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    Clinical Trial Results:
    An open-label extension study to CQTI571A2102 to evaluate the long-term safety, tolerability and efficacy of QTI571 (imatinib) in the treatment of severe pulmonary arterial hypertension

    Summary
    EudraCT number
    2010-021960-14
    Trial protocol
    DE   IT  
    Global end of trial date
    26 Mar 2014

    Results information
    Results version number
    v1(current)
    This version publication date
    13 Jul 2016
    First version publication date
    01 Aug 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CQTI571A2102E1
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01392495
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
    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
    26 Mar 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Mar 2014
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    • To evaluate the long-term safety and tolerability of QTI571 in patients with severe pulmonary arterial hypertension
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial. Subjects who experience worsening of PAH requiring hospitalization may require the use of diuretics and supplemental oxygen. If severe, subjects may require intensive care treatment and use of vasopressors. PAH therapies, not used at time of enrollment, such as subcutaneous, oral, inhaled or intravenous prostacyclin derivatives, should only be added if the subject meets a TTCW event, including overnight hospitalization for worsening of PAH, worsening of WHO functional class by one or more levels, or a decrease in 6MWD of 15% measured on two occasions. Subjects who have a TTCW event may remain in the study and perform assessments per the visit schedule.
    Background therapy
    Specific PAH medications include: all endothelin receptor antagonists, phosphodiesterase 5 inhibitors and inhaled, oral, intravenous, or subcutaneous prostacyclin analogues. Background PAH therapies include: oxygen, digoxin, all diuretics, and calcium channel blockers. Background PAH therapies may be adjusted as necessary during the study.
    Evidence for comparator
    Open label
    Actual start date of recruitment
    22 Jun 2011
    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
    Australia: 3
    Country: Number of subjects enrolled
    United States: 1
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Germany: 8
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Lithuania: 2
    Worldwide total number of subjects
    17
    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)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    12
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study population was composed of patients with PAH who completed the CQTI571A2102 study, did not meet withdrawal criteria for safety reasons during study conduct, and met the eligibility criteria for the current study.

    Pre-assignment
    Screening details
    Subjects were enrolled from the Core study CQTI571A2102.

    Period 1
    Period 1 title
    Open Label 400 or 200 mg (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    QTI571 200 mg
    Arm description
    QTI571 200 mg (highest tolerated dose in CQTI571A2102).
    Arm type
    Experimental

    Investigational medicinal product name
    Imatinib mesylate
    Investigational medicinal product code
    QTI571
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Patients were instructed to take 2 100 mg tablets (highest tolerated dose in core study) once daily with a meal and a 200 mL glass of water and to swallow the tablet whole. If at any time, the dose of 200 mg q.d was not tolerated, treatment was to be discontinued and patient was to be discontinued from the study. Dosing was to be stopped immediately for unacceptable values (defined in protocol) of thrombocytopenia, neutropenia, all events of syncope, receipt of lung transplant, and significant arrhythmia or LV dysfunction.

    Arm title
    QTI571 400 mg
    Arm description
    QTI571 400 mg (highest tolerated dose in CQTI571A2102).
    Arm type
    Experimental

    Investigational medicinal product name
    Imatinib mesylate
    Investigational medicinal product code
    QTI571
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Patients were instructed to take 4 100 mg tablets (highest tolerated dose in core study) once daily with a meal and a 200 mL glass of water and to swallow the tablet whole. For patients unable to tolerate 400mg q.d, based on specific criteria outlined in the protocol, a dose reduction to 200mg q.d was to have taken place . If criteria persisted after two weeks of dose reduction, patient was to have been discontinued. After a dose reduction, dose escalation back to 400 mg was permitted if it was safe to do so in the investigator’s clinical judgment. Dosing was to be stopped immediately for unacceptable values (defined in protocol) of thrombocytopenia, neutropenia, all events of syncope, receipt of lung transplant, and significant arrhythmia or LV dysfunction.

