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    Clinical Trial Results:
    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

    Summary
    EudraCT number
    		 2016-002290-35
    Trial protocol
    		 GB  
    Global end of trial date
    25 Oct 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 Aug 2021
    First version publication date
    26 Aug 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    MGT006
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    MeiraGTx II UK Ltd
    Sponsor organisation address
    34-38 Provost Street, London, United Kingdom, N17NH
    Public contact
    Julie Bakobaki, MeiraGTx II UK Ltd, +44 2038664320, julie.bakobaki@meiragtx.com
    Scientific contact
    Julie Bakobaki, MeiraGTx II UK Ltd, +44 2038664320, julie.bakobaki@meiragtx.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    20 Dec 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    25 Oct 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Oct 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective was to assess the safety of an AAV8 vector for hCNGB3 gene replacement in the retina.
    Protection of trial subjects
    Only participants who met the study entry criteria were enrolled in the study. All participants were free to withdraw from the study at any time for any reason. All participants were closely monitored throughout the study. In addition, safety was evaluated based on adverse events (including dose-limiting events), clinical laboratory assessments, vital sign measurements, and physical examinations.
    Background therapy
    		 None
    Evidence for comparator
    		 n/a
    Actual start date of recruitment
    16 Jan 2017
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety
    Long term follow-up duration
    		 5 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 20
    Country: Number of subjects enrolled
    United States: 3
    Worldwide total number of subjects
    23
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    6
    Adolescents (12-17 years)
    7
    Adults (18-64 years)
    10
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 n/a

    Period 1
    Period 1 title
    Dose-escalation phase/ Expansion Phase (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    n/a

    Arms
    Arm title
    		 AAV8-hCARp.hCNGB3 gene therapy
    Arm description
    		 Study participants were administered a single dose in the worse-seeing eye at one of four dose levels.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AAV8-hCARp.hCNGB3
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Subretinal use
    Dosage and administration details
    subjects were administered a single subretinal dose

    Number of subjects in period 1
    		AAV8-hCARp.hCNGB3 gene therapy
    Started
    23
    Completed
    23

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Dose-escalation phase/ Expansion Phase
    Reporting group description
    		 -

    Reporting group values
    		 Dose-escalation phase/ Expansion Phase Total
    Number of subjects
    		 23 23
    Age categorical
    median
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 6 6
        Adolescents (12-17 years)
    		 7 7
        Adults (18-64 years)
    		 10 10
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Age
    Units: years
        median (full range (min-max))
    		 13.0 (5 to 33) -
    Gender categorical
    Units: Subjects
        Female
    		 18 18
        Male
    		 5 5
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 0 0
        White
    		 23 23
    Subject analysis sets

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Safety Analysis Set (all enrolled participants who were administered ATIMP)

    Subject analysis sets values
    		 Safety Analysis Set
    Number of subjects
    23
    Age categorical
    median
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
        Newborns (0-27 days)
    0
        Infants and toddlers (28 days-23 months)
    0
        Children (2-11 years)
    6
        Adolescents (12-17 years)
    7
        Adults (18-64 years)
    10
        From 65-84 years
    0
        85 years and over
    0
    Age continuous
    Age
    Units: years
        median (full range (min-max))
    13.0 (5 to 33)
    Gender categorical
    Units: Subjects
        Female
    18
        Male
    5
    Race
    Units: Subjects
        American Indian or Alaska Native
    0
        Asian
    0
        Native Hawaiian or Other Pacific Islander
    0
        Black or African American
    0
        White
    23

    End points

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    End points reporting groups
    Reporting group title
    AAV8-hCARp.hCNGB3 gene therapy
    Reporting group description
    		 Study participants were administered a single dose in the worse-seeing eye at one of four dose levels.

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Safety Analysis Set (all enrolled participants who were administered ATIMP)

    Primary: Primary Safety outcomes

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    End point title
    		 Primary Safety outcomes [1]
    End point description
    End point type
    Primary
    End point timeframe
    Occurring during the 6 weeks following ATIMP administration
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: This is a single group study.
    End point values
    		 AAV8-hCARp.hCNGB3 gene therapy Safety Analysis Set
    Number of subjects analysed
    23
    23
    Units: Number of events
    23
    23
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    To last visit (Week 24 after ATIMP administration)
    Adverse event reporting additional description
    Most ocular AEs were temporally related to surgery, mild in severity and resolved with little or no intervention. For example, transient reduction in visual acuity was related to manipulation of the eye during surgery and all such AEs resolved within 38 days.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    Safety Analysis Set
    Reporting group description
    		 all enrolled participants who were administered ATIMP

