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    Clinical Trial Results:
    Pilot Clinical Assessment of Ex Vivo Expanded Corneal Limbal Stem Cell Transplantation in Patients with Severe Ocular Surface Disease (OSD) Arising from Limbal Stem Cell Deficiency

    Summary
    EudraCT number
    2010-024409-11
    Trial protocol
    GB  
    Global end of trial date
    14 Mar 2016

    Results information
    Results version number
    v1(current)
    This version publication date
    30 Mar 2017
    First version publication date
    30 Mar 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    LSC-001
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    SNBTS
    Sponsor organisation address
    21 Ellens Glen road,, Edinburgh, United Kingdom, EH17 7QT
    Public contact
    Regulatory Compliance Officer, Scottish National Blood Transfusion Service, 0044 131 314 5591, emily.hargreaves@nhs.net
    Scientific contact
    Associate Director Research Development and Innovation, Scottish National Blood Transfusion Service, 0044 1313145677, johncampbell3@nhs.net
    Sponsor organisation name
    NHS Lothian
    Sponsor organisation address
    47 Little France Cres, , Edinburgh, United Kingdom, EH16 4TJ
    Public contact
    Douglas Young, NHS Lothian, 0044 1312423337, douglas.young@luht.scot.nhs.uk
    Scientific contact
    Douglas Young, NHS Lothian, 0044 1312423337, douglas.young@luht.scot.nhs.uk
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    02 Mar 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    14 Mar 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Mar 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    Pilot study examining the efficacy and safety of transplanting ex vivo expanded corneal epithelial limbal stem cells on Amniotic Membrane (AM) as a procedure to restore sight and relieve pain for patients with severe Ocular Surface Disease (OSD) arising from Limbal Stem Cell Deficiency (LSCD). Aim was to: •Generate the data required for reliable sample size calculations for subsequent studies •Evaluate all the practicalities and logistics of the study including the recruitment process, follow-up procedures, data collection and analysis •Obtain information on actual recruitment rate •The study also aimed to obtain preliminary answers to the following questions:- •Is transplantation of ex vivo expanded corneal limbal stem cells feasible, efficient and safe? •Does this procedure lead to improvements in vision and quality of the ocular surface? •How does immunosuppression and limbal stem cell transplantation compare with using immunosuppression and amniotic membrane alone?
    Protection of trial subjects
    Bandage contact lens was placed atop the graft for comfort and protection, also Botox injections were applied to the affected eye lid this induced temporary ptosis which aimed to facilitate the survival of the graft and to protect the epithelium, also a short course of topical antibiotic and steroid eye drops were prescribed to be applied to the affected eye, post surgery.
    Background therapy
    Immunosuppressive therapy was tailored according to the patient’s individual clinical response. • Prednisolone: Initial dose of 60 mg daily tapering at 5mg weekly until 10 mg maintenance dosage plus • Cyclosporine: Initial dose of 100 mg twice a day then tapered to 50mg twice a day or • Mycophenolate mofetil. Dose between 750mg to 1g twice a day Further, prior to surgery, each patient made a single autologous donation of blood. This allowed preparation of autologous serum eye drops for the patient’s own use post-surgery.
    Evidence for comparator
    The control product amniotic membrane (AM) is a used extensively to treat ocular surface disorders. It is hypothesised that immunosuppressive therapy and AM alone may allow ocular surface reconstruction by eliminating the inflammatory environment that is detrimental to the function of stem cells. Therefore, one group of patients received donor derived ex vivo expanded corneal limbal stem cells on AM and immunosuppressive therapy, and the second group received AM graft and immunosuppressive therapy.
    Actual start date of recruitment
    01 Jul 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
    United Kingdom: 17
    Worldwide total number of subjects
    17
    EEA total number of subjects
    17
    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)
    15
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Recruitment started on 1st July 2011 and ceased early due to slow recruitment at 17 patients, amendment to cease recruitment approved by the MHRA 09/12/2014. A total of 30 patients were screened for the trial of whom 13 were either not suitable (6) or declined (7). 17 were enrolled of whom 16 were treated (one patient withdrew prior to treatment)

    Pre-assignment
    Screening details
    Subjects diagnosed with LSCD were screened for inclusion in trial in compliance with inclusion/exculsion criteria defined in study protocol LSC001. Informed consent for participation in trial gained prior to any study related procedures taking place.

