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    Clinical Trial Results:
    A Double-Blind Efficacy and Safety Study of the Phosphodiesterase Type 5 Inhibitor Tadalafil in Pediatric Patients with Pulmonary Arterial Hypertension

    Summary
    EudraCT number
    		 2012-002354-23
    Trial protocol
    		 GB   DE   BE   IT   AT   NL   PL   ES   RO   FR  
    Global end of trial date

    Results information
    Results version number
    		 v1
    This version publication date
    22 Mar 2020
    First version publication date
    22 Mar 2020
    Other versions
    		 v2 , v3

    Trial information

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    Trial identification
    Sponsor protocol code
    H6D-MC-LVHV
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01824290
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 Trial Number: 10609
    Sponsors
    Sponsor organisation name
    Eli Lilly and Company
    Sponsor organisation address
    Lilly Corporate Center, Indianapolis, IN, United States, 46285
    Public contact
    Available Mon ‐ Fri 9 AM ‐ 5 PM EST, Eli Lilly and Company, 1 877‐CTLilly,
    Scientific contact
    Available Mon ‐ Fri 9 AM ‐ 5 PM EST, Eli Lilly and Company, 1 877‐285‐4559,
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000452-PIP02-10
    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
    18 Mar 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    18 Mar 2019
    Global end of trial reached?
    No
    General information about the trial
    Main objective of the trial
    The main purpose of this study is to evaluate the safety and efficacy of tadalafil in pediatric participants with pulmonary arterial hypertension. Participants will receive study treatment for 6 months in the double-blind period (Period 1), and then will be eligible to enroll into an open-label 2 year extension period (Period 2) during which participants will receive tadalafil.
    Protection of trial subjects
    This study was conducted in accordance with International Conference on Harmonization (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.
    Background therapy
    		 Some participants received endothelin receptor agonist (ERA) background therapy (bosentan or ambrisentan).
    Evidence for comparator
    		 -
    Actual start date of recruitment
    05 Feb 2014
    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
    Japan: 2
    Country: Number of subjects enrolled
    Turkey: 3
    Country: Number of subjects enrolled
    Brazil: 15
    Country: Number of subjects enrolled
    Mexico: 5
    Country: Number of subjects enrolled
    Israel: 6
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Germany: 1
    Country: Number of subjects enrolled
    Poland: 1
    Worldwide total number of subjects
    35
    EEA total number of subjects
    4
    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)
    13
    Adolescents (12-17 years)
    22
    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
    		 No Text Available

    Pre-assignment
    Screening details
    		 Per protocol and statistical analysis plan (SAP), the primary and secondary analysis from period 1 were performed to compare all tadalafil participants together versus all placebo participants together. Period 2 data will be reported after study completion.

    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
    		 Placebo
    Arm description
    		 Period 1: Participants received placebo orally by tablets once a day.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Participants received placebo orally by tablets once a day.

    Arm title
    		 Tadalafil
    Arm description
    		 Period 1: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered orally by tablets once a day.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Tadalafil
    Investigational medicinal product code
    Other name
    LY450190
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    20 mg middle weight cohort or 40 mg for heavy weight cohort administered orally by tablets once a day.

    Number of subjects in period 1
    		Placebo Tadalafil
    Started
    18
    17
    Received at least one dose of study drug
    18
    17
    Received 20 mg
    0 [1]
    4 [2]
    Received 40 mg
    0 [3]
    13 [4]
    Completed
    15
    15
    Not completed
    3
    2
         Parent/Caregiver Decision
    1
    1
         Investigator Reported Clinical Worsening
    1
    1
         Entry Criteria Not Met
    1
    -
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: This milestone represents tadalafil arm only.
    [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The milestone represents how many participants received 20 mg tadalafil.
    [3] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: This milestone represents tadalafil arm only.
    [4] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The milestone represents how many participants received 40 mg tadalafil.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Period 1: Participants received placebo orally by tablets once a day.

    Reporting group title
    Tadalafil
    Reporting group description
    		 Period 1: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered orally by tablets once a day.

    Reporting group values
    		 Placebo Tadalafil Total
    Number of subjects
    		 18 17 35
    Age categorical
    Units: Subjects
    Age continuous
    All participants who received at least one dose. Per protocol and statistical analysis plan (SAP), the primary and secondary analysis were performed to compare all tadalafil participants together versus all placebo participants together.
    Units: years
        arithmetic mean (standard deviation)
    		 12.8 ( 3.39 ) 14.1 ( 3.49 ) -
    Gender categorical
    Units: Subjects
        Female
    		 9 10 19
        Male
    		 9 7 16
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 7 8 15
        Not Hispanic or Latino
    		 6 4 10
        Unknown or Not Reported
    		 5 5 10
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 1 3 4
        Asian
    		 1 1 2
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 2 1 3
        White
    		 14 12 26
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 0 0 0
    Region of Enrollment
    Units: Subjects
        Japan
    		 1 1 2
        Turkey
    		 2 1 3
        Brazil
    		 9 6 15
        Mexico
    		 1 4 5
        Israel
    		 2 4 6
        France
    		 1 1 2
        Germany
    		 1 0 1
        Poland
    		 1 0 1
    6 Minute Walk Distance
    Units: Meters
        arithmetic mean (standard deviation)
    		 476.7 ( 105.11 ) 485.8 ( 160.231 ) -

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Period 1: Participants received placebo orally by tablets once a day.

