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    Summary
    EudraCT Number:2016-003967-21
    Sponsor's Protocol Code Number:MGT009
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2017-09-14
    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 number2016-003967-21
    A.3Full title of the trial
    An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigmentosa owing to defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Gene Therapy Trial for People with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.
    A.3.2Name or abbreviated title of the trial where available
    Gene Therapy Trial for People with Retinitis Pigmentosa: RPGR
    A.4.1Sponsor's protocol code numberMGT009
    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 SponsorMeiraGTx UK II Limited
    B.1.3.4CountryUnited Kingdom
    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 supportMeiraGTx
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMeiraGTx II UK Ltd
    B.5.2Functional name of contact pointOyinda Oluwole
    B.5.3 Address:
    B.5.3.1Street Address92 Britannia Walk
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeN1 7NQ
    B.5.3.4CountryUnited Kingdom
    B.5.6E-mailoyinda.oluwole@meiragtx.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 No
    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/3/16/1715
    D.3 Description of the IMP
    D.3.1Product nameAAV2/5-hRKp.RPGR
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraocular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAAV2/5-hRKp.RPGR
    D.3.9.2Current sponsor codeAAV2/5-hRKp.RPGR
    D.3.10 Strength
    D.3.10.1Concentration unit billion organisms/ml billion organisms/millilitre
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number1000
    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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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
    X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
    E.1.1.1Medical condition in easily understood language
    Progressive reduction in vision, starting with night blindness and progressing to visual field constriction, caused by mutations on Chromosome X (RPGR gene).
    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 PT
    E.1.2Classification code 10038914
    E.1.2Term Retinitis pigmentosa
    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
    The primary research objective is to assess the safety of AAV2/5-hRKp.RPGR, for RGPR-ORF15 gene replacement in the retina of patients with RPGR XLRP.
    Safety is defined as the absence of an ATIMP-related reduction in visual acuity by 15 ETDRS letters or more, severe unresponsive inflammation, infective endophthalmitis, ocular malignancy and grade III or above non-ocular SUSAR.
    E.2.2Secondary objectives of the trial
    The secondary research objective is to determine whether AAV2/5-hRKp.RPGR for RGPR-ORF15 gene replacement in the retina can slow/halt progressive deterioration in retinal structure or visual function, and improve retinal function, visual function and quality of life in patients with RPGR XLRP.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion in the trial will be limited to individuals who meet the following criteria:
    1. Are males aged 5 years or older
    2. Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI)
    3. Have relatively symmetrical retinal disease - both structurally and functionally defined as < 15 letters [Best Corrected Visual Acuity (BCVA)] difference between eyes
    4. Have a predicted disease-causing missense or null mutation in RPGR confirmed in an
    accredited laboratory
    5. Evidence of relative preservation of retinal structure at the macula
    6. Evidence of impaired navigation in dim illumination on Mobility assessment in both eyes monocularly; defined by either an inability to complete the visual mobility maze at the 1 lux level, or the time taken to complete the maze at either 1 or 4 lux being greater or equal to 12 seconds.
    7. Are able to give informed consent or assent, with the guidance of their parent/guardian where appropriate: children aged 5-6 years will not be asked to provide assent
    8. Are able to undertake age appropriate clinical assessments at the trial sites as specified in the protocol
    9. Are willing to use barrier and spermicide form of contraception or will be sexually abstinent (when this is in line with the participant’s preferred and usual lifestyle) for at least 12 months following ATIMP administration
    10. Are willing to give consent for the use of blood and blood components collected through the trial for the investigation of immune responses to the ATIMP
    E.4Principal exclusion criteria
    Individuals will be excluded if they meet any of the following criteria:
    1. Have a known allergy to any of the non-investigational drugs to be used in the trial as defined in Section 5.4.1
    2. Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
    3. Have any other condition that the CI/PI considers makes them inappropriate for entry into the trial, inclusive of but not limited to a history of the following:
    o Uncontrolled hypertension defined as a systolic value ≥160mmHg or diastolic value ≥100mmHg.