    Period 1
    Period 1 title
    Overall Period
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Single blind
    Roles blinded
    Subject
    Blinding implementation details
    The randomisation schedule was generated using Random Permuted Blocks (Pocock S. 1993). Neither the patient, nor the study staff performing the post-operative evaluations, knew to which treatment group they had been allocated. The randomisation was only broken by the Pharmacovigilance Manager at the end of the study, or would have been in the event of an unexpected Serious Adverse Reaction (SAR)

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    IMP Test Arm
    Arm description
    Subjects had either bilateral or unilateral LSCD resulting from one of the following underlying condition: - Aniridia -Chemical injury -Autoimmune disorder All subjects treated met inclusion/exclusion criteria defined in the study protocol LSC001 .
    Arm type
    Experimental

    Investigational medicinal product name
    Allogeneic Ex-vivo Expanded Corneal Epithelial Stem Cells on Amniotic Membrane
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Implant
    Routes of administration
    Ocular use, Ophthalmic use
    Dosage and administration details
    Each product was considered to be a single ‘dose’ based with ≥200mm2 macroscopic outgrowth and a diameter ≥15mm in all directions. Administration was via the removal of abnormal tissue over the cornea and the conjunctiva resected and recessed. The graft was placed atop the defect and the edge sewn to the peripheral cornea with 10-0 nylon. The posterior peripheral edge of the AM was sewn to the resected and recessed conjunctiva. A second AM was sewn on top of the product and a bandage contact lens placed to help protect the cells. One of the explants was sewn in the 12 o’clock position. The AMs dissolved gradually and the stem cells establish to the grafted surface

    Arm title
    Control Arm
    Arm description
    Subjects had bilateral LSCD resulting from one of the following underlying condition: - Aniridia -Chemical injury -Autoimmune disorder All subjects treated met inclusion/exclusion criteria defined in the study protocol LSC001 .
    Arm type
    Active comparator

    Investigational medicinal product name
    Amniotic Membrane
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Implant
    Routes of administration
    Ocular use
    Dosage and administration details
    Control AM for transplantation alone was prepared using an identical technique as for the IMP Allogeneic Ex-vivo Expanded Corneal Epithelial Stem Cells on AM, the AM was administered in the same way as the IMP arm. Each cultured AM was considered to be a single ‘dose’. Administration was via the removal of abnormal tissue over the cornea and the conjunctiva resected and recessed. The graft was placed atop the defect and the edge sewn to the peripheral cornea with 10-0 nylon. The posterior peripheral edge of the AM was sewn to the resected and recessed conjunctiva. A second amniotic membrane was sewn on top of the product and a bandage contact lens placed to help protect the graft.

    Number of subjects in period 1
    IMP Test Arm Control Arm
    Started
    9
    8
    Completed
    8
    5
    Not completed
    1
    3
         Physician decision
    -
    1
         Adverse event, serious fatal
    -
    1
         Adverse event, non-fatal
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    IMP Test Arm
    Reporting group description
    Subjects had either bilateral or unilateral LSCD resulting from one of the following underlying condition: - Aniridia -Chemical injury -Autoimmune disorder All subjects treated met inclusion/exclusion criteria defined in the study protocol LSC001 .

    Reporting group title
    Control Arm
    Reporting group description
    Subjects had bilateral LSCD resulting from one of the following underlying condition: - Aniridia -Chemical injury -Autoimmune disorder All subjects treated met inclusion/exclusion criteria defined in the study protocol LSC001 .

