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    Clinical Trial Results:
    A Phase I, open-label, multi-centre study to determine the Pharmacokinetics and safety of solithromycin as add-on therapy in adolescents and children with suspected or confirmed bacterial infection.

    Summary
    EudraCT number
    		 2014-004041-26
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    12 Oct 2016

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 Jan 2018
    First version publication date
    25 Jan 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CE01-120
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02268279
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Cempra Pharmaceuticals
    Sponsor organisation address
    6320 Quadrangle Drive, Suite 360, Chapel Hill, United States, 27517
    Public contact
    clinicaltrials@melinta.com, Cempra Pharmaceuticals, Inc, clinicaltrials@melinta.com
    Scientific contact
    Clinical Trials Info, Cempra Pharmaceuticals, Inc, clinicaltrials@melinta.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001581-PIP01-13
    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
    07 Aug 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    12 Oct 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Oct 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Determine the safety and PK profile of solithromycin (IV and oral) in the paediatric population.
    Protection of trial subjects
    Solithromycin was administered orally and IV based on weight as add-on to antimicrobial agents administered per routine standard of care to adolescents and children with a suspected or confirmed infection with organisms against which solithromycin was expected to be active.
    Background therapy
    		 The most frequently used concomitant medications included dornase alfa, fluticasone, salbutamol, piperacillin, amoxicillin, ampicillin, azithromycin, cefepime, clindamycin, vancomycin, ceftriaxone, acetaminophen, sodium chloride.
    Evidence for comparator
    		 Not applicable
    Actual start date of recruitment
    03 Mar 2015
    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
    Bulgaria: 13
    Country: Number of subjects enrolled
    United States: 71
    Worldwide total number of subjects
    84
    EEA total number of subjects
    13
    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)
    23
    Children (2-11 years)
    51
    Adolescents (12-17 years)
    10
    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
    		 Patients were enrolled from 34 study sites: 31 in the United States and 3 in Bulgaria.

    Pre-assignment
    Screening details
    		 1. < 18 years of age 2. Suspected or confirmed bacterial infection with organisms against which solithromycin was expected to be active 3. No consumption of Seville oranges, grapefruit 7 days before the first dose of study drug 4. Serum creatinine < 2 mg/dL 5. No hepatic dysfunction (ALT or AST > 3 x upper normal limit or direct bilirubin > ULN

    Period 1
    Period 1 title
    All subject (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Oral suspension formulation
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    solithromycin
    Investigational medicinal product code
    CEM-101
    Other name
    Pharmaceutical forms
    Powder for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    15 mg/kg/day (0 to < 12 years) for up to 5 days. The maximum Day 1 dose could not exceed 800 mg, and Days 2-5 could not exceed 400 mg.

    Arm title
    		 IV formulation
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    solithromycin
    Investigational medicinal product code
    CEM-101
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    6 mg/kg/day (12 to < 18 years), 7 mg/kg/day (6 to < 12 years or < 1 month), 8 mg/kg/day (2 to < 6 years or 1 month to < 2 years) for up to 5 days.

    Arm title
    		 Oral capsule formulation
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    solithromycin
    Investigational medicinal product code
    CEM-101
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    15 mg/kg/day (6 to < 12 years) for up to 5 days. The maximum Day 1 could not exceed 800 mg, and Days 2-5 doses could not exceed 400 mg.

    Number of subjects in period 1
    		Oral suspension formulation IV formulation Oral capsule formulation
    Started
    40
    34
    10
    Completed
    40
    34
    10

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    All subject
    Reporting group description
    		 -

    Reporting group values
    		 All subject Total
    Number of subjects
    		 84 84
    Age categorical
    Units: Subjects
        Adolescents (12-17 years)
    		 10 10
        6 to < 12 years
    		 34 34
        2 to < 6 years
    		 17 17
        0 to < 2 years
    		 23 23
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 5.9 ± 4.6 -
    Gender categorical
    Units: Subjects
        Female
    		 41 41
        Male
    		 43 43

    End points

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    End points reporting groups
    Reporting group title
    Oral suspension formulation
    Reporting group description
    		 -

    Reporting group title
    IV formulation
    Reporting group description
    		 -

    Reporting group title
    Oral capsule formulation
    Reporting group description
    		 -

    Primary: IV dose determination

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    End point title
    		 IV dose determination [1] [2]
    End point description
    End point type
    Primary
    End point timeframe
    For the IV formulation, PK samples collected on Days 1, 3, 4 and 5. For the IV-to-PO switch: PK samples collected on Day 1, last day of IV dosing, first day of PO dosing and last day of PO dosing.
    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: Data from this trial were merged with adolescent PK data from CE01-119 study, and a population PK analysis was performed using the software NONMEM. PK simulation were performed to guide recommendations for the IV dose determination.
    [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: Data from this trial were merged with adolescent PK data from CE01-119 study, and a population PK analysis was performed using the software NONMEM. PK simulation were performed to guide recommendations for the IV dose determination.
    End point values
    		 IV formulation
    Number of subjects analysed
    34
    Units: mg/kg
        patients ages 12 to < 17 years
    8
        patients ages 6 to < 12 years
    8
        patients ages 2 to < 6 years
    8
        patients ages 0 to < 2 years
    8
    No statistical analyses for this end point

