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    Clinical Trial Results:
    An open label Phase 2 clinical trial of retinal gene therapy for choroideremia using an adeno-associated viral vector (AAV2) encoding Rab-escort protein 1 (REP1)

    Summary
    EudraCT number
    2015-001383-18
    Trial protocol
    GB  
    Global end of trial date
    23 Jul 2021

    Results information
    Results version number
    v1(current)
    This version publication date
    28 Oct 2022
    First version publication date
    28 Oct 2022
    Other versions
    Summary report(s)
    BCVA ETDRS Scores, Full Dataset
    BCVA ETDRS Score Change from Baseline
    BCVA ETDRS Score Change from Baseline, Between Eye Comparison
    BCVA ETDRS Score Change from Baseline by Surgery site

    Trial information

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    Trial identification
    Sponsor protocol code
    REGENERATE (PID 11351)
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02407678
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    University of Oxford
    Sponsor organisation address
    Joint Research Office, Boundary Brook House, Churchill Drive, Oxford, United Kingdom, OX3 7LQ
    Public contact
    Research Governance, Ethics & Assurance Team, Research Services, ctrg@admin.ox.ac.uk
    Scientific contact
    Ophthalmology Trials Unit, Nuffield Laboratory of Ophthalmology, trials@eye.ox.ac.uk
    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
    03 Mar 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    23 Jul 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Jul 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Investigation of the efficacy and safety of a single subretinal injection of a recombinant adeno-associated virus serotype 2 (AAV2) vector encoding Rab-escort protein 1 (REP1), designated as AAV2-REP1, in subjects with a confirmed diagnosis of choroideremia over a 2-year assessment period following treatment. Efficacy was primarily quantified by tracking the comparative change from baseline in best corrected visual acuity (BCVA) in the treated eye and untreated contralateral eye (control eye), measured by the number of letters read on an Early Treatment Diabetic Retinopathy Study (ETDRS) chart.
    Protection of trial subjects
    The method of vector administration was refined in an antecedent Phase 1/2 interventional study (EudraCT 2009-014617-27) where initial detachment of the retina was effected as a first step by injecting a small volume of balanced salt solution underneath the retina through a very fine needle that is narrower than a human hair, thereby creating a small fluid-filled blister or bleb under the retina. This was followed by injection of an exact dose of the adeno-associated viral (AAV) vector suspension into the subretinal fluid as a second step. The small area of retinal detachment is temporary and disappears over about 24 hours as the fluid gets slowly absorbed by the retina. This type of surgery normally lasts about an hour, and the operation itself (without administration of the gene therapy) is a routine procedure for patients with conditions such as retinal detachment. The benefit of the preceding two-step procedure is that the slightly unpredictable part of the procedure (the retinal detachment) is completed before any AAV vector is administered to the subject. This allows for the management of any surgical complications without concerns about AAV vector dissemination. An additional safety feature introduced in this Phase 2 interventional study was the use of an ophthalmic operating microscope with an integrated optical coherence tomography (OCT) scanner for conducting the retinal surgery. This special operating microscope shows a cross section of the retina in real time to the surgeon, thereby permitting administration of the gene therapy to be conducted far more precisely, safely and reliably.
    Background therapy
    In order to minimise postoperative inflammation, subjects were given a 45 day course of oral prednisolone, starting 3 days before surgery at a daily dose of 1 mg per kg of body weight (rounded to the nearest multiple of 5 mg), and gradually tapering off to 5 mg over the course of the next 42 days.
    Evidence for comparator
    Not applicable: no comparator was used in the study.
    Actual start date of recruitment
    16 Aug 2016
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Safety, Efficacy
    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: 30
    Worldwide total number of subjects
    30
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    30
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    A total of 30 subjects were recruited over the period 2016-2019, comprising 12 subjects recruited and treated at Oxford Eye Hospital and 18 subjects recruited and treated at Moorfields Eye Hospital (London).