    Reporting group values
    IMP Test Arm Control Arm Total
    Number of subjects
    9 8 17
    Age categorical
    Inclusion Criteria Adult patients, of either sex, with corneal blindness due to limbal stem cell deficiency
    Units: Subjects
        Adults (18-64 years)
    9 6 15
        From 65-84 years
    0 2 2
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    46 ± 12 53 ± 12 -
    Gender categorical
    Units: Subjects
        Female
    5 3 8
        Male
    4 5 9
    Subject analysis sets

    Subject analysis set title
    aniridia
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Aniridia is a congenital, bilateral, panocular disorder which can develop to affect the iris, cornea, anterior chamber angle, lens, retina and optic nerve with frequent association with multiple ocular abnormalities including limbal stem cell deficiency

    Subject analysis set title
    chemical injury
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Burns involving the eye can lead to destruction of part or all of the limbus on a unilateral or bilateral basis causing LSCD.

    Subject analysis set title
    Autoimmune disorder
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    autoimmune including pemphigoid, may lead to LSCD.

    Subject analysis sets values
    aniridia chemical injury Autoimmune disorder
    Number of subjects
    7
    8
    2
    Age categorical
    Inclusion Criteria Adult patients, of either sex, with corneal blindness due to limbal stem cell deficiency
    Units: Subjects
        Adults (18-64 years)
    6
    7
    2
        From 65-84 years
    1
    1
    0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    53 ± 9
    48 ± 16
    43 ± 20
    Gender categorical
    Units: Subjects
        Female
    3
    4
    1
        Male
    3
    4
    1

    End points

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    End points reporting groups
    Reporting group title
    IMP Test Arm
    Reporting group description
    Subjects had either bilateral or unilateral LSCD resulting from one of the following underlying condition: - Aniridia -Chemical injury -Autoimmune disorder All subjects treated met inclusion/exclusion criteria defined in the study protocol LSC001 .

    Reporting group title
    Control Arm
    Reporting group description
    Subjects had bilateral LSCD resulting from one of the following underlying condition: - Aniridia -Chemical injury -Autoimmune disorder All subjects treated met inclusion/exclusion criteria defined in the study protocol LSC001 .

    Subject analysis set title
    aniridia
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Aniridia is a congenital, bilateral, panocular disorder which can develop to affect the iris, cornea, anterior chamber angle, lens, retina and optic nerve with frequent association with multiple ocular abnormalities including limbal stem cell deficiency

    Subject analysis set title
    chemical injury
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Burns involving the eye can lead to destruction of part or all of the limbus on a unilateral or bilateral basis causing LSCD.

    Subject analysis set title
    Autoimmune disorder
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    autoimmune including pemphigoid, may lead to LSCD.

    Primary: visual acuity

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    End point title
    visual acuity
    End point description
    Best corrected visual acuity
    End point type
    Primary
    End point timeframe
    The end point of the trial was 18 months post treatment, subjects were reviewed at pre treatment, then post treatment at day 1, 2/3, 7, 14 and month 1, 3, 6, 9,12, 15 and 18 months
    End point values
    IMP Test Arm Control Arm
    Number of subjects analysed
    8
    5
    Units: average reduction in logmar score
        arithmetic mean (standard deviation)
    -0.82 ± 0.97
    -0.89 ± 0.51
    Statistical analysis title
    visual acuity
    Statistical analysis description
    analysis of reduction in Logmar score by unpaired t-test
    Comparison groups
    IMP Test Arm v Control Arm
    Number of subjects included in analysis
    13
    Analysis specification
    Post-hoc
    Analysis type
    other
    P-value
    = 0.228 [1]
    Method
    t-test, 1-sided
    Confidence interval
    Notes
    [1] - no significant difference shown between IMP and control arm for visual acuity

