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    Clinical Trial Results:
    A Phase II Study of RAD001 in the Treatment of Patients with Plexiform Neurofibromas (PN) associated with Neurofibromatosis Type 1 (NF1) Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com/CtrdWeb/home.novfor complete trial results.

    Summary
    EudraCT number
    		 2016-001563-36
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    26 Apr 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    06 Jul 2018
    First version publication date
    06 Jul 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CRAD001MIL04T
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01365468
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    26 Apr 2015
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Apr 2015
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    -To determine whether the mTOR inhibitor everolimus, administrated orally daily on a continuous dosing schedule:  a) Increases time to disease progression (TTP) based on volumetric MRI measurements in children and adults with NF1 and inoperable documented progressive PN (Stratum 1).  b) Results in objective radiographic responses based on volumetric MRI measurements in children and adults with NF1 and inoperable PN in the absence of documented radiographic progression at the study entry (Stratum 2). -To evaluate the tolerability and toxicity of chronic everolimus administration in this patient population as assessed by the NCI Common Toxicity Criteria, version 4.0.
    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.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    24 Apr 2012
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Israel: 9
    Worldwide total number of subjects
    9
    EEA total number of subjects
    0
    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)
    3
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    3
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Total of 9 patients were enrolled to either Stratum 1 (N=4) or Stratum 2 (N=5)

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Stratum 1
    Arm description
    		 Adults and children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN) with the potential to cause significant morbidity with documented progressive PN prior to study entry wereenrolled in this stratum. Enrolled patients received everolimus (RAD001) in an open label manner. Recommended starting dose of everolimus depend on body surface area, starting from 2.5 mg once daily to 7.5 mg once daily.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Everolimus
    Investigational medicinal product code
    RAD001
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    oral daily dosing of tablet starting with 2.5 mg

    Arm title
    		 Stratum 2
    Arm description
    		 Adults and children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN) with the potential to cause significant morbidity that do not have documented progression of the PN at the time of study entry were enrolled in this stratum. Enrolled patients received everolimus (RAD001) in an open label manner. Recommended starting dose of everolimus depend on body surface area, starting from 2.5 mg once daily to 7.5 mg once daily.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Everolimus
    Investigational medicinal product code
    RAD001
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    oral daily dosing of tablet starting with 2.5 mg

    Number of subjects in period 1
    		Stratum 1 Stratum 2
    Started
    4
    5
    Completed
    0
    5
    Not completed
    4
    0
         Physician decision
    1
    -
         Disease progression
    1
    -
         Lost to follow-up
    2
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Stratum 1
    Reporting group description
    		 Adults and children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN) with the potential to cause significant morbidity with documented progressive PN prior to study entry wereenrolled in this stratum. Enrolled patients received everolimus (RAD001) in an open label manner. Recommended starting dose of everolimus depend on body surface area, starting from 2.5 mg once daily to 7.5 mg once daily.

    Reporting group title
    Stratum 2
    Reporting group description
    		 Adults and children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN) with the potential to cause significant morbidity that do not have documented progression of the PN at the time of study entry were enrolled in this stratum. Enrolled patients received everolimus (RAD001) in an open label manner. Recommended starting dose of everolimus depend on body surface area, starting from 2.5 mg once daily to 7.5 mg once daily.

    Reporting group values
    		 Stratum 1 Stratum 2 Total
    Number of subjects
    		 4 5 9
    Age categorical
    Units: Subjects
        In utero
    		 0
        Preterm newborn infants (gestational age < 37 wks)
    		 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)
    		 0
        From 65-84 years
    		 0
        85 years and over
    		 0
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 22.7 ( 14.3 ) 16.9 ( 9.8 ) -
    Gender, Male/Female
    Units: Participants
        Female
    		 3 1 4
        Male
    		 1 4 5

    End points

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    End points reporting groups
    Reporting group title
    Stratum 1
    Reporting group description
    		 Adults and children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN) with the potential to cause significant morbidity with documented progressive PN prior to study entry wereenrolled in this stratum. Enrolled patients received everolimus (RAD001) in an open label manner. Recommended starting dose of everolimus depend on body surface area, starting from 2.5 mg once daily to 7.5 mg once daily.

    Reporting group title
    Stratum 2
    Reporting group description
    		 Adults and children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN) with the potential to cause significant morbidity that do not have documented progression of the PN at the time of study entry were enrolled in this stratum. Enrolled patients received everolimus (RAD001) in an open label manner. Recommended starting dose of everolimus depend on body surface area, starting from 2.5 mg once daily to 7.5 mg once daily.

