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    Clinical Trial Results:
    A double blind, placebo-controlled study to assess the anti-viral effect, safety and tolerability of inhaled PC786 for the treatment of acute respiratory syncytial virus (RSV) infection in adult hematopoietic stem cell transplant recipients

    Summary
    EudraCT number
    2018-001667-24
    Trial protocol
    GB  
    Global end of trial date
    19 Feb 2019

    Results information
    Results version number
    v1(current)
    This version publication date
    17 May 2020
    First version publication date
    17 May 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    PC_RSV_004
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03715023
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Pulmocide Ltd.
    Sponsor organisation address
    Pulmocide Ltd, 52 Princes Gate, London, United Kingdom, SW7 2PG
    Public contact
    Director of Clinical Development, Pulmocide Ltd, +44 7766250133, Lindsey@pulmocide.com
    Scientific contact
    Director of Clinical Development, Pulmocide Ltd, +44 7766250133, Lindsey@pulmocide.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    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?
    No
    Results analysis stage
    Analysis stage
    Interim
    Date of interim/final analysis
    27 Feb 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    19 Feb 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    19 Feb 2019
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    •To evaluate the anti-viral effect of inhaled PC786 compared with placebo when added to standard of care (SoC) RSV treatment in haematopoietic stem cell transplant (HSCT) recipients with acute RSV infection •To assess the safety and tolerability of 3 days of inhaled PC786 in HSCT subjects with acute RSV infection
    Protection of trial subjects
    This study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki and that are consistent with Good Clinical Practice and applicable regulatory requirements. Known instances of non-conformance were documented and are not considered to have had an impact on the overall conclusions of the study.
    Background therapy
    Subjects received SoC for treatment of RSV at the study site. All medications taken were recorded. Oral ribavirin was permitted at all times on the study. Intravenous ribavirin treatment as SoC may be administered if required, only if a subject was already enrolled in the study. Treatment with intravenous immunoglobulins was permitted throughout the study.
    Evidence for comparator
    -
    Actual start date of recruitment
    11 Dec 2018
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 5
    Worldwide total number of subjects
    5
    EEA total number of subjects
    5
    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)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    5
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Six subjects met all of the eligibility criteria and were randomised to receive treatment at 3 sites in the UK between 11 Dec 2018 and 19 Feb 2019. One subject was withdrawn from the study without being dosed. Five subjects who received 3 doses of PC786 or placebo were therefore included in the safety population.

    Pre-assignment
    Screening details
    It was planned to recruit 30 subjects, with a minimum of 15 subjects to conduct an interim analysis after the winter 18/19 season. A total of six subjects were screened to take part in the study, of which, five subjects were randomised and completed the study.

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Monitor, Assessor
    Blinding implementation details
    Due to a difference in appearance of the active and placebo treatments, the investigational product was prepared and dosed by independent staff team members who did not undertake any other study duties.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    PC786
    Arm description
    Once daily doses of PC786 10mg administered by inhalation via a face mask for a total of 3 doses
    Arm type
    Experimental

    Investigational medicinal product name
    PC786 powder for reconstitution 30mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nebuliser suspension
    Routes of administration
    Inhalation use
    Dosage and administration details
    Nebulised PC786 was administered by inhalation via a facemask at the study site at a dose of 10 mg once daily for three days. In addition to the study drug, subjects were treated according to the SoC at the study site.

    Arm title
    Placebo
    Arm description
    Once daily doses of placebo administered by inhalation via a face mask for a total of 3 doses
    Arm type
    Experimental

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nebuliser solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Nebulised placebo was administered by oral inhalation via a face mask once daily for 3 doses. In addition to the study drug, subjects were treated according to the SoC at the study site.

    Number of subjects in period 1
    PC786 Placebo
    Started
    2
    3
    Completed
    2
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    PC786
    Reporting group description
    Once daily doses of PC786 10mg administered by inhalation via a face mask for a total of 3 doses

    Reporting group title
    Placebo
    Reporting group description
    Once daily doses of placebo administered by inhalation via a face mask for a total of 3 doses

    Reporting group values
    PC786 Placebo Total
    Number of subjects
    2 3 5
    Age categorical
    PC786
    Units: Subjects
        In utero
    0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0
        Newborns (0-27 days)
    0 0 0
        Infants and toddlers (28 days-23 months)
    0 0 0
        Children (2-11 years)
    0 0 0
        Adolescents (12-17 years)
    0 0 0
        Adults (18-64 years)
    2 3 5
        From 65-84 years
    0 0 0
        85 years and over
    0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    41.5 ± 9.19 57.3 ± 6.03 -
    Gender categorical
    Units: Subjects
        Female
    1 1 2
        Male
    1 2 3

    End points

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    End points reporting groups
    Reporting group title
    PC786
    Reporting group description
    Once daily doses of PC786 10mg administered by inhalation via a face mask for a total of 3 doses

    Reporting group title
    Placebo
    Reporting group description
    Once daily doses of placebo administered by inhalation via a face mask for a total of 3 doses

    Subject analysis set title
    PC786
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Pharmacokinetic Parameters for PC786

    Primary: Slope of RSV viral load

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    End point title
    Slope of RSV viral load [1]
    End point description
    Slope of the RSV load over Days 1–3 measured in nasal secretions by reverse transcription quantitative polymerase chain reaction (RT-qPCR)
    End point type
    Primary
    End point timeframe
    Baseline to day 3
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There are no treatment comparisons in this study due to the small sample size (N=5).
    End point values
    PC786 Placebo
    Number of subjects analysed
    2
    3
    Units: Log10 PFUe/mL/day
        arithmetic mean (standard error)
    -1.42 ± 0.193
    -0.73 ± 0.174
    No statistical analyses for this end point

