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    Summary
    EudraCT Number:2014-004884-19
    Sponsor's Protocol Code Number:LENTICOL-F
    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:2015-02-18
    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 ConcernedUK - MHRA
    A.2EudraCT number2014-004884-19
    A.3Full title of the trial
    Phase I study of lentiviral-mediated COL7A1 gene-modified autologous fibroblasts in adults with recessive dystrophic epidermolysis bullosa (RDEB)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Gene Therapy Trial in adults with Inherited Blistering Skin Disease
    A.3.2Name or abbreviated title of the trial where available
    LENTICOL-F
    A.4.1Sponsor's protocol code numberLENTICOL-F
    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 SponsorKing's College London
    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 supportDebRA International
    B.4.2CountryAustria
    B.4.1Name of organisation providing supportSohana Research Fund
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKing's College London and Guy's and St Thomas' NHS Foundation Trust
    B.5.2Functional name of contact pointProfessor John A McGrath
    B.5.3 Address:
    B.5.3.1Street AddressSt John's Institute of Dermatology, King's College London, 9th Floor Tower Wing, Guy's Hospital
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeSE1 9RT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number442071886409
    B.5.5Fax number442071888050
    B.5.6E-mailjohn.mcgrath@kcl.ac.uk
    B.Sponsor: 2
    B.1.1Name of SponsorGuy's and St Thomas' 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 supportDebRA International
    B.4.2CountryAustria
    B.4.1Name of organisation providing supportSohana Research Fund
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGuy's and St Thomas' NHS Foundation Trust and King's College London
    B.5.2Functional name of contact pointProfessor John McGrath
    B.5.3 Address:
    B.5.3.1Street AddressSt John's Institute of Dermatology, King's College London, 9th Floor Tower Wing
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeSE1 9RT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number442071886409
    B.5.5Fax number442071888050
    B.5.6E-mailjohn.mcgrath@kcl.ac.uk
    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 nameSIN LV Mediated ex vivo transduced autologous fibroblasts expressing codon-optimised COL7A1
    D.3.2Product code Not applicable
    D.3.4Pharmaceutical form Suspension for injection in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntradermal use
    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 Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Recessive Dystrophic Epidermolysis Bullosa
    E.1.1.1Medical condition in easily understood language
    Severe inherited blistering disease
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level PT
    E.1.2Classification code 10014989
    E.1.2Term Epidermolysis bullosa
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety of intradermal injections of SIN LV-mediated COL7A1 gene-modified autologous fibroblasts in adults with RDEB.
    E.2.2Secondary objectives of the trial
    1. To evaluate the potential efficacy of intradermal injections of SIN LV-mediated COL7A1 gene-modified autologous fibroblasts in adults with RDEB at week (W) 2, month (M) 3 and M12 after the IMP injections.

    2. To evaluate the immune response against the recombinant type VII collagen (C7) at W2, W4, M3, M6 and M12 after the IMP injections compared to baseline.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Clinical and genetic diagnosis of RDEB with confirmed bi-allelic COL7A1 mutations.
    2. A reduced number or morphologically abnormal anchoring fibrils confirmed by TEM.
    3. At least 5x3cm of intact skin on the trunk and/or extremities that is suitable for cell injections.
    4. Able to undergo local anaesthesia.
    5. Subjects aged ≥ 17years and able to give informed consent prior to the first study intervention.
    E.4Principal exclusion criteria
    1. Subjects who received other investigational medicinal products within 6 months prior to enrolment into this study.
    2. Past medical history of biopsy proven skin malignancy.
    3. Subjects who have received immunotherapy including oral corticosteroids (Prednisolone >1mg/kg) for more than one week (intranasal and topical preparations are permitted) or chemotherapy within 60 days of enrolment into this study.
    4. Known allergy to any of the constituents of the investigational medicinal product (IMP).
    5. Subjects with BOTH:
     - positive serum antibodies to C7 confirmed by ELISA and
     - positive IIF with binding to the base of salt split skin.
    6. Subjects with positive results for HIV, Hepatitis B, Hepatitis C, HTLV or Syphilis.
    7. Subjects who are pregnant or of child-bearing potential who are neither abstinent nor practising an acceptable means of contraception when this is in line with the usual and preferred lifestyle of the subject, as determined by the Investigator, for 12 months after the cell injections.
    E.5 End points
    E.5.1Primary end point(s)
    Adverse events (AEs), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs) at each visit after screening over a 12 month follow up period.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At every visit over a 12 month period post IMP administration. Trial visits are scheduled at Week 1, Week 2, Week 4 , Month 3, Month 6, Month 9, Month 12,
    E.5.2Secondary end point(s)
    Skin biopsy analysis of treated skin at W2, M3 and M12 compared to untreated skin:

    I. C7 protein expression by immunofluorescence microscopy (IF)
    II. Morphology of anchoring fibrils at the dermal-epidermal junction (DEJ) by transmission electron microscopy (TEM)
    III. Vector copy number by quantitative polymerase chain reaction (qPCR)
     Serum analysis for:
    IV. Detection of anti-C7 antibodies by enzyme-linked immunosorbent assay (ELISA) (against NC1 and NC2 domains of C7) and indirect immunofluorescence (IIF) at W2, W4, M1, M3, M6 and M12 post-injections
    V. Detection of T-cell responses to the full length C7 by enzyme-linked immunosorbent spot (ELISPOT) assay at W4, M6 and M12 post-injections.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At the above specified visits over a 12 month follow up period post IMP administration.
    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) Yes
    E.7.1.1First administration to humans Yes
    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) 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.8.2.4Number 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.8.5The 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 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
    Last patient last visit (M12)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days30
    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: 10
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) No
    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 state10
    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)
    Once a participant has ended their participation in the trial, he/she will be followed up as per routine clinical care.
    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 Decision2015-04-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-05-24
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-03-13
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