    Primary: Capsule loading dose determination

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    End point title
    		 Capsule loading dose determination [3] [4]
    End point description
    End point type
    Primary
    End point timeframe
    PK samples collected on Days 1, 3, 4 and 5.
    Notes
    [3] - 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: Data from this trial were merged with adolescent PK data from CE01-119 study, and a population PK analysis was performed using the software NONMEM. PK simulation were performed to guide recommendations for the capsule loading dose determination.
    [4] - 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: Data from this trial were merged with adolescent PK data from CE01-119 study, and a population PK analysis was performed using the software NONMEM. PK simulation were performed to guide recommendations for the capsule dose determination.
    End point values
    		 Oral capsule formulation
    Number of subjects analysed
    10
    Units: mg/kg
        patients ages 6 to < 12 years
    20
    No statistical analyses for this end point

    Primary: Capsule maintenance dose determination

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    End point title
    		 Capsule maintenance dose determination [5] [6]
    End point description
    End point type
    Primary
    End point timeframe
    PK samples collected on Days 1, 3, 4 and 5.
    Notes
    [5] - 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: Data from this trial were merged with adolescent PK data from CE01-119 study, and a population PK analysis was performed using the software NONMEM. PK simulation were performed to guide recommendations for the capsule dose determination.
    [6] - 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: Data from this trial were merged with adolescent PK data from CE01-119 study, and a population PK analysis was performed using the software NONMEM. PK simulation were performed to guide recommendations for the capsule maintenance dose determination.
    End point values
    		 Oral capsule formulation
    Number of subjects analysed
    10
    Units: mg/kg
        patients ages 6 to < 12 years
    10
    No statistical analyses for this end point

    Primary: Oral suspension loading dose determination

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    End point title
    		 Oral suspension loading dose determination [7] [8]
    End point description
    End point type
    Primary
    End point timeframe
    PK samples collected on Days 1, 3, 4 and 5.
    Notes
    [7] - 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: Data from this trial were merged with adolescent PK data from CE01-119 study, and a population PK analysis was performed using the software NONMEM. PK simulation were performed to guide recommendations for the oral suspension loading dose determination.
    [8] - 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: Data from this trial were merged with adolescent PK data from CE01-119 study, and a population PK analysis was performed using the software NONMEM. PK simulation were performed to guide recommendations for the oral suspension loading dose determination from infants to adolescents (0-17 years).
    End point values
    		 Oral suspension formulation
    Number of subjects analysed
    40
    Units: mg/kg
        patients ages 6 to < 12 years
    20
        patients ages 2 to < 6 years
    20
        patients ages 0 to < 2 years
    20
    No statistical analyses for this end point

    Primary: Oral suspension maintenance dose determination

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    End point title
    		 Oral suspension maintenance dose determination [9] [10]
    End point description
    End point type
    Primary
    End point timeframe
    PK samples collected on Days 1, 3, 4 and 5.
    Notes
    [9] - 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: Data from this trial were merged with adolescent PK data from CE01-119 study, and a population PK analysis was performed using the software NONMEM. PK simulation were performed to guide recommendations for the oral suspension maintenance dose determination.
    [10] - 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: Data from this trial were merged with adolescent PK data from CE01-119 study, and a population PK analysis was performed using the software NONMEM. PK simulation were performed to guide recommendations for the oral suspension maintenance dose determination from infants to adolescents (0-17 years).
    End point values
    		 Oral suspension formulation
    Number of subjects analysed
    40
    Units: mg/kg
        patients ages 6 to < 12 years
    10
        patients ages 2 to < 6 years
    10
        patients ages 0 to < 2 years
    10
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first study drug administration to late follow-up visit (day 14 +/- 4).
    Adverse event reporting additional description
    If for any reason the patient could not return for the last follow-up visit and have the study procedures performed, the study staff were to contact the patient/parent/legally authorized representative (LAR) by telephone or other interactive technology.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Safety population
    Reporting group description
    		 All patients who received at least 1 dose of study drug.

    Serious adverse events
    		 Safety population
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 84 (3.57%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Cardiac disorders
    Atrial tachycardia
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Device occlusion
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pyrexia
    Additional description: This SAE was experienced by the same patient who had a device occlusion.
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 2.3%
    Non-serious adverse events
    		 Safety population
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    40 / 84 (47.62%)
    Investigations
    Hepatic enzyme increased
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2
    Injury, poisoning and procedural complications
    Procedural pain
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 84 (4.76%)
         occurrences all number
    5
    General disorders and administration site conditions
    Device occlusion
         subjects affected / exposed
    3 / 84 (3.57%)
         occurrences all number
    3
    infusion site pain
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    3
    Infusion site phlebitis
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2
    medical device complication
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2
    pyrexia
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    6
    Vomiting
         subjects affected / exposed
    3 / 84 (3.57%)
         occurrences all number
    3
    Abdominal pain
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2
    Constipation
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2
    Nausea
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2
    Skin and subcutaneous tissue disorders
    Skin irritation
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences all number
    2

    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.
    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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