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    Clinical Trial Results:
    A Randomized, Double-Blind, Placebo-Controlled Phase II Study to Evaluate the Efficacy and Safety of SPX-101 Inhalation Solution in Subjects with Cystic Fibrosis (HOPE-1 STUDY: HYDRATION FOR OPTIMAL PULMONARY EFFECTIVENESS)

    Summary
    EudraCT number
    		 2016-005230-30
    Trial protocol
    		 GB   PT   IT  
    Global end of trial date
    24 Jun 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    21 Sep 2019
    First version publication date
    21 Sep 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    SPX-101-CF-201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03229252
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Spyryx Biosciences, Inc.
    Sponsor organisation address
    801-10 Capitola Drive, Durham, United States, 27713
    Public contact
    Dr. Rob Tarran, Spyryx Biosciences, Inc., +1 9199667052, contact@spyryxbio.com
    Scientific contact
    Dr. Rob Tarran, Spyryx Biosciences, Inc., +1 9199667052, contact@spyryxbio.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
    Final
    Date of interim/final analysis
    02 Aug 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    24 Jun 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    24 Jun 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of SPX-101 in subjects with CF
    Protection of trial subjects
    None needed
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Aug 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Portugal: 8
    Country: Number of subjects enrolled
    United Kingdom: 36
    Country: Number of subjects enrolled
    France: 27
    Country: Number of subjects enrolled
    Italy: 4
    Country: Number of subjects enrolled
    Canada: 16
    Worldwide total number of subjects
    91
    EEA total number of subjects
    75
    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)
    91
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Recruiting began in October, 2017 and ended in June, 2019. The trial was run in Canada, United Kingdom, France, Italy and Portugal.

    Pre-assignment
    Screening details
    		 Subjects were screened at Visit 1 and then entered a variable-length screening period of 3 to 28 days, to time the randomization so that the first dose of the study drug would coincide, if applicable, to the start of an inhaled antibiotic cycle.

    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, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 SPX-101 Low Dose
    Arm description
    		 Inhalation solution twice daily for 28-days. SPX-101 inhalation solution.
    Arm type
    		 Experimental

    Investigational medicinal product name
    SPX-101
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Inhalation solution twice daily for 28-days

    Arm title
    		 SPX-101 High Dose
    Arm description
    		 Inhalation solution twice daily for 28-days. SPX-101 inhalation solution.
    Arm type
    		 Experimental

    Investigational medicinal product name
    SPX-101
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Inhalation solution twice daily for 28-days

    Arm title
    		 Placebo
    Arm description
    		 Placebo inhalation solution twice daily for 28-days. Normal saline inhalation solution.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Normal Saline
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Inhalation solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    Normal saline inhalation solution

    Number of subjects in period 1
    		SPX-101 Low Dose SPX-101 High Dose Placebo
    Started
    15
    45
    31
    Completed
    15
    39
    29
    Not completed
    0
    6
    2
         Consent withdrawn by subject
    -
    -
    1
         Adverse event, non-fatal
    -
    6
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    SPX-101 Low Dose
    Reporting group description
    		 Inhalation solution twice daily for 28-days. SPX-101 inhalation solution.

    Reporting group title
    SPX-101 High Dose
    Reporting group description
    		 Inhalation solution twice daily for 28-days. SPX-101 inhalation solution.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo inhalation solution twice daily for 28-days. Normal saline inhalation solution.

    Reporting group values
    		 SPX-101 Low Dose SPX-101 High Dose Placebo Total
    Number of subjects
    		 15 45 31 91
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 15 45 31 91
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 33.7 ( 8.62 ) 31.9 ( 8.49 ) 30.8 ( 7.82 ) -
    Gender categorical
    Units: Subjects
        Female
    		 4 22 11 37
        Male
    		 11 23 20 54

    End points

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    End points reporting groups
    Reporting group title
    SPX-101 Low Dose
    Reporting group description
    		 Inhalation solution twice daily for 28-days. SPX-101 inhalation solution.

    Reporting group title
    SPX-101 High Dose
    Reporting group description
    		 Inhalation solution twice daily for 28-days. SPX-101 inhalation solution.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo inhalation solution twice daily for 28-days. Normal saline inhalation solution.

    Primary: Change in percent predicted FEV1

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    End point title
    		 Change in percent predicted FEV1
    End point description
    End point type
    Primary
    End point timeframe
    From baseline to day 28
    End point values
    		 SPX-101 Low Dose SPX-101 High Dose Placebo
    Number of subjects analysed
    15
    45
    31
    Units: Change in baseline in ppFEV1
        arithmetic mean (standard deviation)
    0.800 ( 6.5049 )
    0.890 ( 6.7061 )
    1.633 ( 6.3302 )
    Statistical analysis title
    		 ANCOVA
    Statistical analysis description
    The comparison between each treatment group and placebo will be evaluated by using an analysis of covariance (ANCOVA) model, which will include treatment as the factor, and the stratification factor as a covariate (i.e., baseline lung function categories [ppFEV1 40.0% to 55.0% or 55.1% to 80.0% for Cohort 1] and the concomitant use of hypertonic saline for Cohort 2). Also included in the ANCOVA model for Cohort 2 is baseline ppFEV1. Descriptive statistics will include the number of observations,
    Comparison groups
    SPX-101 Low Dose v SPX-101 High Dose v Placebo
    Number of subjects included in analysis
    91
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.1
    Method
    		 ANCOVA
    Confidence interval

