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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 : February 13, 2020
Sponsor:
Information provided by (Responsible Party):
Gilead Sciences

Tracking Information
First Submitted Date  ICMJE July 13, 2017
First Posted Date  ICMJE July 17, 2017
Last Update Posted Date February 13, 2020
Actual Study Start Date  ICMJE November 28, 2017
Estimated Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 13, 2017)
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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03219164 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 13, 2017)
  • 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.
  • 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.
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of 2 Treatment Regimens of Aztreonam for Inhalation Solution in Children With Cystic Fibrosis and New Onset Pseudomonas Aeruginosa Infection
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE
  • Pseudomonas Aeruginosa Respiratory Tract Infection
  • Cystic Fibrosis
Intervention  ICMJE
  • 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.
Study Arms  ICMJE
  • Experimental: AZLI
    AZLI for 28 days
    Intervention: Drug: AZLI
  • Experimental: AZLI + Placebo
    AZLI for 14 days followed by placebo for 14 days
    Interventions:
    • Drug: AZLI
    • Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 13, 2017)
140
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2021
Estimated Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Months to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Gilead Clinical Study Information Center 1-833-445-3230 (GILEAD-0) GileadClinicalTrials@gilead.com
Listed Location Countries  ICMJE Austria,   Belgium,   Denmark,   France,   Germany,   Greece,   Israel,   Italy,   Netherlands,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03219164
Other Study ID Numbers  ICMJE GS-US-205-1850
2016-002749-42 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Gilead Sciences
Study Sponsor  ICMJE Gilead Sciences
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Gilead Study Director Gilead Sciences
PRS Account Gilead Sciences
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP