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    Clinical Trial Results:
    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

    Summary
    EudraCT number
    		 2016-002749-42
    Trial protocol
    		 BE   GB   IE   AT   FR   DE   ES   NL   IT   GR   DK   Outside EU/EEA  
    Global end of trial date
    23 Sep 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    18 Mar 2022
    First version publication date
    18 Mar 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    GS-US-205-1850
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03219164
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Gilead Sciences
    Sponsor organisation address
    333 Lakeside Drive, Foster City, CA, United States, 94404
    Public contact
    Gilead Clinical Study Information Center, Gilead Sciences, GileadClinicalTrials@gilead.com
    Scientific contact
    Gilead Clinical Study Information Center, Gilead Sciences, GileadClinicalTrials@gilead.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000827-PIP01-09
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    23 Sep 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    27 May 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Sep 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    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.
    Protection of trial subjects
    The protocol and consent/assent forms were submitted by each investigator to a duly constituted Independent Ethics Committee (IEC) or Institutional Review Board (IRB) for review and approval before study initiation. All revisions to the consent/assent forms (if applicable) after initial IEC/IRB approval were submitted by the investigator to the IEC/IRB for review and approval before implementation in accordance with regulatory requirements. This study was conducted in accordance with recognized international scientific and ethical standards, including but not limited to the International Conference on Harmonization guideline for Good Clinical Practice (ICH GCP) and the original principles embodied in the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    28 Nov 2017
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 26 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Austria: 7
    Country: Number of subjects enrolled
    Belgium: 3
    Country: Number of subjects enrolled
    Denmark: 9
    Country: Number of subjects enrolled
    France: 11
    Country: Number of subjects enrolled
    Germany: 7
    Country: Number of subjects enrolled
    Greece: 4
    Country: Number of subjects enrolled
    Israel: 8
    Country: Number of subjects enrolled
    Italy: 12
    Country: Number of subjects enrolled
    Netherlands: 3
    Country: Number of subjects enrolled
    Spain: 20
    Country: Number of subjects enrolled
    United Kingdom: 33
    Country: Number of subjects enrolled
    United States: 32
    Worldwide total number of subjects
    149
    EEA total number of subjects
    76
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    30
    Children (2-11 years)
    85
    Adolescents (12-17 years)
    34
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    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
    Screening details
    		 149 participants were screened.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 AZLI 14 Days + Placebo 14 Days
    Arm description
    		 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.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AZLI
    Investigational medicinal product code
    Other name
    Cayston®, Aztreonam
    Pharmaceutical forms
    Powder and solvent for nebuliser solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    75 mg/ml administered thrice daily.

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solvent for nebuliser solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Administered thrice daily.

    Arm title
    		 AZLI 28 Days
    Arm description
    		 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.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AZLI
    Investigational medicinal product code
    Other name
    Cayston®, Aztreonam
    Pharmaceutical forms
    Powder and solvent for nebuliser solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    75 mg/ml administered thrice daily.

    Number of subjects in period 1
    		AZLI 14 Days + Placebo 14 Days AZLI 28 Days
    Started
    74
    75
    Completed
    49
    57
    Not completed
    25
    18
         Non-Compliance with Study Drug
    1
    -
         Withdrew Consent by Parent/Guardian
    5
    4
         Protocol violation
    -
    1
         Adverse event
    1
    -
         Study Terminated by Sponsor
    16
    12
         Lost to follow-up
    1
    1
         Withdrew Assent
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    AZLI 14 Days + Placebo 14 Days
    Reporting group description
    		 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.

    Reporting group title
    AZLI 28 Days
    Reporting group description
    		 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.

    Reporting group values
    		 AZLI 14 Days + Placebo 14 Days AZLI 28 Days Total
    Number of subjects
    		 74 75 149
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 7.3 ( 5.34 ) 6.5 ( 4.91 ) -
    Gender categorical
    Units: Subjects
        Female
    		 35 33 68
        Male
    		 39 42 81
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 1 0 1
        Asian
    		 1 1 2
        Black or African American
    		 1 0 1
        White
    		 69 73 142
        Other
    		 2 1 3
    Ethnicity
    Not Permitted = collection of ethnicity information not allowed by local regulations.
    Units: Subjects
        Not Hispanic or Latino
    		 66 65 131
        Hispanic or Latino
    		 8 9 17
        Not Permitted
    		 0 1 1

    End points

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    End points reporting groups
    Reporting group title
    AZLI 14 Days + Placebo 14 Days
    Reporting group description
    		 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.

    Reporting group title
    AZLI 28 Days
    Reporting group description
    		 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.

    Subject analysis set title
    Tobramycin Nebulizer Solution (TNS)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The TNS group included historical data for the percentage of participants with PA-negative cultures during 28 days post-treatment period 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.