    Reporting group title
    Tadalafil
    Reporting group description
    		 Period 1: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered orally by tablets once a day.

    Subject analysis set title
    20 mg Tadalafil
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Period 1: 20 mg tadalafil administered orally by tablets once a day with concomitant endothelin receptor antagonist (ERA).

    Subject analysis set title
    40 mg Tadalafil
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Period 1: 40 mg tadalafil administered orally by tablets once a day with concomitant endothelin receptor antagonist (ERA).

    Primary: Period 1: Change from Baseline to Week 24 in a 6 Minute Walk (MW) Distance in Meters

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    End point title
    		 Period 1: Change from Baseline to Week 24 in a 6 Minute Walk (MW) Distance in Meters
    End point description
    6MWD in meters assessed in a subset of participants who are ≥6 to <18 years of age who are developmentally capable of performing a 6MW test. Change from baseline was derived using mixed model repeated measures (MMRM) with terms for treatment group, visit, baseline 6MWD, and treatment-by-visit interaction. Analysis Population Description: All participants who received at least one dose of study drug who were > = 6 to < 18 years of age and were capable of performing a 6MW test.
    End point type
    Primary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Placebo Tadalafil
    Number of subjects analysed
    15
    15
    Units: Meters
        least squares mean (standard error)
    36.60 ( 20.776 )
    60.48 ( 20.410 )
    Statistical analysis title
    		 6 Minute Walk (MW) Distance in Meters
    Comparison groups
    Placebo v Tadalafil
    Number of subjects included in analysis
    30
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Mean difference (final values)
    Point estimate
    23.88
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -14.25
         upper limit
    		 62

    Secondary: Period 1: Time to Adjudicated Clinical Worsening (CW)

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    End point title
    		 Period 1: Time to Adjudicated Clinical Worsening (CW)
    End point description
    Clinical worsening was defined as any of the following: death,lung or heart transplantation,atrial septostomy or Potts' shunt,hospitalization for Pulmonary Arterial Hypertension(PAH) progression,new onset syncope,initiation of new PAH therapy(including increase in the dose of existing PAH specific concomitant therapy,such as endothelin receptor agonist or beraprost medication), or increase of 1 or more in World Health Organization(WHO) Functional Class(except for participants already in Class IV;only for participants unable to perform the 6 minute walk(6MW) test;worsening of WHO functional class by 1 or more for participants who can perform a 6 minute walk(6MW) test and who have a decrease of ≥ 20% in the 6 minute walk distance(for those participants who are ≥6 years of age). Criteria for CW(from Period 1) were adjudicated by an independent,blinded study-specific Clinical Endpoint Committee(CEC).This adjudication was used for data analysis, and was not used to guide subject treatment.
    End point type
    Secondary
    End point timeframe
    Baseline through Week 24 Analysis Population Description: All participants who received at least one dose of study drug.
    End point values
    		 Placebo Tadalafil
    Number of subjects analysed
    18 [1]
    17 [2]
    Units: Weeks
        median (confidence interval 95%)
    9999 (9999 to 9999)
    9999 (9999 to 9999)
    Notes
    [1] - 9999=Data Not Available (NA). There was no confirmed adjudicated CW case to report.
    [2] - 9999=Data Not Available (NA). There was no confirmed adjudicated CW case to report.
    No statistical analyses for this end point

    Secondary: Period 1: Percentage of Participants Who Experience CW

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    End point title
    		 Period 1: Percentage of Participants Who Experience CW
    End point description
    Clinical worsening was defined as any of the following: death,lung or heart transplantation,atrial septostomy or Potts' shunt,hospitalization for Pulmonary Arterial Hypertension(PAH) progression,new onset syncope, initiation of new PAH therapy(including increase in the dose of existing PAH specific concomitant therapy,such as endothelin receptor agonist or beraprost medication),or increase of 1 or more in World Health Organization(WHO) Functional Class(except for participants already in Class IV; only for participants unable to perform the 6 minute walk(6MW) test;worsening of WHO functional class by 1 or more for participants who can perform a 6 minute walk(6MW) test and who have a decrease of ≥ 20% in the 6 minute walk distance(for those participants who are ≥6 years of age).Criteria for CW(from Period 1) were adjudicated by an independent,blinded study-specific Clinical Endpoint Committee(CEC).This adjudication was used for data analysis, and was not used to guide subject treatment.
    End point type
    Secondary
    End point timeframe
    Baseline through Week 24 Analysis Population Description: All participants who received at least one dose of study drug.
    End point values
    		 Placebo Tadalafil
    Number of subjects analysed
    18
    17
    Units: percentage of participants
        number (not applicable)
    0
    0
    No statistical analyses for this end point