    o Uncontrolled diabetes mellitus defined as an HbA1c ≥9% (75mmol/mol) at screening.
    o Uncontrolled heart failure (NYHA class II-IV).
    o Any history of tuberculosis
    o Chronic kidney disease (defined as eGFR ≤60ml/min calculated using Cockroft Gault or MDRD equations.
    o Immunocompromised state (including long term immunosuppressant therapy).
    o Osteoporosis (defined as presence of 1 or more non-traumatic “fragility” fractures or proven BMD of 2.5SD less than anticipated as demonstrated on DEXA scan).
    o Active peptic ulcer disease or uncontrolled gastro-oesophageal reflux. o Severe affective disorder or past history of drug induced psychosis
    4. Use of high dose regular non-steroidal anti-inflammatory drugs at the time of screening.
    5. Have an ocular or systemic disorder that may preclude subretinal surgery and/or interfere with interpretation of the study results
    6. Have had intraocular surgery within 3 months of screening
    7. Have recently (within 4 weeks of last dose of concomitant immunosuppressive therapy) received a live vaccine
    8. Have a condition that requires glucocorticoid replacement therapy, such as in endocrine diseases, if this would interfere with the immunosuppressive regime.
    9. Have known chronic hepatitis B infection as indicated by detectable surface antigen. Entry with negative surface antigens but positive hepatitis core antibody will be allowed however additional monitoring throughout the trial may be required.
    10. Are unwilling to consider the possibility of entry into a subsequent longer term follow up study
    E.5 End points
    E.5.1Primary end point(s)
    The primary outcome is defined as absence of any of the below occurring during the 9 weeks following administration, at least possibly related to the ATIMP, not surgery alone:
    Reduction in visual acuity by 15 ETDRS letters or more
    Severe unresponsive inflammation
    Infective endophthalmitis
    Ocular malignancy
    Grade III or above non-ocular SUSAR
    E.5.1.1Timepoint(s) of evaluation of this end point
    This endpoint will be evaluated at 1 day, 3 days, 1 week, 2 weeks, 4 weeks, 6 weeks, 9 weeks, 12 weeks, 24 weeks, 9 months, 12 months, and 18 months after subretinal administration of the intervention.

    Safety will be assessed for 12 months after the intervention in this study, and a further 4 years in a separate follow on study.
    E.5.2Secondary end point(s)
    The secondary outcomes are measures of the efficacy of the ATIMP; these will be performed on an individual participant basis and will be descriptive in nature.
    - Slowing or halting of progressive deterioration in retinal structure or visual function that is greater than the baseline variation for that test and is sustained for at least 2 consecutive assessments. Slowing or halting of progression over time may be facilitated by comparing identical structural and functional assessments acquired prior to intervention/injection to better determine rate of change over time in this slowly progressive disease #.
    #Historical images will be collected wherever possible with written informed consent from the participants
    - Any improvements in visual function from baseline that are greater than the baseline variation and are sustained for at least two consecutive assessments.
    - Any improvement in retinal function from pre-intervention that is greater than basleine variation and measurable by electrophysiology (pattern ERG, multifocal ERG or full-field ERG).
    - Quality of life will be measured by the Impact of Visual Impairment (IVI) questionnaire, the low Luminance Questionnaire (LLQ) and the EQ5D-5L and EQ5D-5Y as appropriate.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary endpoints will be evaluated at various time-points between 12 weeks and 18 months after subretinal administration of the intervention.
    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 Yes
    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) 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 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 Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United Kingdom
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days24
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days3
    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: 23
    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: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 13
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 48
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children under 6 years
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Children above 6 years
    F.4 Planned number of subjects to be included
    F.4.1In the member state48
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 48
    F.4.2.2In the whole clinical trial 71
    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 intervention will only be provided one time to participants. At the end of this trial, participants will be invited to enrol in a separate long-term follow-up study that will continue to monitor safety and efficacy for 5 years after administration of the intervention.
    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 CRN: North Thames Central Office
    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 Decision2017-06-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-05-04
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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