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    Summary
    EudraCT Number:2016-002290-35
    Sponsor's Protocol Code Number:MGT006
    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:2016-11-17
    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 number2016-002290-35
    A.3Full title of the trial
    An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/8-hCARp.hCNGB3) for gene therapy of adults and children with achromatopsia owing to defects in CNGB3
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Gene Therapy Trial for People with Achromatopsia (unable to see colours) due to a gene defect
    A.3.2Name or abbreviated title of the trial where available
    Gene Therapy for Achromatopsia: CNGB3
    A.4.1Sponsor's protocol code numberMGT006
    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 pointAmy De Sa
    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-mailamy.de.sa@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/3/15/1578
    D.3 Description of the IMP
    D.3.1Product nameAAV2/8-hCARp.hCNGB3
    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.3.10 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
    Achromatopsia caused by mutations in the CNGB3 gene
    E.1.1.1Medical condition in easily understood language
    Impairment of colour vision and visual acuity caused by mutations in the CNGB3 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 LLT
    E.1.2Classification code 10000454
    E.1.2Term Achromatopsia
    E.1.2System Organ Class 100000004850
    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 a AAV2/8 vector for hCNGB3 gene replacement in the retina. 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
    • Grade III or above non-ocular SUSAR
    E.2.2Secondary objectives of the trial
    The secondary research objective is to determine whether a AAV2/8 vector for hCNGB3 gene replacement in the retina can improve retinal function, visual function and quality of life.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion in the trial will be limited to individuals who:
    • Are aged 3 years or older (children will be included only once the maximal tolerated dose has been determined)
    • Have Achromatopsia caused by mutations in CNGB3
    • Present evidence of preservation of photoreceptors at the macula
    • Are able to undertake age-appropriate clinical assessments
    • Are willing to give consent for the use of blood and blood components collected throughout the trial for the investigation of immune response to ATIMP
    E.4Principal exclusion criteria
    Individuals will be excluded who:
    • Are females who are pregnant or breastfeeding
    • Had intra-ocular surgery within 6 months of screening
    • Have an ocular or systemic disorder that may preclude subretinal surgery and/or interfere with interpretation of the study results.
    • Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months
    • Have any other condition that the PI considers makes them inappropriate for entry into the trial, inclusive of but not limited to a history of the following: hypertension, diabetes mellitus, tuberculosis, renal impairment, immunocompromised
    state, osteoporosis, gastric ulceration or severe affective disorder
    • 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 any of the below occurring during the 6 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, 12 weeks, and 24 weeks after subretinal administration of the intervention.
    Safety will be assessed for a further 4.5 years in a separate subsequent study.
    E.5.2Secondary end point(s)
    The secondary outcomes are measures of the efficacy of the intervention, which will be performed on an individual participant basis and will be descriptive in nature.
    • Any improvements in visual function from baseline that are greater than the test-retest variation and are sustained for at least two consecutive assessments.
    • Any improvement in retinal function from pre-intervention that is greater than test-retest 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 and the EQ5D-5L and EQ5D-Y
    E.5.2.1Timepoint(s) of evaluation of this end point
    This endpoint will be evaluated at 3 months and 6 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 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
    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 months11
    E.8.9.1In the Member State concerned days19
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days7
    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: 9
    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: 4
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 18
    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 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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 27
    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 Decision2016-12-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-10-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-10-25
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