Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   39787   clinical trials with a EudraCT protocol, of which   6529   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    An Open-Label, Single Arm, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

    Summary
    EudraCT number
    2017-000813-22
    Trial protocol
    BE  
    Global end of trial date
    02 Oct 2019

    Results information
    Results version number
    v1(current)
    This version publication date
    14 Mar 2021
    First version publication date
    14 Mar 2021
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    PQ-110-001
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03140969
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    ProQR Therapeutics
    Sponsor organisation address
    Zernikedreef 9 , Leiden, Netherlands, 2333CK
    Public contact
    Lola Tome, ProQR Therapeutics, +31 881667000, Ltome@proqr.com
    Scientific contact
    Lola Tome, ProQR Therapeutics, +31 881667000, Ltome@proqr.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
    21 Oct 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    02 Oct 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Oct 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection (IVT) in subjects with Leber Congenital Amaurosis type 10 (LCA10) due to the c.2991+1655A>G (p.Cys998X) mutation The secondary objectives of the study were to evaluate the: • Serum pharmacokinetics of QR-110 administered by IVT injection in subjects with LCA10 due to the c.2991+1655A>G (p.Cys998X) mutation • Efficacy of QR-110 administered by IVT injection in subjects with LCA10 due to the c.2991+1655A>G (p.Cys998X) mutation
    Protection of trial subjects
    The study was conducted in accordance with ethical principles that have their origin in the Declaration of Helsinki and are consistent with ICH GCP. QR-110 was administered via IVT injection to maximize exposure to study drug at the intended target tissue, the retina. QR-110 was administered in a unilateral manner to the subject’s worse eye, a common practice in the development of ophthalmic products. Following IVT injection of QR-110, subjects were monitored for increases in intraocular pressure (IOP) and signs of inflammation and endophthalmitis during the post-injection period. Subjects were also seen at 1 and 7 days after each injection. If sedation or anesthesia was performed during the administration of the IVT injection, general health monitoring was provided by the study anesthesiologist pre- and post-sedation/anesthesia, according to local institutional guidelines. A Data Monitoring Committee (DMC) was involved to recommend on dose escalation and provided safety oversight for the study.
    Background therapy
    There are currently no approved therapies for the treatment of LCA10.
    Evidence for comparator
    No placebo or sham injections were administered.
    Actual start date of recruitment
    15 May 2017
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Safety, Efficacy
    Long term follow-up duration
    24 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 9
    Country: Number of subjects enrolled
    Belgium: 2
    Worldwide total number of subjects
    11
    EEA total number of subjects
    2
    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)
    2
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    6
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    11 subjects were enrolled in this study. The study plan included up to 6 cohorts (3 adult and 3 pediatric). Each planned cohort was to have at least 2 subjects, and each dose level was to be tested in 1 adult cohort and 1 pediatric cohort.

    Pre-assignment
    Screening details
    Key inclusion criteria were: >= 6 years at Screening, a clinical diagnosis of LCA with a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A>G (p.Cys998X) mutation in the CEP290 gene and a best-corrected visual acuity equal or better than light perception and equal or worse than logMAR +0.6 in the treatment eye

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded
    Blinding implementation details
    No randomization or masking has been used in this study.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Cohort 1: 160/80 μg (loading dose/maintenance dose)
    Arm description
    One 160 μg loading dose, followed by up to three 80 μg maintenance doses.
    Arm type
    Experimental

    Investigational medicinal product name
    QR-110
    Investigational medicinal product code
    QR-110
    Other name
    sepofarsen
    Pharmaceutical forms
    Concentrate for solution for injection
    Routes of administration
    Intravitreal use
    Dosage and administration details
    QR-110 solution for injection, 10 mg/mL. Subjects receive QR-110 by IVT injection. QR-110 was to be administered once every 3 months for up to 4 doses per subject (ie, an initial loading dose at Day 1, followed by 3 maintenance doses at Month 3, 6 and 9).

    Arm title
    Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Arm description
    One 320 μg loading dose, followed by up to three 160 μg maintenance doses.
    Arm type
    Experimental

    Investigational medicinal product name
    QR-110
    Investigational medicinal product code
    QR-110
    Other name
    sepofarsen
    Pharmaceutical forms
    Concentrate for solution for injection
    Routes of administration
    Intravitreal use
    Dosage and administration details
    QR-110 solution for injection, 10 mg/mL. Subjects receive QR-110 by IVT injection. QR-110 was to be administered once every 3 months for up to 4 doses per subject (ie, an initial loading dose at Day 1, followed by 3 maintenance doses at Month 3, 6 and 9.). Following the observed incidence of cataracts and retinal events, the Sponsor decided in consultation with the DMC to stop administration of the 320/160 µg doses, and to prolong the dosing interval for subjects in the 160/80 µg dose group. While the protocol allowed testing of up to 3 dose levels, no dose escalation was performed to the 500 μg/270 μg dose based on efficacy observations; instead, additional subjects were allocated to the 160 μg/80 μg and 320 μg/160 μg dose cohorts.

    Number of subjects in period 1
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Started
    6
    5
    Completed
    6
    5

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1: 160/80 μg (loading dose/maintenance dose)
    Reporting group description
    One 160 μg loading dose, followed by up to three 80 μg maintenance doses.

    Reporting group title
    Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Reporting group description
    One 320 μg loading dose, followed by up to three 160 μg maintenance doses.