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    screening to day 30
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.0
    Reporting groups
    Reporting group title
    SPX-101 Low Dose
    Reporting group description
    		 -

    Reporting group title
    SPX-101 High Dose
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Serious adverse events
    		 SPX-101 Low Dose SPX-101 High Dose Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 15 (0.00%)
    3 / 45 (6.67%)
    1 / 31 (3.23%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 45 (2.22%)
    0 / 31 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Intestinal Obstruction
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 45 (0.00%)
    1 / 31 (3.23%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Cough and Dyspnea
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 45 (2.22%)
    0 / 31 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Infective Pulmonary Exacerbation of Cystic Fibrosis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 45 (2.22%)
    0 / 31 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 SPX-101 Low Dose SPX-101 High Dose Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 15 (66.67%)
    29 / 45 (64.44%)
    20 / 31 (64.52%)
    Investigations
    Forced expiratory volume decreased
         subjects affected / exposed
    0 / 15 (0.00%)
    4 / 45 (8.89%)
    0 / 31 (0.00%)
         occurrences all number
    0
    4
    0
    Nervous system disorders
    Dysgeusia
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 45 (0.00%)
    2 / 31 (6.45%)
         occurrences all number
    0
    0
    2
    Headache
         subjects affected / exposed
    0 / 15 (0.00%)
    4 / 45 (8.89%)
    1 / 31 (3.23%)
         occurrences all number
    0
    4
    1
    Migraine
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 45 (0.00%)
    0 / 31 (0.00%)
         occurrences all number
    1
    0
    0
    General disorders and administration site conditions
    Chest discomfort
         subjects affected / exposed
    2 / 15 (13.33%)
    2 / 45 (4.44%)
    2 / 31 (6.45%)
         occurrences all number
    2
    3
    2
    Fatigue
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 45 (0.00%)
    2 / 31 (6.45%)
         occurrences all number
    0
    0
    2
    Pyrexia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 45 (0.00%)
    0 / 31 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 45 (4.44%)
    0 / 31 (0.00%)
         occurrences all number
    1
    2
    0
    Diarrhoea
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 45 (4.44%)
    1 / 31 (3.23%)
         occurrences all number
    1
    2
    1
    Frequent bowel movements
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 45 (0.00%)
    0 / 31 (0.00%)
         occurrences all number
    1
    0
    0
    Tongue coated
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 45 (0.00%)
    0 / 31 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Bronchospasm
         subjects affected / exposed
    0 / 15 (0.00%)
    5 / 45 (11.11%)
    0 / 31 (0.00%)
         occurrences all number
    0
    5
    0
    Cough
         subjects affected / exposed
    4 / 15 (26.67%)
    6 / 45 (13.33%)
    6 / 31 (19.35%)
         occurrences all number
    4
    6
    6
    Dyspnoea
         subjects affected / exposed
    0 / 15 (0.00%)
    4 / 45 (8.89%)
    1 / 31 (3.23%)
         occurrences all number
    0
    4
    1
    Epistaxis
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 45 (4.44%)
    0 / 31 (0.00%)
         occurrences all number
    1
    2
    0
    Increased viscosity of bronchial secretion
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 45 (0.00%)
    0 / 31 (0.00%)
         occurrences all number
    1
    0
    0
    Infective exacerbation of cystic fibrosis
         subjects affected / exposed
    1 / 15 (6.67%)
    3 / 45 (6.67%)
    4 / 31 (12.90%)
         occurrences all number
    1
    3
    4
    Lower respiratory tract congestion
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 45 (0.00%)
    2 / 31 (6.45%)
         occurrences all number
    0
    0
    2
    Nasal congestion
         subjects affected / exposed
    3 / 15 (20.00%)
    0 / 45 (0.00%)
    0 / 31 (0.00%)
         occurrences all number
    3
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 15 (0.00%)
    3 / 45 (6.67%)
    2 / 31 (6.45%)
         occurrences all number
    0
    3
    2
    Sputum discoloured
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 45 (0.00%)
    0 / 31 (0.00%)
         occurrences all number
    1
    0
    0
    Sputum increased
         subjects affected / exposed
    3 / 15 (20.00%)
    4 / 45 (8.89%)
    2 / 31 (6.45%)
         occurrences all number
    3
    4
    2
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 45 (0.00%)
    0 / 31 (0.00%)
         occurrences all number
    2
    0
    0
    Wheezing
         subjects affected / exposed
    1 / 15 (6.67%)
    4 / 45 (8.89%)
    0 / 31 (0.00%)
         occurrences all number
    2
    7
    0
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 45 (0.00%)
    0 / 31 (0.00%)
         occurrences all number
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    14 Jun 2017
    Added procedures for emergency unblinding to protocol
    14 May 2018
    Removal of PK blood draw and update to randomization ratios

    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.
    None reported
    For support, Contact us.
    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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