Study of Aztreonam for Inhalation in Children With Cystic Fibrosis and New Infection of the Airways by Pseudomonas Aeruginosa Bacteria (ALPINE 2)
![]() |
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 |
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
|
Sponsor:
Gilead Sciences
Information provided by (Responsible Party):
Gilead Sciences
- Study Details
- Tabular View
- Study Results
- Disclaimer
- How to Read a Study Record
Study Type | Interventional |
---|---|
Study Design | Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double (Participant, Investigator); Primary Purpose: Treatment |
Conditions |
Pseudomonas Aeruginosa Respiratory Tract Infection/Colonization Cystic Fibrosis |
Interventions |
Drug: AZLI Drug: Placebo |
Enrollment | 149 |
Participant Flow
Recruitment Details | Participants were enrolled at study sites in Europe, Israel, and the United States. The first participant was screened on 28 November 2017. The last study visit occurred on 23 September 2021. |
Pre-assignment Details | 149 participants were screened. |
Arm/Group Title | AZLI 14 Days + Placebo 14 Days | AZLI 28 Days |
---|---|---|
![]() |
75 milligrams per milliliter (mg/ml) of aztreonam was administered thrice daily (TID) for 14 days followed by placebo to match (PTM) aztreonam TID for 14 days, both aztreonam and PTM aztreonam were delivered via the PARI Altera® Nebulizer System. Participants below 2 years received aztreonam for inhalation solution (AZLI) and PTM aztreonam via the SmartMask® Baby, 2 to below 6 years via the SmartMask Kids® and above 6 years via the nebulizer mouthpiece. | 75 mg/ml of aztreonam was administered TID for 28 days via the PARI Altera® Nebulizer System. Participants below 2 years received AZLI via the SmartMask® Baby, 2 to below 6 years via the SmartMask Kids® and above 6 years via the nebulizer mouthpiece. |
Period Title: Overall Study | ||
Started | 74 | 75 |
Completed | 49 | 57 |
Not Completed | 25 | 18 |
Reason Not Completed | ||
Study Terminated by Sponsor | 16 | 12 |
Withdrew Consent by Parent/Guardian | 5 | 4 |
Lost to Follow-up | 1 | 1 |
Adverse Event | 1 | 0 |
Non-Compliance with Study Drug | 1 | 0 |
Protocol Violation | 0 | 1 |
Withdrew Assent | 1 | 0 |
Baseline Characteristics
Arm/Group Title | AZLI 14 Days + Placebo 14 Days | AZLI 28 Days | Total | |
---|---|---|---|---|
![]() |
75 mg/ml of aztreonam was administered TID for 14 days followed by PTM aztreonam TID for 14 days, both aztreonam and PTM aztreonam were delivered via the PARI Altera® Nebulizer System. Participants below 2 years received AZLI and PTM aztreonam via the SmartMask® Baby, 2 to below 6 years via the SmartMask Kids® and above 6 years via the nebulizer mouthpiece. | 75 mg/ml of aztreonam was administered TID for 28 days via the PARI Altera® Nebulizer System. Participants below 2 years received AZLI via the SmartMask® Baby, 2 to below 6 years via the SmartMask Kids® and above 6 years via the nebulizer mouthpiece. | Total of all reporting groups | |
Overall Number of Baseline Participants | 74 | 75 | 149 | |
![]() |
Safety Analysis Set included participants who were randomized and received at least one dose of study drug.
|
|||
Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
||||
Number Analyzed | 74 participants | 75 participants | 149 participants | |
7.3 (5.34) | 6.5 (4.91) | 6.9 (5.13) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
||||
Number Analyzed | 74 participants | 75 participants | 149 participants | |
Female |
35 47.3%
|
33 44.0%
|
68 45.6%
|
|
Male |
39 52.7%
|
42 56.0%
|
81 54.4%
|
|
Race/Ethnicity, Customized
[1] Measure Type: Count of Participants Unit of measure: Participants |
||||
Ethnicity | Number Analyzed | 74 participants | 75 participants | 149 participants |
Not Hispanic or Latino |
66 89.2%
|
65 86.7%
|
131 87.9%
|
|
Hispanic or Latino |
8 10.8%
|
9 12.0%
|
17 11.4%
|
|
Not Permitted |
0 0.0%
|
1 1.3%
|
1 0.7%
|
|
[1]
Measure Description: Not Permitted = collection of ethnicity information not allowed by local regulations.
|
||||
Race/Ethnicity, Customized
Measure Type: Count of Participants Unit of measure: Participants |
||||
Race | Number Analyzed | 74 participants | 75 participants | 149 participants |
American Indian or Alaska Native |
1 1.4%
|
0 0.0%
|
1 0.7%
|
|
Asian |
1 1.4%
|
1 1.3%
|
2 1.3%
|
|
Black or African American |
1 1.4%
|
0 0.0%
|
1 0.7%
|
|
White |
69 93.2%
|
73 97.3%
|
142 95.3%
|
|
Other |
2 2.7%
|
1 1.3%
|
3 2.0%
|
|
Region of Enrollment
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 74 participants | 75 participants | 149 participants |
United Kingdom | 16 | 17 | 33 | |
United States | 16 | 16 | 32 | |
Spain | 6 | 14 | 20 | |
Italy | 8 | 4 | 12 | |
France | 6 | 5 | 11 | |
Denmark | 4 | 5 | 9 | |
Israel | 4 | 4 | 8 | |
Austria | 4 | 3 | 7 | |
Germany | 4 | 3 | 7 | |
Greece | 3 | 1 | 4 | |
Belgium | 3 | 0 | 3 | |
Netherlands | 0 | 3 | 3 |
Outcome Measures
Adverse Events
Limitations and Caveats
The study was terminated early due to Coronavirus disease 2019 (COVID-19) pandemic. At the time of study termination, all evaluable participants completed the initial eradication period and provided data for the primary analysis.
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
After conclusion of the study and without prior written approval from Gilead, investigators in this study may communicate, orally present, or publish in scientific journals or other media only after the following conditions have been met:
- The results of the study in their entirety have been publicly disclosed by or with the consent of Gilead in an abstract, manuscript, or presentation form; or
- The study has been completed at all study sites for at least 2 years
Results Point of Contact
Name/Title: | Gilead Clinical Study Information Center |
Organization: | Gilead Sciences |
Phone: | 1-833-445-3230 (GILEAD-0) |
EMail: | GileadClinicalTrials@gilead.com |
Responsible Party: | Gilead Sciences |
ClinicalTrials.gov Identifier: | NCT03219164 |
Other Study ID Numbers: |
GS-US-205-1850 2016-002749-42 ( EudraCT Number ) |
First Submitted: | July 13, 2017 |
First Posted: | July 17, 2017 |
Results First Submitted: | March 2, 2022 |
Results First Posted: | May 16, 2022 |
Last Update Posted: | June 14, 2022 |