    Primary: Time to disease progression (TTP) based on change in volumetric MRI measurements in children and adults (In Stratum I only)

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    End point title
    		 Time to disease progression (TTP) based on change in volumetric MRI measurements in children and adults (In Stratum I only) [1] [2]
    End point description
    This endpoint was planned to be analyzed for only Stratum 1 patients. Progression of disease defined as a ≥ 20% increase in the volume (by volumetric MRI) of at least one of the index plexiform neurofibromas (PN) compared to the pretreatment volume measured prior to the start of the current treatment phase. The Full Analysis Set (FAS) consisted of all enrolled patients. Median was not achieved because only one progression event occurred. The system does not accept "NA" for "Not available" or "Not achievable" data, not it allows user to leave the data field blank. To avoid system error, 9999.9 is used as placeholder.
    End point type
    Primary
    End point timeframe
    Screening, after course #6, #12, #18, #24, End of Treatment(1 course=28days)
    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: No statistical analysis was planned for this endpoint.
    [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: This primary endpoint was only planned to assess Stratum 1 patients.
    End point values
    		 Stratum 1
    Number of subjects analysed
    4
    Units: Days
        median (confidence interval 95%)
    9999.9 (-9999.9 to 9999.9)
    No statistical analyses for this end point

    Primary: Number of patients with objective radiographic responses based on volumetric MRI measurements (In Stratum 2 Only)

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    End point title
    		 Number of patients with objective radiographic responses based on volumetric MRI measurements (In Stratum 2 Only) [3] [4]
    End point description
    Response was assessed at the time that a follow up volumetric MRI scan is performed (after course 6 and then every 6 months and at the end of treatment). - Complete response (CR): complete resolution of all measurable or palpable PN for ≥ 28days and no appearance of new lesions. - Partial response (PR): A ≥ 20% reduction in the sum of the volume of all index PN lesions for ≥ 28days. - Stable disease (SD): A ‹ 20% increase and ‹ 20% decrease in the sum of the volume of all index PN lesions for ≥ 28days.
    End point type
    Primary
    End point timeframe
    Screening, after course #6, then every 6 months and end of treatment(1 course=28days)
    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: No statistical analysis was planned for this endpoint.
    [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: This primary endpoint was only planned to assess Stratum 2 patients.
    End point values
    		 Stratum 2
    Number of subjects analysed
    5
    Units: Patients
        Complete Response
    0
        Partial Response
    0
        Stable Disease
    5
    No statistical analyses for this end point

    Primary: Number of patients with adverse events assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04

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    End point title
    		 Number of patients with adverse events assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04 [5]
    End point description
    Adverse events were assessed according to the NCI Common Toxicity Criteria for Adverse Events (CTCAE) version 4.0. If CTCAE grading does not exist for an adverse event, the severity of mild, moderate, severe, and life-threatening, corresponding to grades 1 - 4 respectively, were used. CTCAE grade 5 (death) was not used in this study.
    End point type
    Primary
    End point timeframe
    From the time ICF was signed until 28 days after End of Treatment (up to a maximum of 25 months)
    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: No statistical analysis was planned for this endpoint.
    End point values
    		 Stratum 1 Stratum 2
    Number of subjects analysed
    4
    5
    Units: Patients
        At least one Grade 1 AE
    4
    5
        At least one Grade 2 AE
    4
    5
        At least one Grade 3 AE
    1
    0
        At least one Grade 4 AE
    1
    0
    No statistical analyses for this end point

    Other pre-specified: Number of patients with clinical response

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    End point title
    		 Number of patients with clinical response
    End point description
    Clinical response is defined as improvement of function, performance status, or decrease in PN related pain persisting for at least 28 days on treatment.
    End point type
    Other pre-specified
    End point timeframe
    Screening, Day 1, after course #3, #6, #12, #18, #24, End of Treatment (1 course = 28 days)
    End point values
    		 Stratum 1 Stratum 2
    Number of subjects analysed
    0 [6]
    0 [7]
    Units: Patients
    Notes
    [6] - Trial was terminated with only a few patients enrolled. Hence no statistical analysis was done
    [7] - Trial was terminated with only a few patients enrolled. Hence no statistical analysis was done
    No statistical analyses for this end point

    Other pre-specified: Physician’s Global Assessment of Clinical Condition (PGA) of skin lesions