    Pre-assignment
    Screening details
    Subjects were males aged 18 years or older with: • A clinical diagnosis of choroideremia and a molecular diagnosis of a null mutation in the gene encoding REP1. • Active disease visible clinically within the macula region. • BCVA better than or equal to 6/60 (20/200) in the eye to be treated.

    Period 1
    Period 1 title
    Overall study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded
    Blinding implementation details
    This was an open label study with no masking. However, in order to minimise bias evaluation of the treated eye and untreated contralateral eye (control eye), ophthalmic assessments were conducted by an appropriately qualified masked observer once the subjects' treated eyes had regained their normal appearance and function following the surgical procedure.

    Arms
    Are arms mutually exclusive
    No

    Arm title
    Cohort 1
    Arm description
    Cohort 1 comprised all subjects that participated in the study, and compared changes in BCVA and other measures of visual function in the treated eye against baseline values.
    Arm type
    Experimental

    Investigational medicinal product name
    AAV2-REP1
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for injection
    Routes of administration
    Subretinal use
    Dosage and administration details
    AAV2-REP1 vector suspension (10e12 vector particles per mL) was supplied by Nightstar Therapeutics. Up to 0.1 mL of AAV2-REP1 vector suspension, corresponding to a dose of up to 10e11 vector particles, was administered to the treated eye by subretinal injection.

    Arm title
    Cohort 2
    Arm description
    Cohort 2 comprised the subset of subjects having symmetrical disease for whom selection of the treated eye was randomized, and compared changes in BCVA and other measures of visual function in the treated eye and the untreated contralateral (control) eye. Note that randomization was not used for assigning treatment (versus placebo/standard care as in randomized controlled trials), but solely for selection of the eye to be treated in these subjects for whom the progress of retinal degeneration was relatively symmetrical for both eyes, defined as a difference in BCVA of no more than one line of letters measured on an ETDRS chart, and no more than 25% difference in the area of surviving retinal pigment epithelium measured by fundus autofluorescence.
    Arm type
    Experimental

    Investigational medicinal product name
    AAV2-REP1
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Suspension for injection
    Routes of administration
    Subretinal use
    Dosage and administration details
    AAV2-REP1 vector suspension (10e12 vector particles per mL) was supplied by Nightstar Therapeutics. Up to 0.1 mL of AAV2-REP1 vector suspension, corresponding to a dose of up to 10e11 vector particles, was administered to the treated eye by subretinal injection.

    Number of subjects in period 1
    Cohort 1 Cohort 2
    Started
    30
    28
    Completed
    30
    28

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall study
    Reporting group description
    -

    Reporting group values
    Overall study Total
    Number of subjects
    30 30
    Age categorical
    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)
    0 0
        Adolescents (12-17 years)
    0 0
        Adults (18-64 years)
    30 30
        From 65-84 years
    0 0
        85 years and over
    0 0
    Gender categorical
    Units: Subjects
        Female
    0 0
        Male
    30 30

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    Cohort 1 comprised all subjects that participated in the study, and compared changes in BCVA and other measures of visual function in the treated eye against baseline values.

    Reporting group title
    Cohort 2
    Reporting group description
    Cohort 2 comprised the subset of subjects having symmetrical disease for whom selection of the treated eye was randomized, and compared changes in BCVA and other measures of visual function in the treated eye and the untreated contralateral (control) eye. Note that randomization was not used for assigning treatment (versus placebo/standard care as in randomized controlled trials), but solely for selection of the eye to be treated in these subjects for whom the progress of retinal degeneration was relatively symmetrical for both eyes, defined as a difference in BCVA of no more than one line of letters measured on an ETDRS chart, and no more than 25% difference in the area of surviving retinal pigment epithelium measured by fundus autofluorescence.

