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    Summary
    EudraCT Number:2011-000608-16
    Sponsor's Protocol Code Number:5466
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-03-30
    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 ConcernedUK - MHRA
    A.2EudraCT number2011-000608-16
    A.3Full title of the trial
    To evaluate the safety and effectiveness of human ex vivo expanded autologous limbal stem cells for the treatment of unilateral total limbal stem cell deficiency
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    To evaluate how safe and efficient the treatment of blind patients with total corneal scar caused by stem cell failure is, when using cells from the patient's healthy other eye grown in the laboratory.
    A.3.2Name or abbreviated title of the trial where available
    Treatment of LSCD using cultured limbal epithelium expanded ALSC. V3.0
    A.4.1Sponsor's protocol code number5466
    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 SponsorThe Newcastle upon Tyne Hospitals NHS Foundation Trust
    B.1.3.4CountryUnited Kingdom
    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 supportMedical Research Council
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    B.Sponsor: 2
    B.1.1Name of SponsorThe Newcastle upon Tyne Hospitals NHS Foundation Trust
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsor
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 nameex vivo expanded autologous limbal stem cells
    D.3.2Product code N/A
    D.3.4Pharmaceutical form Living tissue equivalent
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPLocal use (Noncurrent)
    Unknown use (Noncurrent)
    Ophthalmic use (Noncurrent)
    Ocular use
    Route of administration not applicable
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAutologous stem cells
    D.3.9.4EV Substance CodeAS1
    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) Yes
    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 No
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    unilateral total limbal stem cell deficiency
    E.1.1.1Medical condition in easily understood language
    front of the eye cell failure (in one eye)
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and efficacy (or capacity to produce a desired effect) of ex vivo expanded autologous Limbal Stem Cells transplantation (transplant derived from the same patient, animal free and grown ex vivo or outside the body) for the treatment of unilateral total Limbal Stem Cell Deficiency (a severe corneal disease resulting in painful blindness). The product is manufactured according to GMP guidelines and under a Quality Management System.
    E.2.2Secondary objectives of the trial
    1. To evaluate the impact on quality of life of unilateral total Limbal Stem Cell Deficiency and changes after transplantation; 2. To evaluate the management of Limbal Stem Cell Deficiency in relation to clinical, economic and humanistic outcomes; 3. To evaluate the reversal of LSCD by impression cytology; 4. To evaluate an Optical Coherence Tomography (OCT) pattern as a means of predicting visual outcome after cultured LSC transplantation and the potential need for secondary intervention (e.g. corneal transplantation) after transplantation; 5. To evaluate the pre-corneal tear film in patients with unilateral LSCD before and after limbal stem cell transplantation; 6. To evaluate improvement in LogMAR visual acuity; 7. To evaluate reversal of central corneal vascularisation; 8. To evaluate reduction in corneal opacity; 9. To evaluate corneal/limbal epithelial changes by confocal microscopy; 10.To evaluate the molecular changes in the corneal tear film, particularly cytokines a
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    SUB STUDY 1 Assessment of precorneal tear film in patients with unileteral total Limbal Stem Cell Deficiency (as per Protocol version 3.0, 15th July 2011) Objectives: 1. To determine key factors affecting tear film stability in patients with LSCD and during the recovery process after ex vivo autologous LSC transplantation 2. To analyse pre and postoperative patterns of change in the characteristics of mucins (proteins) dissolved in tears 3. To quantify inflammatory mediators in precorneal tear film in patients with unilateral LSCD compared with other healthy eye and after ex vivo autologous LSC transplantation 4. To assess inflammatory phenotype in ocular surface glycans in patients with unilateral LSCD compared with other healthy eye and after ex vivo autologous LSC transplantation. SUBSTUDY 2 Cryopreservation of cultured human limbal epithelium (as per Protocol version 3.0, 15th July 2011) Objectives: 1. Optimization of a protocol for cryopreservation of human limbal epithelial cell cultures based on cell survival 2. Characterization of cryopreserved human limbal epithelial cultures 3. Establishment of good manufacturing practice and transportation protocols for cryopreservation of human limbal epithelial cultures
    E.3Principal inclusion criteria
    • Patient has provided written informed consent for participation in the study prior to any study specific procedures; • Patients must be 18 years of age or older and consent to have their data included in the database for research purposes; • Patients must be prepared and able to complete questionnaires; • Diagnosis of unilateral total LSCD (confirmed by impression cytology), with normal B scan ultrasound & electrophysiology; • No other ocular abnormality in recipient eye(s); • Women of child bearing potential must be using adequate contraception for duration of study and have a negative baseline pregnancy test as part of screening (post-consent).
    E.4Principal exclusion criteria
    • Significant co-morbidity in which compliance with the study procedures would not be expected e.g. suspected insufficient cognitive ability to perform the tests (assessed using the 11-item Telephone Interview for Cognitive Status instrument); • Dry eye and eyelid abnormality in the affected eye; • Previous surgery to ocular surface of healthy contralateral donor eye; • Abnormal corneal impression cytology in healthy contralateral donor eye. • Pregnancy, or women planning to become pregnant within next 12 months, or women who are breast feeding; • Participating in other investigational study within 30 days prior to study entry (defined as date of enrolment/baseline visit into study); • Previous participation in this study.
    E.5 End points
    E.5.1Primary end point(s)
    1. Reversal of LSCD by impression cytology; 2. Ocular surface reconstruction (i.e., corneal re-epithelisation); 3. Improvement in patient’s reported ocular surface pain.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Reversal of LSCD by impression cytology - 6 month & 12 month post-op 2. Ocular surface reconstruction (i.e., corneal re-epithelisation) - all visits post-op up to 12 months 3. Improvement in patient’s reported ocular surface pain - 12 weeks, 6 month & 12 month post-op Some of the above procedures/investigations will also continue after 12 months post-op, as per routine clinical practice.
    E.5.2Secondary end point(s)
    1. Improvement in LogMAR Visual acuity; 2. Reversal in central corneal vascularisation; 3. Reduction in corneal opacity; 4. Improvement in patient’s reported outcomes; 5. Presence of complications; 6. Corneal opacity assessment by anterior segment OCT; 7. Corneal/limbal epithelial assessment by confocal microscopy.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Improvement in LogMAR Visual acuity - all visits post-op up to 12 months 2. Reversal in central corneal vascularisation - all visits post-op up to 12 months 3. Reduction in corneal opacity - all visits post-op up to 12 months 4. Improvement in patient’s reported outcomes - 12 weeks, 6 month & 12 month post-op 5. Presence of complications - all visits post-op up to 12 months 6. Corneal opacity assessment by anterior segment OCT - 2 weeks, 4 weeks, 8 weeks, 12 weeks, 6 months & 12 months post-op 7. Corneal/limbal epithelial assessment by confocal microscopy - 6 months & 12 months post-op Some of the above procedures/investigations will also continue after 12 months post-op, as per routine clinical practice.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    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 Yes
    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 No
    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.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    E.8.5.1Number of sites anticipated in the EEA1
    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 Yes
    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
    The end of study will be the last participant's final study contact at 12 months follow-up
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state24
    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)
    The ex vivo autologous limbal stem cell intervention is permanent following transplantation. The end of the study will be the date of last subjects final assessment (12 months follow up). At the end of the study, participants will be offered NOT to be discharged, allowing 18, 24 and 36 months clinical data collection and continued treatment for their unilateral total limbal stem cell deficiency, as per normal routine clinical practice. ….
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Northumberland and Tyne & Wear CLRN
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-12-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-08-16
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-02-09
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