    Number of subjects in period 1
    QTI571 200 mg QTI571 400 mg
    Started
    4
    13
    Completed
    0
    1
    Not completed
    4
    12
         Death
    2
    1
         Administrative problems
    1
    7
         Adverse event, non-fatal
    1
    2
         Consent withdrawn by subject
    -
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Open Label 400 or 200 mg
    Reporting group description
    -

    Reporting group values
    Open Label 400 or 200 mg Total
    Number of subjects
    17 17
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    12 12
        From 65-84 years
    5 5
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    53.5 ± 14.3 -
    Gender categorical
    Units: Subjects
        Female
    11 11
        Male
    6 6

    End points

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    End points reporting groups
    Reporting group title
    QTI571 200 mg
    Reporting group description
    QTI571 200 mg (highest tolerated dose in CQTI571A2102).

    Reporting group title
    QTI571 400 mg
    Reporting group description
    QTI571 400 mg (highest tolerated dose in CQTI571A2102).

    Primary: Number of patients with adverse events, serious adverse events and deaths

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    End point title
    Number of patients with adverse events, serious adverse events and deaths [1]
    End point description
    Adverse event monitoring was conducted throughout the trial. Safety Analysis Set: The safety analysis set included all participants who received at least one dose of study drug during the extension and had at least one post-baseline safety assessment.
    End point type
    Primary
    End point timeframe
    144 weeks
    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: Study was terminated and statistical analysis was not performed.
    End point values
    QTI571 200 mg QTI571 400 mg
    Number of subjects analysed
    4
    13
    Units: Patients
        Adverse Events (serious and non-serious)
    4
    13
        Serious Adverse Events
    4
    5
        Deaths
    2
    1
    No statistical analyses for this end point

    Secondary: Change from baseline in the six minute walk distance (6MWD)

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    End point title
    Change from baseline in the six minute walk distance (6MWD)
    End point description
    Efficacy outcomes were not powered for statistical analysis due to insufficient data resulting from early termination. No data displayed because Outcome Measure has zero total participants analyzed.
    End point type
    Secondary
    End point timeframe
    baseline, 144 weeks
    End point values
    QTI571 200 mg QTI571 400 mg
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: Meters
        arithmetic mean (standard deviation)
    ±
    ±
    Notes
    [2] - Efficacy outcomes were not powered for statistical analysis due to early termination of trial.
    [3] - No data displayed because Outcome Measure has zero total participants analyzed.
    No statistical analyses for this end point

    Secondary: Time to clinical worsening (TTCW) endpoints

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    End point title
    Time to clinical worsening (TTCW) endpoints
    End point description
    Efficacy outcomes were not powered for statistical analysis due to insufficient data resulting from early termination. No data displayed because Outcome Measure has zero total participants analyzed.
    End point type
    Secondary
    End point timeframe
    144 weeks
    End point values
    QTI571 200 mg QTI571 400 mg
    Number of subjects analysed
    0 [4]
    0 [5]
    Units: events
    Notes
    [4] - No data displayed because Outcome Measure has zero total participants analyzed.
    [5] - No data displayed because Outcome Measure has zero total participants analyzed.
    No statistical analyses for this end point

    Secondary: Medical resource utilization

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    End point title
    Medical resource utilization
    End point description
    Efficacy outcomes were not powered for statistical analysis due to insufficient data resulting from termination of the study. No data displayed because Outcome Measure has zero total participants analyzed.
    End point type
    Secondary
    End point timeframe
    144 weeks
    End point values
    QTI571 200 mg QTI571 400 mg
    Number of subjects analysed
    0 [6]
    0 [7]
    Units: Events
    Notes
    [6] - No data displayed because Outcome Measure had zero total participants analyzed.
    [7] - No data displayed because Outcome Measure had zero total participants analyzed.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). 
    Adverse event reporting additional description
    Consistent with EudraCT disclosure specifications, Novartis has reported under the Serious adverse events field “number of deaths resulting from adverse events” all those deaths, resulting from serious adverse events that are deemed to be causally related to treatment by the investigator.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    16.1
    Reporting groups
    Reporting group title
    QTI571 200mg
    Reporting group description
    QTI571 200mg

    Reporting group title
    QTI571 400mg
    Reporting group description
    QTI571 400mg

