or requirement of more than 2 L/minute at night
  • Hospitalization for a respiratory event within 30 days prior to screening
  • Changes in bronchodilator, corticosteroid, dornase alfa, or hypertonic saline medications within 7 days prior to screening.
  • Significant changes (per investigators discretion) in physiotherapy technique or schedule within 7 days prior to screening
  • 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
  • Presence of a condition or abnormality that would compromise the subject's safety or the quality of the study data, in the opinion of the Investigator
  • Known hypersensitivity to aztreonam, its metabolites, or formulation excipients in AZLI
  • Respiratory cultures performed within 24 months prior to screening that are positive for ANY Burkholderia spp. or Non-tuberculous Mycobacteria (NTM)

Note: Other protocol defined Inclusion/Exclusion criteria may apply


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03219164


Contacts
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Contact: Gilead Clinical Study Information Center 1-833-445-3230 (GILEAD-0) GileadClinicalTrials@gilead.com

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Sponsors and Collaborators
Gilead Sciences
Investigators
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Study Director: Gilead Study Director Gilead Sciences

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Responsible Party: Gilead Sciences
ClinicalTrials.gov Identifier: NCT03219164     History of Changes
Other Study ID Numbers: GS-US-205-1850
2016-002749-42 ( EudraCT Number )
First Posted: July 17, 2017    Key Record Dates
Last Update Posted: August 14, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

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