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The European Union Clinical Trials Register allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   41229   clinical trials with a EudraCT protocol, of which   6756   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    EudraCT Number:2015-001344-11
    Sponsor's Protocol Code Number:CCD-GPLSCD01-03-FU
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-09-29
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2015-001344-11
    A.3Full title of the trial
    Multinational, multicenter, prospective, long-term safety and efficacy follow-up study after Autologous Cultivated Limbal Stem Cells Transplantation (ACLSCT) for restoration of corneal epithelium in patients with limbal stem cell deficiency due to ocular burns (HOLOCORE-FU)
    Studio di controllo (follow up) multinazionale, multicentrico, prospettico, di efficacia e di sicurezza a lungo termine dopo trapianto di cellule staminali limbari autologhe coltivate (ACLSCT) finalizzato al ripristino dell’epitelio corneale in pazienti con deficit di cellule staminali limbari dovuto ad ustioni oculari (HOLOCORE-FU)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to follow up the implantation of Holoclar on the cornea of patients with ocular burns
    Studio clinico per seguire l'impianto di Holoclar sulla cornea di pazienti con ustioni oculari
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberCCD-GPLSCD01-03-FU
    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.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 SpA
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationChiesi Farmaceutici Spa
    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.5.4Telephone number003905211689281
    B.5.5Fax number00390521774468
    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. name HOLOCLAR
    D. of the Marketing Authorisation holderChiesi Farmaceutici S.p.A.
    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.3.2Product code [Holoclar]
    D.3.4Pharmaceutical form Implant
    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 INNEx vivo autologous corneal epithelial cells including stem cells
    D.3.9.2Current sponsor codeGPLSCD01
    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 number4
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product Yes
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product Yes
    D. 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 Yes
    D. medicinal product typeATIMP
    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
    Lesioni corneali con associato deficit di cellule staminali limbari da moderato a grave dovuto a ustioni oculari
    E.1.1.1Medical condition in easily understood language
    corneal damage due to ocular burns
    Danneggiamento corneale dovuto a ustioni oculari
    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 long term safety of one or two implants with Holoclar in patients suffering from moderate to severe LSCD secondary to ocular burns.
    Dimostrare la sicurezza a lungo termine di uno o due ACLSCT con Holoclar in pazienti affetti da LSCD da moderato a grave in seguito ad ustioni oculari.
    E.2.2Secondary objectives of the trial
    1)To evaluate the long-term efficacy of one or two ACLSCT(s), the degree of superficial corneal neo-vascularization and corneal epithelial stability, clinical symptoms, conjunctival inflammation, visual acuity, quality of life, tear secretion compared to baseline (i.e. before the first ACLSCT) and long term efficacy based on clinical judgment of the investigator;
    2) To evaluate safety and clinical outcomes (i.e. superficial corneal neo-vascularization, epithelial defects, visual acuity, conjunctival inflammation, and symptoms) after keratoplasty
    in patients previously treated with Holoclar.
    1) Valutare l'efficacia a lungo termine di uno o due ACLSCT, il grado di neo-vascolarizzazione corneale superficiale e la stabilità epiteliale corneale, i sintomi clinici, l’infiammazione congiuntivale, l’acuità visiva, la qualità di vita, la secrezione lacrimale rispetto alla baseline (cioè antecedente al primo ACLSCT) ed l’efficacia a lungo termine secondo il giudizio clinico dello sperimentatore;
    2) Valutare gli esiti clinici e di sicurezza (cioè neo-vascolarizzazione corneale superficiale, difetti epiteliali, acuità visiva, infiammazione congiuntivale e sintomi) dopo cheratoplastica nei pazienti sottoposti in precedenza a trattamento con Holoclar.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    All patients (adults and pediatrics) who completed the HOLOCORE core study and who consent to roll over to the present extension study at the end of the HOLOCORE follow-up.
    Tutti i pazienti (adulti e pediatrici) che hanno completato lo studio principale HOLOCORE e che acconsentonoall'arruolamento in questo studio di estensione al termine del controllo di HOLOCORE.
    E.4Principal exclusion criteria
    No specific exclusion criterion is considered for this study, except for patients dropping out from the HOLOCORE study or withdrawing
    Non si considerano criteri di esclusione specifici in questo studio, fatto salvo laddove i pazienti abbandonino lo studio HOLOCORE o ritirino il consenso.
    E.5 End points
    E.5.1Primary end point(s)
    Percentage of patients reporting Treatment-emergent adverse events (TEAEs), treatment-related adverse events (TRAEs), Adverse Event of Special Interest in accordance with the Risk Management Plan for Holoclar (AESI), including glaucoma and blepharitis.
    Percentuale di pazienti che riportano eventi avversi emersi durante il trattamento (TEAE), eventi avversi correlate al trattamento (TRAE), eventi avversi di speciale interesse (AESI), inclusi glaucoma e blefarite, in accordo con il Piano di Gestione del Rischio di Holoclar.
    E.5.1.1Timepoint(s) of evaluation of this end point
    All study duration
    Per tutta la durata dello studio
    E.5.2Secondary end point(s)
    Percentage of patients with successful transplantation at Visit 3 (success is defined on the basis of the degree of "superficial corneal neovascularization" and "epithelial defects")
    Percentuale di pazienti con trapianto riuscito a Visit 3 (il successo è definito sulla base del grado di "neovascolarizzazione corneale superificiale" e "difetti epiteliali")
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months after patient inclusion
    12 mesi dopo l'inclusione del paziente nello studio
    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 No
    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. 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 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 Yes
    E. trial design description
    Follow up dei pazienti che hanno ricevuto HOLOCLAR nel protocollo HOLOCORE.
    Follow up of patients who received HOLOCLAR in HOLOCORE protocol.
    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 concerned3
    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 Information not present in EudraCT
    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
    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 months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months9
    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 Yes
    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. of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 54
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 11
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2020-09-29. Yes
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women Yes
    F.3.3.4Nursing women Yes
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F. of subjects incapable of giving consent
    Minor patients
    Pazienti minorenni
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 70
    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)
    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 Decision2017-06-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-08
    P. End of Trial
    P.End of Trial StatusOngoing
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