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    End point title
    		 Physician’s Global Assessment of Clinical Condition (PGA) of skin lesions
    End point description
    The Physician‟s Global Assessment of Clinical Condition (PGA) is a 7-point grading scale for the investigator's assessment of the overall extent of improvement or worsening of the patient‟s skin disease as compared to baseline. Responses must be confirmed by at least two assessments separated in time by at least 4 weeks. The grading ranges from 0 to 6; 0 is Completely clear where as 6 is for worse condition. A complete clinical response (CCR) requires a grading of 0 indicating the absence of disease (histological confirmation is not required). Grades 1, 2, and 3 constitute partial response, indicating improvement of at least 50 percent, but less than 100 percent improvement.
    End point type
    Other pre-specified
    End point timeframe
    Screening, after course #3, #6, #12, #18, #24, End of Treatment (1 course = 28 days)
    End point values
    		 Stratum 1 Stratum 2
    Number of subjects analysed
    0 [8]
    0 [9]
    Units: Unit on scale
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [8] - Trial was terminated with only a few patients enrolled. Hence no statistical analysis was done
    [9] - Trial was terminated with only a few patients enrolled. Hence no statistical analysis was done
    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). All Adverse events are reported in this record from First Patient First Treatment until Last Patient Last Visit.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Stratum 2
    Reporting group description
    		 Adults and children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN) with the potential to cause significant morbidity that do not have documented progression of the PN at the time of study entry were enrolled in this stratum. Enrolled patients received everolimus (RAD001) in an open label manner. Recommended starting dose of everolimus depend on body surface area, starting from 2.5 mg once daily to 7.5 mg once daily.

    Reporting group title
    Stratum 1
    Reporting group description
    		 Adults and children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN) with the potential to cause significant morbidity with documented progressive PN prior to study entry wereenrolled in this stratum. Enrolled patients received everolimus (RAD001) in an open label manner. Recommended starting dose of everolimus depend on body surface area, starting from 2.5 mg once daily to 7.5 mg once daily.

    Serious adverse events
    		 Stratum 2 Stratum 1
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Stratum 2 Stratum 1
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 5 (100.00%)
    4 / 4 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Fibroma
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Surgical and medical procedures
    Cytoreductive surgery
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Tooth extraction
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Chest pain
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Discomfort
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Fatigue
         subjects affected / exposed
    2 / 5 (40.00%)
    2 / 4 (50.00%)
         occurrences all number
    2
    2
    Influenza like illness
         subjects affected / exposed
    1 / 5 (20.00%)
    3 / 4 (75.00%)
         occurrences all number
    1
    8
    Mucosal inflammation
         subjects affected / exposed
    2 / 5 (40.00%)
    2 / 4 (50.00%)
         occurrences all number
    3
    3
    Oedema peripheral
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Pain
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    1
    Peripheral swelling
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    2
    Reproductive system and breast disorders
    Breast mass
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Metrorrhagia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Vaginal haemorrhage
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Dysphonia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Dyspnoea
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    2 / 5 (40.00%)
    1 / 4 (25.00%)
         occurrences all number
    2
    1
    Pharyngeal erythema
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Insomnia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Sleep disorder
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Tic
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Investigations
    Blood cholesterol increased
         subjects affected / exposed
    2 / 5 (40.00%)
    1 / 4 (25.00%)
         occurrences all number
    2
    2
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Blood triglycerides increased
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    1
    Drug level increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Eosinophil count increased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Low density lipoprotein increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    2
    Lymphocyte count decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    2
    Platelet count decreased
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    4
    1
    Injury, poisoning and procedural complications
    Tendonitis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    1
    Headache
         subjects affected / exposed
    4 / 5 (80.00%)
    3 / 4 (75.00%)
         occurrences all number
    4
    3
    Paraesthesia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    0
    2
    External ear inflammation
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Ocular hyperaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    2 / 5 (40.00%)
    1 / 4 (25.00%)
         occurrences all number
    2
    1
    Diarrhoea
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    0
    3
    Dysphagia
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    0
    2
    Flatulence
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Mouth ulceration
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    5
    0
    Nausea
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Stomatitis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Toothache
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    1
    Vomiting
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    1
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Dermatitis acneiform
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Rash
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    0
    2
    Musculoskeletal and connective tissue disorders
    Musculoskeletal chest pain
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Pain in extremity
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    0
    5
    Infections and infestations
    Eye infection
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Pneumonia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Skin infection
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    1
    Hypertriglyceridaemia
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Study got terminated because of poor patient's accrual. Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.n
    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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