    Secondary: Ocular Surface Score

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    End point title
    Ocular Surface Score
    End point description
    A composite score for the ocular surface disease status of the patients was obtained throughout the study according to the following 5 criteria: corneal epithelium conjunctivalisation corneal neovascularisation corneal opacification conjunctival hyperaemia Each criteria was scored from 0-3 (normal-severe damage) by the trial physicians, and an aggregate score out of 15 obtained.
    End point type
    Secondary
    End point timeframe
    18 months , measurements at pre treatment, post treatment at day 1, 2/3, 7, 14 and month 1, 3, 6, 9,12, 15 and 18 months
    End point values
    IMP Test Arm Control Arm
    Number of subjects analysed
    8
    5
    Units: ocular surface score
        number (not applicable)
    2.07
    1.42
    Attachments
    results summary charts
    Statistical analysis title
    ocular surface score
    Statistical analysis description
    8 patients in the IMP test arm and 5 patients in the control arm had ocular surface scores which were evaluated throughout, and at conclusion of the trial. All patients showed lower (improved) ocular surface scores at the conclusion of the trial. However, only patients in the test arm who received the IMP Allogeneic Ex-vivo Expanded Corneal Epithelial Stem Cells on Amniotic Membrane product showed a statistically significant higher mean improvement in combined ocular surface score.
    Comparison groups
    IMP Test Arm v Control Arm
    Number of subjects included in analysis
    13
    Analysis specification
    Post-hoc
    Analysis type
    equivalence
    P-value
    = 0.004 [2]
    Method
    t-test, 1-sided
    Confidence interval
    Notes
    [2] - Statistically significant improvement in combined ocular surface scores when treated with IMP (p=0.0040, unpaired t-test) compared with Control arm, based on the mean change from pre treatment to 18 months post treatment ocular surface score +/- SD.

    Secondary: Quality of Life

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    End point title
    Quality of Life
    End point description
    Quality of life (QoL) assessments using the SF-36 scoring system were analysed as a whole data set, and as delta in each patient relative to each individual’s starting score in each category. There were no statistically significant changes in QoL scoring throughout the trial. The one exception was a significant decrease at 6 months (p= 0.0024) in the Social Function component of the SF36 score which returned to non-significance over baseline thereafter. This likely contributed to a trend towards initial reduction in overall SF-36 score at 6 months which recovered subsequently. Although there was fluctuation over time, SF-36 scores closely tracked to the pre-trial baseline levels by the end of the study
    End point type
    Secondary
    End point timeframe
    18 months
    End point values
    IMP Test Arm Control Arm
    Number of subjects analysed
    8 [3]
    5 [4]
    Units: number
        number (not applicable)
    0
    0
    Attachments
    Quality of Life analysis
    Notes
    [3] - Outcome of anaysis is given in the appended document
    [4] - Outcome of anaysis is given in the appended document
    No statistical analyses for this end point

    Post-hoc: WBC and Cytokine levels

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    End point title
    WBC and Cytokine levels
    End point description
    12/13 patients showed normal WBC levels throughout the study. 1 patient with chemical injury had an elevated WBC – this patient had ongoing ocular inflammation and had to be withdrawn from the study for corneal transplant. Patients could be stratified into normal or high serum cytokines at the start of the study – the normal group consisted of chemical burns patients and the one patient with autoimmune disease, while the high group consisted of all aniridia patients, 2 chemical burns, and the other patient with an autoimmune disease (pemphigus). In the high cytokine group, IL-8 was highly elevated in all patients, but IFN-gamma and TNF-alpha levels were not increased. The high group also showed variably increased levels of IL-2, 4, 10 and 12, but there was no consistent pattern. There was no correlation between IL-8 levels and WBC. the results of this post hoc analysis are appended.
    End point type
    Post-hoc
    End point timeframe
    up to 18 months
    End point values
    IMP Test Arm Control Arm
    Number of subjects analysed
    8 [5]
    5 [6]
    Units: number
        number (not applicable)
    0
    0
    Attachments
    wbc cytokine analysis
    Notes
    [5] - Outcome of anaysis is given in the appended document
    [6] - Outcome of anaysis is given in the appended document
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    18 months post treatment
    Adverse event reporting additional description
    Patients were monitored for any adverse events during the study period. Any serious adverse event which arose following use of the limbal stem cell graft or the AM was immediately notified to the SNBTS .
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    18
    Reporting groups
    Reporting group title
    IMP Corneal epithelial stem cells on amniotic membrane
    Reporting group description
    AEs from patients receiving the IMP

