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    Summary
    EudraCT Number:2014-002845-23
    Sponsor's Protocol Code Number:CCD-GPLSCD01-03
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-09-30
    Trial results View 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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2014-002845-23
    A.3Full title of the trial
    Multinational, multicentre, prospective, open-label, uncontrolled clinical trial to assess the efficacy and safety of Autologous Cultivated Limbal Stem Cells Transplantation (ACLSCT) for restoration of corneal epithelium in patients with limbal stem cell deficiency due to ocular burns
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to follow the implantation of Holoclar on the cornea in patients with occular burns
    A.3.2Name or abbreviated title of the trial where available
    HOLOCORE
    A.4.1Sponsor's protocol code numberCCD-GPLSCD01-03
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/199/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorChiesi Farmaceutici S.p.A.
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportChiesi Farmaceutici S.p.A.
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationChiesi Farmaceutici S.p.A.
    B.5.2Functional name of contact pointClinical Project Manager
    B.5.3 Address:
    B.5.3.1Street AddressVia Palermo 26A
    B.5.3.2Town/ cityPARMA
    B.5.3.3Post code43122
    B.5.3.4CountryItaly
    B.5.4Telephone number3905211689281
    B.5.5Fax number390521774468
    B.5.6E-mailclinicaltrials_info@chiesi.com
    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 Yes
    D.2.1.1.1Trade name HOLOCLAR
    D.2.1.1.2Name of the Marketing Authorisation holderChiesi farmaceutici SpA
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/08/579
    D.3 Description of the IMP
    D.3.1Product nameHoloclar
    D.3.4Pharmaceutical form Living tissue equivalent
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOphthalmic use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNA
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeCCD-GPLSCD01-03
    D.3.9.3Other descriptive nameEX VIVO EXPANDED AUTOLOGOUS HUMAN CORNEAL EPITHELIAL CELLS CONTAINING STEM CELLS
    D.3.9.4EV Substance CodeSUB172912
    D.3.10 Strength
    D.3.10.1Concentration unit cm2 square centimeter
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3.8
    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 No
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberTissue Engineered Product (EMA/54465/2011)
    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
    corneal lesions with associated moderate to severe limbal stem cell deficiency due to ocular burns
    E.1.1.1Medical condition in easily understood language
    corneal damage due to ocular burns
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10011012
    E.1.2Term Corneal epithelium opacity
    E.1.2System Organ Class 100000004853
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate the efficacy of Holoclar® at one year after the first
    treatment in patients suffering from moderate (vascularization in two-three
    corneal quadrants with central corneal involvement) to severe
    (vascularization in four corneal quadrants with central corneal
    involvement) LSCD with severe visual impairment and secondary to
    ocular burns, in terms of percentage of patients with a success of
    transplantation at approximately 12 months from the first Holoclar®
    treatment.
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of one or two treatments with Holoclar at one year after the last treatment.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent prior to any study-related procedures;
    2. Adult male and female patients (≥18 years old) - [Five Paediatric
    patients aged 2 to 17 years will be also enrolled for safety purposes
    only];
    3. LSCD secondary to unilateral or bilateral physical or chemical
    ocular burns, with at least 1-2 mm2 of undamaged limbus to harvest
    stem cells for expansion in culture. LSCD will be considered for inclusion
    in presence of superficial neo-vascularization invading at least two
    corneal quadrants with evidence of central corneal (central 6 mm
    diameter) involvement (including central corneal neo-vascularisation or
    corneal opacity) according to the independent assessors;
    4. Stability of LSCD, defined by a duration of disease of at least 24
    months (12 months for minors) at the time of the Screening Visit and as
    presence of continuum epithelium as per fluorescein staining scored as
    none or trace;
    5. Presence of severe impairment in visual acuity defined by a score
    after best correction (i.e. Best Corrected Visual Acuity) equal or below
    1/10 (or 20/200) at the Snellen chart (legal blindness);
    6.      Absence of other clinical contraindications to ACLSC transplantation based upon investigator’s judgment;
    7.      A cooperative attitude to follow up the study procedures (Caregivers in case of minors).
    E.4Principal exclusion criteria
    1. LSCD of mild degree (i.e. below 2 quadrants of vascularization
    invasion), due to a recent burn (less than 24 months before screening
    for adults and 12 months for minors), or secondary to medical conditions
    other than burns (i.e. radiotherapy);
    2. Severe ocular inflammation according to the Efron Grading Scale for Contact Lens Complications. Patient can be re-screened after appropriate treatment;
    3. Presence of eyelids malposition;
    4. Conjunctival scarring with fornix shortening;
    5. Tear secretion deficiency, determined by Schirmer’s test (<5 mm/ 5 min);
    6. Corneal anaesthesia and conjunctival anaesthesia or severe hypoesthesia;
    7. Clinically significant or unstable concurrent disease or other
    clinical contraindications to stem cell transplantation based upon
    investigator’s judgment or other concomitant medical conditions
    affecting grafting procedure (i.e. use of contrast medium in the
    last two weeks) or any other invasive procedure which could
    affect the integrity of the epithelium
    E.5 End points
    E.5.1Primary end point(s)
    - Percentage of patients with a success of transplantation (success is defined on the basis of the degree of “superficial corneal neo-vascularization” and “epithelial defects”)
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months from the first Holoclar treatment
    E.5.2Secondary end point(s)
    - Percentage of patients with clinical success after one or two ACLSCTs (same definition of success as primary endpoint)
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months after the last treatment with Holoclar.
    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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 Yes
    E.8.1.7.1Other trial design description
    prospective trial
    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 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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 Patient Last Visit
    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 months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months8
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 5
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 73
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 14
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 92
    F.4.2.2In the whole clinical trial 92
    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)
    After their participation in the trial, the subjects will be treated according to the standard treatment care expected for their condition. The patients will be proposed to enter the safety and efficacy long-term follow-up study.
    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 Decision2015-10-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-09-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-03-11
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