Trial record 1 of 1 for:
NCT03219164 | Cystic Fibrosis
Study of Aztreonam for Inhalation in Children With Cystic Fibrosis and New Infection of the Airways by Pseudomonas Aeruginosa Bacteria (ALPINE 2)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT03219164 |
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Recruitment Status :
Terminated
(Study was terminated early by sponsor due to the challenges and risks introduced by Coronavirus disease 2019 (COVID-19) pandemic.)
First Posted : July 17, 2017
Results First Posted : May 16, 2022
Last Update Posted : June 14, 2022
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Sponsor:
Gilead Sciences
Information provided by (Responsible Party):
Gilead Sciences
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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 pediatric participants with new onset Pseudomonas aeruginosa respiratory tract infection or colonization.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Pseudomonas Aeruginosa Respiratory Tract Infection/Colonization Cystic Fibrosis | Drug: AZLI Drug: Placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 149 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 |
| Actual Primary Completion Date : | May 27, 2020 |
| Actual Study Completion Date : | September 23, 2021 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
| Arm | Intervention/treatment |
|---|---|
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Experimental: AZLI + Placebo
75 mg/ml of aztreonam will be administered thrice daily (TID) for 14 days followed by placebo to match (PTM) aztreonam TID for 14 days.
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Drug: AZLI
Administered 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 Names:
Drug: Placebo Administered 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. |
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Experimental: AZLI
75 mg/ml of aztreonam will be administered TID for 28 days.
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Drug: AZLI
Administered 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 Names:
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Primary Outcome Measures :
- Percentage of Participants With Pseudomonas Aeruginosa (PA)-Negative Cultures Through 28 Days Post-Treatment in the 14-Day Treatment Group vs 28-Day Treatment Group [ Time Frame: 28 days post treatment (Weeks 4 to 6 for the 14 Day treatment group and Weeks 4 to 8 for the 28 Day treatment group) ]
Secondary Outcome Measures :
- Time From Primary Eradication to PA Recurrence Over a 108-Week Post-Treatment Follow-up Period [ Time Frame: Last dose date of AZLI up to Week 112 ]The primary eradication was achieved when all cultures through 28 days post AZLI treatment were PA negative. Recurrence after PA eradication was defined as first positive PA culture result in participant who successfully met primary endpoint and had no PA-positive culture from local lab at Week 4 through Week 6 for AZLI 14 Days group or through Week 8 for AZLI 28 Days group.
- Percentage of Participants With PA-negative Cultures Through 28 Days Post-Treatment in the 14-Day Treatment Group vs Historical Pooled Data for PA Eradication at 28 Days Post-Treatment in Participants Treated With Tobramycin Nebulizer Solution (TNS) [ Time Frame: 28 days post treatment (Weeks 4 to 6 for the 14 Day treatment group) ]
- Time to PA Recurrence for a Sub-Group of Participants Matching the Population in the TNS ELITE Study Over a 108-Week Post-Treatment Follow-up Period [ Time Frame: Last dose date of AZLI up to Week 112 ]
In ELITE study (NCT00391976), participants with cystic fibrosis who had early PA infection received TNS. Published criteria for efficacy analysis population in ELITE study included:
- Participants must be ≥ 6 months at randomization
- No history of positive anti-PA antibody (no anti-PA immunoglobulin G [IgG] antibody interpretation at Screening/Baseline) on record
- Did not use anti-pseudomonal antibiotics through 28 days after completion of active treatment and within 2 years of Screening
- Non-missing PA culture result at 28 days after last dose of AZLI
- PA negative through 28 days after completion of active treatment
- No important protocol deviation related to compliance with study drug administration
- 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).
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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 intravenous (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
Locations
Show 53 study locations
Sponsors and Collaborators
Gilead Sciences
Investigators
| Study Director: | Gilead Study Director | Gilead Sciences |
Study Documents (Full-Text)
Documents provided by Gilead Sciences:
Documents provided by Gilead Sciences:
Study Protocol [PDF] April 15, 2020
Statistical Analysis Plan [PDF] December 1, 2021
| Responsible Party: | Gilead Sciences |
| ClinicalTrials.gov Identifier: | NCT03219164 |
| Other Study ID Numbers: |
GS-US-205-1850 2016-002749-42 ( EudraCT Number ) |
| First Posted: | July 17, 2017 Key Record Dates |
| Results First Posted: | May 16, 2022 |
| Last Update Posted: | June 14, 2022 |
| Last Verified: | May 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | Qualified external researchers may request IPD for this study after study completion. For more information, please visit our website at https://www.gileadclinicaltrials.com/transparency-policy/ |
| Supporting Materials: |
Study Protocol Statistical Analysis Plan (SAP) |
| Time Frame: | 18 months after study completion |
| Access Criteria: | A secured external environment with username, password, and RSA code. |
| URL: | https://www.gileadclinicaltrials.com/transparency-policy/ |
| 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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Cystic Fibrosis Fibrosis Infections Communicable Diseases Respiratory Tract Infections Pseudomonas Infections Disease Attributes Pathologic Processes Pancreatic Diseases Digestive System Diseases |
Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Gram-Negative Bacterial Infections Bacterial Infections Bacterial Infections and Mycoses Aztreonam Anti-Bacterial Agents Anti-Infective Agents |