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    Summary
    EudraCT Number:2020-002255-37
    Sponsor's Protocol Code Number:MGT-RPGR-022
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-11-02
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2020-002255-37
    A.3Full title of the trial
    Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene
    Estudio de fase 3 de seguimiento de AAV5-hRKp.RPGR para el tratamiento de la retinitis pigmentosa ligada al cromosoma X asociada con variantes en el gen RPGR
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.
    Estudio de seguimiento a largo plazo de un ensayo de terapia génica para pacientes con retinitis pigmentosa (reducción progresiva de la visión) debido a un defecto en el cromosoma X.
    A.3.2Name or abbreviated title of the trial where available
    Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR
    Estudio de seguimiento a largo plazo de un ensayo de terapia génica para pacientes con retinitis pig
    A.4.1Sponsor's protocol code numberMGT-RPGR-022
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/125/2021
    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 UK II Limited
    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 Limited
    B.5.2Functional name of contact pointDominic Fitzsimmons
    B.5.3 Address:
    B.5.3.1Street Address34-38 Provost Street
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeN1 7NQ
    B.5.3.4CountryUnited Kingdom
    B.5.6E-maildominic.fitzsimmons@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 nameAAV5-hRKp.RPGR
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubretinal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNbotaretigene sparoparvovec
    D.3.9.3Other descriptive nameAAV5-hRKp.RPGR
    D.3.9.4EV Substance CodeSUB188497
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400000000000
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberGene therapy medicinal product; EMA/CAT/14302/2020
    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.IMP: 2
    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 nameAAV5-hRKp.RPGR
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubretinal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNbotaretigene sparoparvovec
    D.3.9.3Other descriptive nameAAV5-hRKp.RPGR
    D.3.9.4EV Substance CodeSUB188497
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200000000000
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberGene therapy medicinal product; EMA/CAT/14302/2020
    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
    Retinitis pigmentosa ligada al cromosoma X causada por mutaciones en el gen RPGR
    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).
    Reducción progresiva de la visión, comenzando con ceguera nocturna y progreso a restricción del campo visual, causado por mutaciones en el cormosoma X (gen RPGR)
    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
    To assess the long-term safety of AAV5-hRKp.RPGR in individuals with RPGR-XLRP
    To assess the long-term efficacy of treatment with AAV5-hRKp.RPGR in individuals with RPGR-XLRP based on assessments of retinal function.
    Evaluar la seguridad a largo plazo de AAV5-hRKp.RPGR en personas con RPGR-XLRP
    Evaluar la eficacia a largo plazo del tratamiento con AAV5-hRKp.RPGR en personas con RPGR-XLRP según las evaluaciones de la función retiniana
    E.2.2Secondary objectives of the trial
    To assess changes in all participants after treatment in: retinal function, functional vision, visual function and to assess the safety and tolerability of bilateral subretinal delivery of AAV5-hRKp.RPGR.
    Evaluar los cambios después del tratamiento en todos los participantes, con respecto a función retiniana, visión funcional, función visual y evaluar la seguridad y tolerabilidad de la administración subretiniana bilateral de AAV5-hRKp.RPGR
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Previously completed participation in Study MGT-RPGR-021.
    2.Must reconfirm that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study.
    1. Participación previamente completada en el estudio MGT-RPGR-021.
    2. Debe volver a confirmar que entiende el propósito y los procedimientos exigidos para el estudio, y que está dispuesto a participar en el estudio.
    E.4Principal exclusion criteria
    There are no specific exclusion criteria.
    No hay criterios de exclusión específicos
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy: Change in retinal sensitivity by pointwise comparison of individual loci in a 185-point customized grid over 60 months
    Eficacia: Cambios en la sensibilidad retiniana mediante la comparación puntual de loci individuales en una cuadrícula personalizada de 185 puntos después de la administración subretiniana bilateral durante 60 meses
    E.5.1.1Timepoint(s) of evaluation of this end point
    For participants in the long-term follow-up group; (Month 18, 24, 36, 48, and 60)
    For participants in the MGT-RPGR-022 treatment group, from baseline to week 52; and follow up at Months 18, 24, 36, 48 and 60.
    Para los participantes en el grupo de seguimiento de seguridad a largo plazo; (mes 18, 24m 36, 48 y 60)
    Para participantes en el grupo de tratamiento MGT-RPGR-022, desde el valor de referencia a semana 52; y seguimiento en el mes 8, 24, 36, 48 y 60
    E.5.2Secondary end point(s)
    Major secondary efficacy endpoints will include the following:
    •Retinal Function assessed by
    -Static perimetry: V30 from Visual Field Modeling Analysis (VFMA) modeling
    -Average threshold by microperimetry, under mesopic condition
    •Functional Vision assessed by
    -visual mobility assessment
    -Impact of Vision Impairment on Adults (IVI-A) – Reading and Accessing Information domain score – for adults (age 18 years and older)
    •Visual Function assessed by
    -Low luminance BCVA by ETDRS chart letter score
    -Contrast sensitivity by Pelli-Robson chart in LogMAR
    Los criterios de valoración orincipales incluirán los siguiente:
    • Función retiniana evaluada por
    -V30 mediante análisis de modelado de campo visual (VFMA),
    - umbral promedio de microperimetría en condiciones mesópicas
    • Visión funcional evaluada por
    - evaluación de movilidad visual.
    - Impacto de la insuficiencia visual en adultos (IVI-A)- Puntuación del
    dominio lectura y acceso a la información - para adultos (mayores de 18 años)
    • Función visual evaluada por
    - Baja luminancia BCVA según la puntuación de las letras del gráfico
    ETDRS
    - Sensibilidad al contraste según la gráfica de Pelli-Robson en LogMAR
    E.5.2.1Timepoint(s) of evaluation of this end point
    Major secondary efficacy endpoints up to 60 months
    Criterios de valoración secundarios principales hasta los 60 meses
    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) 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) Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Grp tratamiento aplazado y seguridad largo plazo.Esfuerzos enmascarar evaluadores de vision
    Deferred treatment grp & long-term safety. Efforts to mask vision assessors to treatment assignment
    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 trial2
    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 EEA14
    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
    Canada
    Israel
    United States
    Belgium
    Denmark
    France
    Germany
    Ireland
    Italy
    Netherlands
    Spain
    Switzerland
    United Kingdom
    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
    LVLS
    Última visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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.1.1Number of subjects for this age range: 6
    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: 3
    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: 60
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children aged 3 years and older
    Niños de 3 años en adelante
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    F.4.2.2In the whole clinical trial 66
    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 is a one-time treatment. Subjects that were randomised to deferred treatment will be offered bilateral treatment as part of the MGT-RPGR-022 study and followed for 5 years. No further treatment will be offered after subjects end participation.
    La intervención es un tratamiento único. A los sujetos aleatorizados para el tratamiento aplazado se les ofrecerá tratamiento bilateral como parte del estudio MGT-RPGR-022 y se les realizará un seguimiento durante 5 años. No se ofrecerá ningún tratamiento adicional una vez que los sujetos terminen su participación.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-03-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-22
    P. End of Trial
    P.End of Trial StatusOngoing
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