    Serious adverse events
    		 Safety Analysis Set
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 23 (8.70%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Pregnancy, puerperium and perinatal conditions
    Ectopic pregnancy
         subjects affected / exposed
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Uveitis
         subjects affected / exposed
    2 / 23 (8.70%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Safety Analysis Set
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    23 / 23 (100.00%)
    Investigations
    Intraocular pressure increased
         subjects affected / exposed
    6 / 23 (26.09%)
         occurrences all number
    6
    Nervous system disorders
    Headache
         subjects affected / exposed
    6 / 23 (26.09%)
         occurrences all number
    6
    Visual field defect
         subjects affected / exposed
    5 / 23 (21.74%)
         occurrences all number
    5
    Dizziness
         subjects affected / exposed
    4 / 23 (17.39%)
         occurrences all number
    4
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    3 / 23 (13.04%)
         occurrences all number
    3
    Eye disorders
    Conjunctival haemorrhage
         subjects affected / exposed
    20 / 23 (86.96%)
         occurrences all number
    20
    Lenticular opacities
         subjects affected / exposed
    15 / 23 (65.22%)
         occurrences all number
    15
    Visual acuity reduced
         subjects affected / exposed
    15 / 23 (65.22%)
         occurrences all number
    15
    Subretinal fluid
         subjects affected / exposed
    9 / 23 (39.13%)
         occurrences all number
    9
    foreign body sensation in eye
         subjects affected / exposed
    8 / 23 (34.78%)
         occurrences all number
    8
    Ocular discomfort
         subjects affected / exposed
    8 / 23 (34.78%)
         occurrences all number
    8
    Chorioretinal folds
         subjects affected / exposed
    6 / 23 (26.09%)
         occurrences all number
    6
    Retinal haemorrhage
         subjects affected / exposed
    6 / 23 (26.09%)
         occurrences all number
    6
    Conjunctival hyperaemia
         subjects affected / exposed
    4 / 23 (17.39%)
         occurrences all number
    4
    Eye inflammation
         subjects affected / exposed
    4 / 23 (17.39%)
         occurrences all number
    4
    Maculopathy
         subjects affected / exposed
    4 / 23 (17.39%)
         occurrences all number
    4
    Retinal depigmentation
         subjects affected / exposed
    4 / 23 (17.39%)
         occurrences all number
    4
    Retinal oedema
         subjects affected / exposed
    4 / 23 (17.39%)
         occurrences all number
    4
    Retinal tear
         subjects affected / exposed
    4 / 23 (17.39%)
         occurrences all number
    4
    eye pain
         subjects affected / exposed
    3 / 23 (13.04%)
         occurrences all number
    3
    Photophobia
         subjects affected / exposed
    3 / 23 (13.04%)
         occurrences all number
    3
    Vitreous floaters
         subjects affected / exposed
    3 / 23 (13.04%)
         occurrences all number
    3
    Conjunctival oedema
         subjects affected / exposed
    2 / 23 (8.70%)
         occurrences all number
    2
    Corneal disorder
         subjects affected / exposed
    2 / 23 (8.70%)
         occurrences all number
    2
    Macular fibrosis
         subjects affected / exposed
    2 / 23 (8.70%)
         occurrences all number
    2
    Retinal disorder
         subjects affected / exposed
    2 / 23 (8.70%)
         occurrences all number
    2
    Retinal pigment epitheliopathy
         subjects affected / exposed
    2 / 23 (8.70%)
         occurrences all number
    2
    Uveitis
         subjects affected / exposed
    2 / 23 (8.70%)
         occurrences all number
    2
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    8 / 23 (34.78%)
         occurrences all number
    8
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 23 (8.70%)
         occurrences all number
    2
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    2 / 23 (8.70%)
         occurrences all number
    2
    Infections and infestations
    Postoperative wound infection
         subjects affected / exposed
    2 / 23 (8.70%)
         occurrences all number
    2
    Rhinitis
         subjects affected / exposed
    5 / 23 (21.74%)
         occurrences all number
    5

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    23 Oct 2017
    Protocol v1.0 to v2.0 - Clarifications to outcome assessments, definitions and risks
    08 Mar 2018
    Protocol v2.0 to v3.0 - Clarifications in risks and trial design for expansion phase, addition of sample
    15 May 2018
    Protocol v3.0 to V4.0 - Updates to risks, clarifications in outcome assessments & surgical procedure.
    19 Dec 2018
    Protocol V4.0 to v5.0 - Increase in sample size, clarifications in dosing 19-Dec-2018 - Protocol v5.0 to v6.0 - HRA requested clarification on dosing (This led SA04 to be withdrawn and subsequent submission of SA05)
    07 Feb 2019
    Protocol v6.0 to v7.0 - Updates to secondary outcome assessments

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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