    Reporting group title
    control arm amniotic membrane
    Reporting group description
    Control group receiving amniotic membrane

    Serious adverse events
    IMP Corneal epithelial stem cells on amniotic membrane control arm amniotic membrane
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 9 (33.33%)
    2 / 7 (28.57%)
         number of deaths (all causes)
    0
    1
         number of deaths resulting from adverse events
    0
    0
    Surgical and medical procedures
    Death
    Additional description: The patient’s death due to a perforated bowel and sepsis was not considered to be related to the product.
    alternative dictionary used: MedDRA 15
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Laser therapy
    alternative dictionary used: MedDRA 15.1
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    colostomy
    alternative dictionary used: MedDRA 15
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Eye disorders
    Corneal epithelium defect
    Additional description: patient withdrawn
    alternative dictionary used: MedDRA 16.1
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Ulcerative keratitis
    alternative dictionary used: MedDRA 16.1
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Haematuria
    alternative dictionary used: MedDRA 16.1
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Spinal compression fracture
    alternative dictionary used: MedDRA 15
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         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: 0%
    Non-serious adverse events
    IMP Corneal epithelial stem cells on amniotic membrane control arm amniotic membrane
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 9 (88.89%)
    7 / 7 (100.00%)
    Investigations
    Intraocular pressure increased
    alternative dictionary used: MedDRA 15
         subjects affected / exposed
    1 / 9 (11.11%)
    2 / 7 (28.57%)
         occurrences all number
    1
    2
    Blood pressure ambulatory increased
    alternative dictionary used: MedDRA 16.0
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Cardiac disorders
    Presyncope
    alternative dictionary used: MedDRA 15
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
    alternative dictionary used: MedDRA 15.1
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Eye swelling
    alternative dictionary used: MedDRA 15
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Corneal abrasion
    alternative dictionary used: MedDRA 16.1
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Eye inflammation
    alternative dictionary used: MedDRA 16.1
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Conjunctival scar
    alternative dictionary used: MedDRA 16.1
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Corneal deposits
    alternative dictionary used: MedDRA 17.0
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Ocular hyperaemia
    alternative dictionary used: MedDRA 17.1
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Vomiting
    alternative dictionary used: MedDRA 15
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 7 (14.29%)
         occurrences all number
    1
    1
    Reproductive system and breast disorders
    Testicular mass
    alternative dictionary used: MedDRA 16.1
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Erectile dysfunction
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Renal and urinary disorders
    Urinary tract infection
    alternative dictionary used: MedDRA 16.0
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Urinary retention postoperative
    alternative dictionary used: MedDRA 16.0
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Lithotripsy
    alternative dictionary used: MedDRA 17.0
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Stasis dermatitis
    alternative dictionary used: MedDRA 16.1
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    Pruritus
    alternative dictionary used: MedDRA 17.0
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Endocrine disorders
    Pituitary cyst
    alternative dictionary used: MedDRA 16.1
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    11 Sep 2012
    Following a Serious Adverse Event, that was unrelated to the study product but resulted in the death of the patient, Information for Patients was updated to include additional information on the possible risks of concomitant trial medication, including immunosuppressive therapy. in addition, new investigator site at St Pauls Eye unit Liverpool added as an additional investigator site.

    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 was terminted early due to slow recruitment, therefore only 17 patients were recruited instead of the planned 20 (10 in each arm), 13 patients completed the study.
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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