    Primary: BCVA change from baseline in treated eye

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    End point title
    BCVA change from baseline in treated eye [1]
    End point description
    Change from baseline in BCVA (measured as change in ETDRS letters) in the treated eye. [Statistical analysis: 95% Confidence Interval of the mean change based on t-test.]
    End point type
    Primary
    End point timeframe
    The 24-month assessment period following treatment.
    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: Statistical analysis is provided in the attached PDF file "BCVA ETDRS Score Change from Baseline", with additional information provided in the attached PDF file "BCVA ETDRS Score Change from Baseline by Surgery site".
    End point values
    Cohort 1 Cohort 2
    Number of subjects analysed
    30
    28
    Units: ETDRS letters
        arithmetic mean (confidence interval 95%)
    -2.6 (-8.3 to 3.0)
    -3.2 (-9.2 to 2.8)
    No statistical analyses for this end point

    Primary: BCVA change from baseline in treated eye compared with control eye

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    End point title
    BCVA change from baseline in treated eye compared with control eye [2] [3]
    End point description
    Change from baseline in BCVA (measured as change in ETDRS letters) in the treated eye compared with the control eye. [Statistical analysis: 95% Confidence Intervals of the least squares mean difference from the untreated eye. The ANCOVA (analysis of covariance) model includes the least squares mean difference in BCVA (treated eye versus control eye) as a factor and baseline BCVA as a covariate (p-value 0.059).]
    End point type
    Primary
    End point timeframe
    The 24-month assessment period following treatment.
    Notes
    [2] - 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: Statistical analysis is provided in the attached PDF file "BCVA ETDRS Score Change from Baseline, Between Eye Comparison".
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis is provided in the attached PDF file "BCVA ETDRS Score Change from Baseline, Between Eye Comparison".
    End point values
    Cohort 2
    Number of subjects analysed
    28
    Units: ETDRS letters
        least squares mean (confidence interval 95%)
    -5.9 (-12 to 0.2)
    No statistical analyses for this end point

    Secondary: Safety

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    End point title
    Safety [4]
    End point description
    Serious Adverse Events (SAEs) related to the study drug (AAV2-REP1 vector) and/or the surgical procedure (vitrectomy, retinal detachment and subretinal injection of the vector suspension).
    End point type
    Secondary
    End point timeframe
    The 24-month assessment period following treatment.
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Serious adverse events are detailed in the "Adverse events" section of the report.
    End point values
    Cohort 1
    Number of subjects analysed
    30
    Units: SAEs related to the study drug/procedure
    6
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    The recording and follow-up of all adverse events was carried out until the end of the study, corresponding to the date of the last subject's final follow-up visit at the end of the 2-year assessment period following treatment.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    22
    Reporting groups
    Reporting group title
    All subjects
    Reporting group description
    -