    Primary: 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

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    End point title
    		 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
    End point description
    Evaluable Analysis Set included participants who completed study drug with at least 75% compliance, did not use any anti-PA antibiotics while on study treatment with AZLI.
    End point type
    Primary
    End point timeframe
    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)
    End point values
    		 AZLI 14 Days + Placebo 14 Days AZLI 28 Days
    Number of subjects analysed
    68
    71
    Units: percentage of participants
        number (confidence interval 95%)
    55.9 (43.3 to 67.9)
    63.4 (51.1 to 74.5)
    Statistical analysis title
    		 AZLI 14 Days + Placebo 14 Days vs AZLI 28 Days
    Comparison groups
    AZLI 14 Days + Placebo 14 Days v AZLI 28 Days
    Number of subjects included in analysis
    139
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority [1]
    Method
    Parameter type
    		 Difference in percentage
    Point estimate
    -8
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -24.6
         upper limit
    		 8.6
    Notes
    [1] - Non-inferiority of the 14-day treatment regimen was claimed if the lower bound of 1-sided 97.5% confidence limit of the treatment difference (14-day course group vs 28-day course group) was above the noninferiority margin of -20%.

    Secondary: Time From Primary Eradication to PA Recurrence Over a 108-Week Post-Treatment Follow-up Period

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    End point title
    		 Time From Primary Eradication to PA Recurrence Over a 108-Week Post-Treatment Follow-up Period
    End point description
    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. Participants in the Evaluable Analysis Set were analyzed. 9999=Not Available as the calculated percentiles of event rate were not reached.
    End point type
    Secondary
    End point timeframe
    Last dose date of AZLI up to Week 112
    End point values
    		 AZLI 14 Days + Placebo 14 Days AZLI 28 Days
    Number of subjects analysed
    68
    71
    Units: months
        median (inter-quartile range (Q1-Q3))
    19.3 (10.5 to 9999)
    9999 (4.9 to 9999)
    No statistical analyses for this end point

    Secondary: 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

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    End point title
    		 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
    End point description
    ELITE study (NCT00391976): Participants with CF who had early PA infection received TNS. ELITE Study Matching Analysis Set=Participants from Evaluable Analysis set who satisfied published criteria for efficacy analysis population: - Participants must be ≥ 6 months at randomization, no history of positive (+) anti-PA antibody (no anti-PA IgG antibody interpretation at Screening/Baseline) on record - Did not use anti-pseudomonal antibiotics and PA negative (-) through 28 days after completion of treatment, within 2 years of Screening - Non-missing PA culture result at 28 days after AZLI last dose - No protocol deviation from study drug administration compliance and documented new onset of + respiratory tract culture for PA within 30 days of Screening [as either first lifetime documented PA+ culture/PA recovered after at least a 2-year history of PA- respiratory cultures (at least 2 cultures/year)] 9999=Not Available as the calculated percentiles of event rate were not reached.
    End point type
    Secondary
    End point timeframe
    Last dose date of AZLI up to Week 112
    End point values
    		 AZLI 14 Days + Placebo 14 Days AZLI 28 Days
    Number of subjects analysed
    23
    25
    Units: months
        median (inter-quartile range (Q1-Q3))
    19.3 (10.5 to 9999)
    15.2 (4.7 to 9999)
    No statistical analyses for this end point

    Secondary: 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)

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    End point title
    		 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) [2]
    End point description
    The historical data for the percentage of participants with PA-negative cultures during 28 days post-treatment period was 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. AZLI 14 Day treatment group: Evaluable Analysis Set; TNS group: Participants with PA-negative cultures during 28 days post-treatment period whose historical data were pooled from published results from studies conducted on the participants with new onset of PA infection and similar TNS treatment duration and follow-up.
    End point type
    Secondary
    End point timeframe
    28 days post treatment (Weeks 4 to 6 for the 14 Day treatment group)
    Notes
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The data was reported for AZLI 14 Day group and for the TNS group historical data 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 was reported.
    End point values
    		 AZLI 14 Days + Placebo 14 Days Tobramycin Nebulizer Solution (TNS)
    Number of subjects analysed
    68
    31
    Units: percentage of participants
        number (not applicable)
    55.9
    77.0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events: First dose date up to 28 days plus 30 days; All-Cause Mortality: Randomization up to 112 weeks
    Adverse event reporting additional description
    Adverse Events: Safety Analysis Set included participants who were randomized and received at least one dose of study drug. All-Cause Mortality: Intent-To-Treat Analysis Set included all participants who were randomized in the study.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.1
    Reporting groups
    Reporting group title
    AZLI 14 Days Placebo 14 Days
    Reporting group description
    		 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.

    Reporting group title
    AZLI 28 Days
    Reporting group description
    		 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.