    Serious adverse events
    QTI571 200mg QTI571 400mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 4 (100.00%)
    5 / 13 (38.46%)
         number of deaths (all causes)
    2
    1
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Adenocarcinoma of colon
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Right ventricular failure
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary mass
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Death
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Psychiatric disorders
    Catatonia
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Diarrhoea
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 4 (25.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Inguinal hernia
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Mesenteric panniculitis
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nausea
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Salivary gland cyst
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Urethral haemorrhage
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal failure acute
         subjects affected / exposed
    1 / 4 (25.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 4 (25.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Bacteraemia
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Peritonitis
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Septic shock
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    QTI571 200mg QTI571 400mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 4 (100.00%)
    13 / 13 (100.00%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    2
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    2
    Oedema peripheral
         subjects affected / exposed
    1 / 4 (25.00%)
    3 / 13 (23.08%)
         occurrences all number
    1
    3
    Psychiatric disorders
    Sleep disorder
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Insomnia
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    3
    Depression
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    1 / 4 (25.00%)
    1 / 13 (7.69%)
         occurrences all number
    1
    1
    Blood potassium decreased
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    2
    Blood sodium decreased
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Platelet count decreased
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    International normalised ratio increased
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Leukopenia
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Lymphopenia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Anaemia
         subjects affected / exposed
    2 / 4 (50.00%)
    3 / 13 (23.08%)
         occurrences all number
    2
    3
    Thrombocytopenia
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    2
    Dysphonia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Epistaxis
         subjects affected / exposed
    0 / 4 (0.00%)
    3 / 13 (23.08%)
         occurrences all number
    0
    4
    Haemoptysis
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Nasal congestion
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Respiratory distress
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Neuropathy peripheral
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Dizziness
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Syncope
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Sciatica
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Eye disorders
    Periorbital oedema
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Cataract
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    2
    Constipation
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Diarrhoea
         subjects affected / exposed
    0 / 4 (0.00%)
    6 / 13 (46.15%)
         occurrences all number
    0
    6
    Abdominal pain upper
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Abdominal discomfort
         subjects affected / exposed
    1 / 4 (25.00%)
    1 / 13 (7.69%)
         occurrences all number
    1
    1
    Gastritis
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Large intestine polyp
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Vomiting
         subjects affected / exposed
    0 / 4 (0.00%)
    4 / 13 (30.77%)
         occurrences all number
    0
    7
    Nausea
         subjects affected / exposed
    0 / 4 (0.00%)
    3 / 13 (23.08%)
         occurrences all number
    0
    4
    Toothache
         subjects affected / exposed
    1 / 4 (25.00%)
    1 / 13 (7.69%)
         occurrences all number
    1
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Proteinuria
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Renal failure
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Renal impairment
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Night sweats
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Rash
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Arthralgia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Scleroderma
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Pain in extremity
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    2
    Muscle spasms
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    3
    Myalgia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Gout
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    2
    Hypercholesterolaemia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Hypokalaemia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Iron deficiency
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Hypoglycaemia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Infections and infestations
    Bacteriuria
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Ear infection
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Bronchitis
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Respiratory tract infection
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Otitis externa
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 4 (0.00%)
    6 / 13 (46.15%)
         occurrences all number
    0
    7
    Haemophilus infection
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    Gastroenteritis
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    2
    Staphylococcal skin infection
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 4 (0.00%)
    3 / 13 (23.08%)
         occurrences all number
    0
    3
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 4 (25.00%)
    1 / 13 (7.69%)
         occurrences all number
    1
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    22 Nov 2010
    The protocol was amended to specify an end date for the study and to clarify an exclusion criterion for female contraception requirements.
    17 Dec 2010
    The protocol was amended to remove the requirements for reporting pregnancy outcome from the male participant’s partner. Mandatory collection of the information could be an unwarranted intrusion into the privacy of the male participant’s partner.
    23 Jun 2012
    Relevant Data Summary was added to include the currently available clinical data, safety and tolerability data related to imatinib. Corrections of visit numbers in Section 6-Visit schedule and assessments were done. Fluid retention information for imatinib and the requirements for weight measurement and edema assessment were added. References were updated.
    14 Dec 2012
    This study has been designed to run over the course of three years. However, the current protocol mistakenly does not provide for study site visits during the final year. These visits have now been added to the assessment schedule. Patients will return to their study site twice during this period. This is an open label and requirement for an independent statistician and programmer was removed.

    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.
    In 2013, Novartis discontinued the development program of imatinib in pulmonary arterial hypertension (PAH) due to requirement of regulatory authorities for additional data to secure marketing approval; all global extension studies were closed.
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