    Serious adverse events
    All subjects
    Total subjects affected by serious adverse events
         subjects affected / exposed
    6 / 30 (20.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Surgical and medical procedures
    Cholelithotomy
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Blindness unilateral
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Visual acuity reduced
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Visual impairment
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    3 / 3
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Cholecystitis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    All subjects
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    30 / 30 (100.00%)
    Investigations
    Inflammatory marker increased
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Colonoscopy
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Post procedural inflammation
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences all number
    4
    Corneal abrasion
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Suture related complication
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Vascular disorders
    Syncope
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Immune system disorders
    Drug hypersensitivity
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Seasonal allergy
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Motion sickness
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Eye disorders
    Hypotony of eye
         subjects affected / exposed
    11 / 30 (36.67%)
         occurrences all number
    11
    Conjunctival haemorrhage
         subjects affected / exposed
    8 / 30 (26.67%)
         occurrences all number
    8
    Dry eye
         subjects affected / exposed
    7 / 30 (23.33%)
         occurrences all number
    7
    Ocular hypertension
         subjects affected / exposed
    7 / 30 (23.33%)
         occurrences all number
    9
    Cystoid macular oedema
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences all number
    4
    Iridocyclitis
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences all number
    4
    Uveitis
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    4
    Vitreous floaters
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    3
    Diplopia
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Photopsia
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Vitritis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    2
    Cataract
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Chorioretinal folds
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Chromatopsia
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Conjunctival cyst
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Conjunctival granuloma
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Corneal epithelium defect
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Corneal oedema
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Eye irritation
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Eye oedema
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Metamorphopsia
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Night blindness
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Keratitis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Ulcerative keratitis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Conjunctivitis
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Dyschromatopsia
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Choroiditis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Visual field defect
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Optic disc disorder
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Retinal depigmentation
         subjects affected / exposed
    6 / 30 (20.00%)
         occurrences all number
    6
    Chalazion
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Punctate keratitis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Retinal haemorrhage
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Eyelid rash
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Acne
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    3
    Dermatitis atopic
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Blepharitis allergic
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Myalgia
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Infections and infestations
    Conjunctivitis viral
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Eyelid folliculitis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Skin infection
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Cellulitis
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Laryngitis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Nasopharyngitis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    2
    Influenza
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Herpes zoster
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Gastroenteritis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Tooth infection
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1
    Infectious mononucleosis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    15 Feb 2016
    Version number of the Investigational Medicinal Product Dossier (IMPD) updated from 1.0 to 2.0 following approval by the Medicines and Healthcare products Regulatory Agency (MHRA) of requested corrections.
    12 May 2016
    Version number of the IMPD updated from 2.0 to 3.0 following the addition of supplementary vector stability/potency data required for MHRA approval of a 12-month extension of the AAV2-REP1 vector shelf life to 29 June 2017.
    14 Dec 2016
    Version numbers of the Protocol, Participant Information Sheet (PIS) and Informed Consent Form (ICF) updated from 2.0 to 3.0 following MHRA and research ethics committee approval of: • Change of the Principal Investigator at the Moorfields Eye Hospital site. • Amendments and clarifications to the schedule of assessments, and other clarifications in regard to data management for the study.
    06 Feb 2017
    Version numbers of the Protocol, PIS and ICF updated from 3.0 to 4.0 following MHRA and research ethics committee approval of amendments to the schedule of assessments (vital signs, immunology, fundus photography and refraction/BVCA).
    05 Apr 2017
    Version number of the Protocol updated from 4.0 to 5.0 following MHRA and research ethics committee approval of an amendment to the inclusion criterion specifying that candidates must have BCVA better than or equal to 6/60 (20/200) in the treated eye.
    25 May 2017
    Version number of the IMPD updated from 3.0 to 4.0 following the addition of supplementary vector stability/potency data required for MHRA approval of a 12-month extension of the AAV2-REP1 vector shelf life to 29 June 2018.
    10 Nov 2017
    Version number of the Protocol updated from 5.0 to 6.0 and the version number of the PIS updated from 4.0 to 5.0 following MHRA and research ethics approval of: • Extension of the oral prednisolone course. • Amendment permitting a total volume of no less than 0.2 mL of the AAV2-REP1 vector to be loaded into the injection system. • Amendment to the Conflict of Interest Statement.
    12 Sep 2018
    Version number of the Investigator Brochure (IB) updated from 1.0 to 2.0 following MHRA and research ethics committee approval of the inclusion of safety data from other studies (NCT01461213, NCT02077361). Version number of the IMPD updated from 4.0 to 5.0 following the addition of supplementary vector stability/potency data required for MHRA approval of a 12-month extension of the AAV2-REP1 vector shelf life to 29 June 2019.
    29 Jul 2020
    Version number of the IB updated from 2.0 to 3.0 following MHRA and research ethics committee approval of the inclusion of clinical data from preceding/parallel studies (NCT01461213, NCT02077361, NCT02553135, NCT02671539), this study (NCT02407678) and studies sponsored by Nightstar Therapeutics (NCT03496012, NCT03507686). The Reference Safety Information now includes “visual acuity reduced” as an expected risk of treatment. Version number of the Protocol updated from 6.0 to 7.0 following following MHRA and research ethics committee approval of updates to the Safety Reporting section to include parameters for determining the clinical significance of changes in BCVA.

    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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