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    Summary
    EudraCT Number:2020-002873-88
    Sponsor's Protocol Code Number:MGT-RPGR-021
    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 number2020-002873-88
    A.3Full title of the trial
    Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene
    Estudio de fase 3 aleatorizado y controlado 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
    Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.
    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
    Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR
    A.4.1Sponsor's protocol code numberMGT-RPGR-021
    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 UK II 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/14302/2020EMA/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 effect of bilateral treatment with AAV5-hRKp.RPGR on retinal function as measured by static perimetry.
    Evaluar la eficacia del tratamiento bilateral con AAV5-hRKp.RPGR en la función retiniana de acuerdo con medición mediante perimetría estática
    E.2.2Secondary objectives of the trial
    To assess changes after treatment administration 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 de la administración del tratamiento en 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. Male or female.
    2. 3 years of age or older.
    3. Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by a sponsor-approved laboratory.
    1. Ser hombre o mujer.
    2. Tener 3 o más años de edad.
    3. Tener XLRP confirmada por un especialista en retinas y tener una variante de secuencia que se predice que causa la enfermedad en el gen RPGR confirmada por un laboratorio aprobado por el promotor.
    E.4Principal exclusion criteria
    1. Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration.
    2. Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule.
    3. Has undergone prior retinal surgery involving the macula, vitrectomy, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery (except cataract surgery).
    4. History of an ocular implant, with the exception of an intraocular lens.
    1. Se le ha realizado una cirugía ocular dentro de los 3 meses previos a la selección o se prevé que necesite cirugía ocular dentro de los 6 meses posteriores a la administración de la intervención del estudio.
    2. Cualquier tratamiento ocular experimental o cualquier otro tratamiento ocular que pudiera confundir la interpretación de los resultados de eficacia o afectar el cumplimiento del participante con el calendario de visitas.
    3. Se ha sometido a una cirugía de retina previa que afecta la mácula, vitectromía, fotocoagulación macular con láser, radioterapia de haz externo, termoterapia transpupilar, cirugía de filtración de glaucoma o cirugía de córnea (excepto la cirugía de cataratas).
    4. Antecedentes de implante ocular, con la excepción de una lente intraocular.
    E.5 End points
    E.5.1Primary end point(s)
    Change in retinal sensitivity by pointwise comparison of individual loci in a 185-point customized grid at Week 52.
    Cambio en la sensibilidad retiniana mediante comparación puntual de loci individuales en una cuadrícula personalizada de 185 puntos en la semana 52
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline to week 52
    Desde el valor de referencia a la semana 52
    E.5.2Secondary end point(s)
    •Retinal Function assessed by
    Static perimetry: V30 from 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
    • Función retiniana evaluada por V30 mediante el modelado V 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
    Baseline to week 52
    Desde el valor de referencia a la semana 52
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Evaluadores de la visión a ser enmascarados para la asignación de tratamiento
    Vision assessors to be masked 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 Yes
    E.8.2.3.1Comparator description
    Ningún tratamiento (tratamiento aplazado para el grupo control)
    No treatment (Deferred treatment for control group).
    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 years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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 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 randomised to immediate treatment will receive bilateral treatment during the study, no further treatment will be provided. Subjects receiving treatment will be followed for 1 year and an additional 4 years as part of the follow-up study. Subjects 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 1tratamiento único.Los sujetos aleatorizados a tto.inmediat recibirán tto.bilateral durante el estudio,no se proporcionará ningún tto. adicional.A los que reciben tto.se les realizará seguimient durant 1año y 4adicionales como parte de estudio de seguimient.A sujetos aleatorizads a tto.aplazado se les ofrecerá tto.bilateral como parte d estudio MGT-RPGR-022 y se les realizará seguimient durant 5años.No se ofrecerá ningún tto.adicional 1vez q los sujets terminen su participac
    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 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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