    Serious adverse events
    		 AZLI 14 Days Placebo 14 Days AZLI 28 Days
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 74 (6.76%)
    4 / 75 (5.33%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Investigations
    Pseudomonas test positive
         subjects affected / exposed
    1 / 74 (1.35%)
    0 / 75 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Congenital, familial and genetic disorders
    Cystic fibrosis
         subjects affected / exposed
    1 / 74 (1.35%)
    0 / 75 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    1 / 74 (1.35%)
    0 / 75 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Subileus
         subjects affected / exposed
    1 / 74 (1.35%)
    0 / 75 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Bronchial obstruction
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Pseudomonas infection
         subjects affected / exposed
    1 / 74 (1.35%)
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Abscess
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    1 / 74 (1.35%)
    0 / 75 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 74 (0.00%)
    1 / 75 (1.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 AZLI 14 Days Placebo 14 Days AZLI 28 Days
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    28 / 74 (37.84%)
    22 / 75 (29.33%)
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    4 / 74 (5.41%)
    6 / 75 (8.00%)
         occurrences all number
    4
    6
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    5 / 74 (6.76%)
    1 / 75 (1.33%)
         occurrences all number
    6
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    12 / 74 (16.22%)
    11 / 75 (14.67%)
         occurrences all number
    13
    15
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 74 (5.41%)
    3 / 75 (4.00%)
         occurrences all number
    4
    3
    Influenza
         subjects affected / exposed
    1 / 74 (1.35%)
    5 / 75 (6.67%)
         occurrences all number
    1
    5
    Rhinitis
         subjects affected / exposed
    5 / 74 (6.76%)
    0 / 75 (0.00%)
         occurrences all number
    5
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    31 Mar 2017
    • All computed tomography (CT) scan assessments were removed • Exploratory objectives and endpoints related to CT scans were removed • Clarified that all retreatment options listed in the protocol were non-exclusive • Clarified that spirometry was only to be performed on participants who can reliably perform spirometry assessments • Clarified that clinical observation should also be performed pre-and poststudy drug administration for participants 6 years of age and older who can’t reliably perform spirometry assessments • Removed collection of body weight, height and vital signs after baseline, with the exception of unscheduled visits • Removed all nasal swabs assessments after Day 1 • Eligibility criteria clarified as needed • Corrected the follow-up culture phase schedule to specify that the first visit will occur at Week 16 and then every twelve weeks after that • Removed hematology and serum chemistry samples from Baseline (if results are available within the last 12 months) and from the follow-up culture phase • Clarified that participants must switch IP kits at Day 15.
    06 Nov 2017
    • The number of study centres were increased from 75 to 85 to mitigate risk of slow recruitment • Exclusion criterion was amended so that antibiotics such as azithromycin were allowable in 7 days prior to Screening • Respiratory sample collection methods expanded • Biomarker sections updated to allow PA-specific antibody titre blood draw to be avoided if results were available within 24 months • Change made to prohibited concomitant medication section to clarify that concurrent use of oral antipseudomonal antibiotics for a respiratory event was prohibited from Screening to Day 28 • Spirometry sections updated to clarify that percentage of forced expiratory volume (FEV1%) predicted was reported at all visits and was calculated by site • Clarification throughout study procedure section as to what types of AEs/SAEs were reported at each visit • Medical History section updated to clarify that the participant`s PA infection history, including the number of previous PA infections, number of cultures taken and sampling methods in the previous 2 years were also recorded.
    15 Apr 2020
    • Clarification on how the missing PA culture data was evaluated • Documentation of which personnel wiould be blinded/unblinded throughout the study • Clarification that 130 evaluable participants were required for the efficacy analyses • Unblinding of Gilead personnel was required for the primary analysis • Confirmed that respiratory samples collected outside of the study as standard of care may be sent to local laboratory • Local lab culture data may be used for analysis purposes • The Biofire testing panel would be used for microbiological assessment of the nasal swabs.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    23 Sep 2021
    During ongoing Coronavirus disease 2019 (COVID-19) pandemic, the risk of continuing the trial in this vulnerable CF pediatric participant population considerably outweighed the benefits of trial continuation. Therefore, the study was terminated early and at the time of study termination, all evaluable participants completed the initial eradication period and provided data for the primary analysis; more than 60% of participants evaluable for the primary analysis also completed 108‑weeks follow‑up period, in line with the terms of the agreed pediatric investigational plan (PIP) for Cayston approved by the Pediatric Committee (PDCO) of the European Medicines Agency (EMA). Throughout the COVID-19 pandemic, exceptional measures were adopted where necessary to mitigate the impact to the conduct of the trial. These measures included: on site participant visits rearranged into remote visits, on site participant visits performed however without all per protocol required study procedures, remote site close out visits and etc. Expected periodic reports detailing all exceptional measures were submitted to applicable National Regulatory Authorities in EU countries where required.
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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