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    Summary
    EudraCT Number:2013-004247-24
    Sponsor's Protocol Code Number:CCRG13-003
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2013-12-04
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2013-004247-24
    A.3Full title of the trial
    Translational stem cell research in ophthalmology - regenerating the anterior cornea through standardized transplantation of limbal epithelial stem cells: a phase II multicenter trial
    Translationeel stamcelonderzoek in de oogheelkunde - regeneratie van het anterieure hoornvlies door een gestandaardiseerde transplantatie van limbale epitheliale stamcellen: een fase II multicentrische studie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Regenerating the cornea through transplantation of limbal stem cells
    Regeneratie van het hoornvlies door een transplantatie van limbale stamcellen
    A.4.1Sponsor's protocol code numberCCRG13-003
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAntwerp University Hospital (UZA)
    B.1.3.4CountryBelgium
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAgentschap voor Innovatie door Wetenschap en Technologie
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAntwerp University Hospital (UZA)
    B.5.2Functional name of contact pointNadia Zakaria
    B.5.3 Address:
    B.5.3.1Street AddressWilrijkstraat 10
    B.5.3.2Town/ cityEdegem
    B.5.3.3Post code2650
    B.5.3.4CountryBelgium
    B.5.4Telephone number003238214066
    B.5.6E-mailnadia.zakaria@uza.be
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameLimbal Epithelial Cell (LEC) graft
    D.3.2Product code LEC
    D.3.4Pharmaceutical form Ophthalmic insert
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOphthalmic use (Noncurrent)
    Ocular use
    Auricular use
    Implantation
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNLEC
    D.3.9.2Current sponsor codeLEC
    D.3.9.3Other descriptive nameLimbal Epithelial Cells
    D.3.9.4EV Substance CodeSUB119898
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number50000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product Yes
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary and secondary Limbal Stem Cell Deficiencies
    E.1.1.1Medical condition in easily understood language
    Limbal Stem Cell Deficiencies
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level PT
    E.1.2Classification code 10069798
    E.1.2Term Amniotic membrane graft
    E.1.2System Organ Class 10042613 - Surgical and medical procedures
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level PT
    E.1.2Classification code 10072138
    E.1.2Term Limbal stem cell deficiency
    E.1.2System Organ Class 10015919 - Eye disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to evaluate the success rate of transplanting non-xenogenic, bioengineered, composite grafts of cultured limbal epithelial stem cells on standardized amniotic membranes – in patients with LSCD. Based on the past experience from our phase I/II monocenter clinical trial, our primary hypothesis is that it will be well-tolerated, and not associated with serious adverse side effects. Assuming a success percentage of 67% based on our previous study, a sample size of 60 patients produces a two-sided 95% confidence interval with a width equal to 0.238. The larger sample size and inclusion of more allogenic transplant recipients, together with a longer follow-up, will help ascertain safety and efficacy of the proposed treatment.
    E.2.2Secondary objectives of the trial
    The secondary objective of this study is to develop and implement a sub-clinical Ocular Surface Inflammation Monitoring (OSIM) tool based on biomarkers in tear samples. Further we would like to improve intra-operative visibility for the surgeon by introducing FD-OCT assisted pannus dissection prior to limbal stem cell transplantation. We further wish to introduce improved non-contact methods for transplanted corneal epithelial thickness monitoring and graft tracking via FD-OCT assisted follow-up.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Voluntary written informed consent
    2. Patients suffering from LSCD IIa and IIb. Those suffering from IIc may be included once inflammation has subsided and cornea can be staged as IIb.
    3. Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before registration in the trial.
    4. Women of child-bearing potential should use adequate contraception prior to study entry and for the duration of study participation. Negative serum or urine β-HCG pregnancy test at screening.
    E.4Principal exclusion criteria
    1. Subjects who are pregnant or lactating
    2. Subjects who have sensitivity to drugs that provide local anesthesia
    3. Subjects suffering from active infection of the external eye
    4. Medical conditions that prohibit the use of systemic immunosuppression (in cases of allogenic transplantation)
    E.5 End points
    E.5.1Primary end point(s)
    We will assess the success rate of transplanted limbal stem cells by a clinical observation of:

    Short term:
    1. Increased visual acuity
    2. Absence of conjunctivalization
    3. Resolution of corneal vascularization
    4. Absence of persistent epithelial defect
    5. Decreased pain
    6. Decreased photophobia

    Long term:
    1. Prolonged subsequent corneal graft survival time
    E.5.1.1Timepoint(s) of evaluation of this end point
    Monitoring will be performed according to a fixed time schedule:

    * First month: weekly
    * Months 1, 2, 3, 6
    * Every six months thereafter until minimun of 2 years follow-up
    E.5.2Secondary end point(s)
    1. Development and implementation of a sub-clinical Ocular Surface Inflammation Monitoring (OSIM) tool based on biomarkers in tear samples
    2. Implementation of FD-OCT assisted limbal stem cell transplantation and post operative follow-up
    3. Time until complete corneal epithelization is visualised using slit lamp
    4. Presence or absence of normal corneal epithelial thickness using FD-OCT
    5. Determine evolution of corneal pachymetry over time
    6. Determine evolution of corneal epithelial thickness over time
    7. In cases where a second operation needs to be preformed for improved visual rehabilitation, for example, penetrating keratoplasty, the removed corneal button will be sent for immunohistopathology in order to provide further insight into disease progression.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Monitoring will be performed according to a fixed time schedule:

    * First month: weekly
    * Months 1, 2, 3, 6
    * Every six months thereafter until minimun of 2 years follow-up
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    last visit of last subject
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 30
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state60
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 60
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Normal treatment and follow-up
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-01-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-12-16
    P. End of Trial
    P.End of Trial StatusOngoing
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