    Secondary: Change in Viral load to Day 7

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    End point title
    Change in Viral load to Day 7
    End point description
    Change in RSV load from baseline to day 7 measured in nasal secretions by reverse transcription quantitative polymerase chain reaction (RT-qPCR)
    End point type
    Secondary
    End point timeframe
    Days 1 to 7
    End point values
    PC786 Placebo
    Number of subjects analysed
    2
    3
    Units: Log 10 PFUe/mL
        arithmetic mean (standard error)
    -4.27 ± 0.58
    -2.70 ± 0.625
    No statistical analyses for this end point

    Secondary: Duration of Viral Shedding

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    End point title
    Duration of Viral Shedding
    End point description
    Duration of viral shedding
    End point type
    Secondary
    End point timeframe
    Day 1 to day 28
    End point values
    PC786 Placebo
    Number of subjects analysed
    2
    3
    Units: day
        arithmetic mean (standard error)
    4.0 ± 2
    19.67 ± 8.33
    No statistical analyses for this end point

    Secondary: Change in viral load to day 4

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    End point title
    Change in viral load to day 4
    End point description
    Change in RSV load from baseline (before first dose of PC786 or placebo) to Day 4
    End point type
    Secondary
    End point timeframe
    Baseline to day 4 am
    End point values
    PC786 Placebo
    Number of subjects analysed
    2
    3
    Units: Log10 PFUe/mL
        arithmetic mean (standard error)
    -4.27 ± 0.58
    -2.18 ± 0.523
    No statistical analyses for this end point

    Secondary: Cmax

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    End point title
    Cmax
    End point description
    Maximum plasma concentration
    End point type
    Secondary
    End point timeframe
    Time 0h to day 28
    End point values
    PC786
    Number of subjects analysed
    2
    Units: pg/mL
        number (not applicable)
    3410
    No statistical analyses for this end point

    Secondary: Tmax

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    End point title
    Tmax
    End point description
    time to maximum concentration
    End point type
    Secondary
    End point timeframe
    time 0h to day 28
    End point values
    PC786
    Number of subjects analysed
    2
    Units: hour
        number (not applicable)
    0.25
    No statistical analyses for this end point

    Secondary: Lower Respiratory Tract Infection/Pneumonia and Oxygen Requirements and Ventilation

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    End point title
    Lower Respiratory Tract Infection/Pneumonia and Oxygen Requirements and Ventilation
    End point description
    Development of LRTI or pneumonia (due to RSV or secondary [bacterial or fungal] infection), Oxygen Requirements and Invasive Ventilation
    End point type
    Secondary
    End point timeframe
    Baseline to Day 28
    End point values
    PC786 Placebo
    Number of subjects analysed
    2
    3
    Units: Percent of Subjects
        Developed Pneumonia
    0
    100
        Increased oxygen saturation index
    0
    67
        Oxygen saturation values <97% on multiple days
    0
    100
        Required invasive ventilation
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Consent until completion of the subject's last study-related procedure (which may include contact for follow-up of safety).
    Adverse event reporting additional description
    All clinically relevant changes (including laboratory safety testing), with the exception of expected signs and symptoms of RSV-related illness, observed during the study were recorded as an AE.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    21.1
    Reporting groups
    Reporting group title
    PC786
    Reporting group description
    Once daily doses of PC786 10mg administered by inhalation via a face mask for a total of 3 doses

    Reporting group title
    Placebo
    Reporting group description
    Once daily doses of placebo administered by inhalation via a face mask for a total of 3 doses

    Serious adverse events
    PC786 Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
    2 / 3 (66.67%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Neutropenic sepsis
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Influenza A virus test
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Superinfection bacterial
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         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: 0%
    Non-serious adverse events
    PC786 Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 2 (100.00%)
    3 / 3 (100.00%)
    Vascular disorders
    Vasodilatation
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Blood magnesium decreased
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    Blood potassium decreased
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    Clostridium test positive
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    Oxygen saturation decreased
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    Cardiac disorders
    Sinus tachycardia
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    0 / 2 (0.00%)
    3 / 3 (100.00%)
         occurrences all number
    0
    3
    Cough
         subjects affected / exposed
    1 / 2 (50.00%)
    2 / 3 (66.67%)
         occurrences all number
    1
    2
    Productive cough
         subjects affected / exposed
    1 / 2 (50.00%)
    1 / 3 (33.33%)
         occurrences all number
    1
    1
    Sneezing
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 2 (0.00%)
    2 / 3 (66.67%)
         occurrences all number
    0
    2
    Ear and labyrinth disorders
    Excessive cerumen production
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Peripheral swelling
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    Pyrexia
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 2 (0.00%)
    2 / 3 (66.67%)
         occurrences all number
    0
    2
    Vomiting
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Dry skin
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    Infections and infestations
    Febrile infection
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    Oral candidiasis
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    Pneumonia
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    Pulmonary mycosis
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? No

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Poor recruitment during the 2018/19 RSV season meant that an interim analysis to adjust the sample size was not possible. The chances of completing enrolment in the study in the 2019/20 RSV season were low and the study was terminated prematurely.
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