    Secondary: Period 1: Pharmacokinetics (PK): Apparent Clearance (CL/F) of tadalafil

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    End point title
    		 Period 1: Pharmacokinetics (PK): Apparent Clearance (CL/F) of tadalafil
    End point description
    Period 1: Pharmacokinetics (PK): Apparent Clearance (CL/F) of Tadalafil at Steady-state. Analysis Population Description: All participants who received at least one dose of study drug and had evaluable PK data. 9999= NA. For 20 mg tadalafil with concomitant bosentan, n=3. For 20 mg tadalafil no bosentan (ERA: macitentan), n = 1. For n = 1, geometric mean and geometric coefficient of variation could not be calculated. Individual value is 2.68. For 40 mg tadalafil No bosentan (ERA: Macitentan), n=0.
    End point type
    Secondary
    End point timeframe
    Week 2, Week 4, Week16 and Week 24
    End point values
    		 20 mg Tadalafil 40 mg Tadalafil
    Number of subjects analysed
    4 [3]
    13 [4]
    Units: Liter Per Hour (L/hr)
    geometric mean (geometric coefficient of variation)
        With concomitant bosentan
    3.63 ( 38.1 )
    4.49 ( 28.2 )
        No bosentan (ERA: Macitentan)
    9999 ( 9999 )
    9999 ( 9999 )
    Notes
    [3] - 9999= NA. For 20 mg tadalafil No bosentan (ERA: Macitentan), n = 1.
    [4] - 9999= NA. For 40 mg tadalafil No bosentan (ERA: Macitentan), n=0.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Period 1: Up To 24 Weeks
    Adverse event reporting additional description
    All participants who received at least one dose of study drug. Period 2 data will be reported at study completion.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    21.1
    Reporting groups
    Reporting group title
    Tadalafil
    Reporting group description
    		 Period 1: 20 mg or 40 mg administered orally by tablets once a day.

    Reporting group title
    Placebo
    Reporting group description
    		 Period 1: Participants received placebo orally by tablets once a day.

    Serious adverse events
    		 Tadalafil Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 18 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Tadalafil Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 17 (88.24%)
    8 / 18 (44.44%)
    Vascular disorders
    flushing
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    hypotension
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    pyrexia
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Social circumstances
    menarche
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed [1]
    0 / 10 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Reproductive system and breast disorders
    menorrhagia
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed [2]
    1 / 10 (10.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    penis disorder
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed [3]
    1 / 7 (14.29%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    spontaneous penile erection
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed [4]
    1 / 7 (14.29%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Respiratory, thoracic and mediastinal disorders
    epistaxis
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 18 (5.56%)
         occurrences all number
    2
    1
    oropharyngeal pain
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    pulmonary arterial hypertension
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Investigations
    hepatic enzyme increased
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    bone contusion
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Cardiac disorders
    palpitations
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    tachycardia
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    headache
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    5 / 17 (29.41%)
    2 / 18 (11.11%)
         occurrences all number
    5
    6
    presyncope
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    somnolence
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Blood and lymphatic system disorders
    eosinophilia
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    haemorrhage subcutaneous
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    3
    0
    livedo reticularis
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    rash
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    swelling face
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    arthralgia
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 18 (5.56%)
         occurrences all number
    2
    1
    back pain
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    bronchitis
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    influenza
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    3 / 17 (17.65%)
    0 / 18 (0.00%)
         occurrences all number
    3
    0
    pharyngitis
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    rhinitis
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    sinusitis
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    upper respiratory tract infection
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    3 / 17 (17.65%)
    1 / 18 (5.56%)
         occurrences all number
    3
    1
    urinary tract infection
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 18 (5.56%)
         occurrences all number
    1
    1
    vaginal infection
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed [5]
    1 / 10 (10.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    viral tonsillitis
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    viral upper respiratory tract infection
    alternative dictionary used: MedDRA 21.1
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    Notes
    [1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: This event is gender specific, only occurring in male or female subjects. The number of subjects exposed has been adjusted accordingly.
    [2] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: This event is gender specific, only occurring in male or female subjects. The number of subjects exposed has been adjusted accordingly.
    [3] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: This event is gender specific, only occurring in male or female subjects. The number of subjects exposed has been adjusted accordingly.
    [4] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: This event is gender specific, only occurring in male or female subjects. The number of subjects exposed has been adjusted accordingly.
    [5] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: This event is gender specific, only occurring in male or female subjects. The number of subjects exposed has been adjusted accordingly.

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    27 Sep 2012
    Change of primary endpoint for US from right heart catheterization (RHC) to Cardiac Echo. Per requirement from FDA that RHC can no longer be used as primary endpoint in Pediatric PAH trials.
    14 Dec 2012
    Per FDA removal of Tricuspid Annular Plane Systolic Excursion (TAPSE) as primary endpoint for US. Change of primary endpoint for US to improving time of 6-minute walk (6MW) test from baseline to week 24.
    13 Dec 2018
    Change of primary endpoint and study sample size.

    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.
    The study is mainly descriptive in a small number of children with PAH and there were no participants enrolled in the light weight cohort.
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