    Reporting group values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose) Total
    Number of subjects
    6 5 11
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    3 3 6
        Children (6-17 years)
    3 2 5
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    23.7 ± 15.06 19.4 ± 6.88 -
    Gender categorical
    Units: Subjects
        Female
    2 4 6
        Male
    4 1 5

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Cohort 1: 160/80 μg (loading dose/maintenance dose)
    Reporting group description
    One 160 μg loading dose, followed by up to three 80 μg maintenance doses.

    Reporting group title
    Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Reporting group description
    One 320 μg loading dose, followed by up to three 160 μg maintenance doses.

    Primary: Frequency of Ocular AEs in Treatment Eyes

    Close Top of page
    End point title
    Frequency of Ocular AEs in Treatment Eyes [1]
    End point description
    Frequency of all ocular treatment emergent adverse events (TEAEs) in the treatment eyes (ie, treated eyes)
    End point type
    Primary
    End point timeframe
    From baseline until End of Study (12 months)
    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: Incidence and severity of adverse events were summarized descriptively.
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    5
    Units: number of events
    15
    45
    No statistical analyses for this end point

    Primary: Severity of Ocular AEs

    Close Top of page
    End point title
    Severity of Ocular AEs [2]
    End point description
    Number of subjects with mild, moderate or severe ocular treatment emergent adverse events (TEAEs)
    End point type
    Primary
    End point timeframe
    From baseline until End of Study (12 months)
    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: Incidence and severity of adverse events were summarized descriptively.
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    5
    Units: nr of subjects
        mild
    3
    1
        moderate
    1
    1
        severe
    1
    3
    No statistical analyses for this end point

    Primary: Frequency of Ocular AEs in Contralateral Eyes (CE)

    Close Top of page
    End point title
    Frequency of Ocular AEs in Contralateral Eyes (CE) [3]
    End point description
    Frequency of all ocular treatment emergent adverse events (TEAEs) in contralateral eyes (ie, untreated eyes)
    End point type
    Primary
    End point timeframe
    From baseline to End of Study (Month 12)
    Notes
    [3] - 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: Incidence and severity of adverse events were summarized descriptively.
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    5
    Units: Number of events
    0
    1
    No statistical analyses for this end point

    Secondary: Frequency of non-ocular AEs

    Close Top of page
    End point title
    Frequency of non-ocular AEs
    End point description
    Frequency of all non-ocular Adverse Events (AEs)
    End point type
    Secondary
    End point timeframe
    From baseline until End of Study (12 months)
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    5
    Units: number of events
    21
    20
    No statistical analyses for this end point

    Secondary: Severity of non-ocular AEs

    Close Top of page
    End point title
    Severity of non-ocular AEs
    End point description
    Number of subjects with mild, moderate or severe non-ocular adverse events (AEs).
    End point type
    Secondary
    End point timeframe
    From baseline until End of Study (12 months)
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    5
    Units: nr of AEs
        mild
    5
    4
        moderate
    0
    1
        severe
    1
    0
    No statistical analyses for this end point

    Secondary: BCVA change from baseline in Treatment Eye (TE)

    Close Top of page
    End point title
    BCVA change from baseline in Treatment Eye (TE)
    End point description
    Change of BCVA value at Month 12 versus BCVA value at baseline in the treatment eye (= treated eye). Baseline defined as the average pre-treatment value (ie, the average of Screening and Day 1 [pre-dose] values). Smaller values are indicative of better status.
    End point type
    Secondary
    End point timeframe
    From baseline until End of study (12 months).
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    5
    Units: logMAR
        arithmetic mean (standard deviation)
    -0.93 ± 1.049
    -0.11 ± 0.155
    No statistical analyses for this end point

    Secondary: Red FST Change from Baseline in Treatment Eye (TE)

    Close Top of page
    End point title
    Red FST Change from Baseline in Treatment Eye (TE)
    End point description
    Change from Month 12 to baseline in retinal sensitivity in the treatment eye (= treated eye) based on red FST. Data from 1 subject is missing. Month 12 data for 1 subject was imputed using LOCF. Smaller values are indicative of better status.
    End point type
    Secondary
    End point timeframe
    From baseline until End of Study (Month 12)
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    4
    Units: log cd/m2
        arithmetic mean (standard deviation)
    -0.7 ± 0.33
    -1.3 ± 0.69
    No statistical analyses for this end point

    Secondary: Blue FST Change from Baseline in Treatment Eye (TE)

    Close Top of page
    End point title
    Blue FST Change from Baseline in Treatment Eye (TE)
    End point description
    Change from Month 12 to baseline in retinal sensitivity based on blue FST in the treatment eye (= treated eye). Data from 1 subject is missing. Month 12 data for 1 subject was imputed using LOCF. Smaller values are indicative of better status.
    End point type
    Secondary
    End point timeframe
    From Baseline until End of Study (Month 12)
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    4
    Units: log cd/m2
        arithmetic mean (standard deviation)
    -0.6 ± 0.76
    -1.0 ± 0.72
    No statistical analyses for this end point

    Secondary: Mobility Course Composite Score Change from Baseline in Treatment Eye (TE)

    Close Top of page
    End point title
    Mobility Course Composite Score Change from Baseline in Treatment Eye (TE)
    End point description
    Change from Month 12 to baseline in mobility course composite score in the treatment eye (ie, treated eye). The mobility course composite score represents the most difficult course and light level combination passed. Data from 1 subject is missing.
    End point type
    Secondary
    End point timeframe
    From baseline until End of Study (Month 12)
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    4
    Units: score
        arithmetic mean (standard deviation)
    4.00 ± 3.114
    0.25 ± 1.323
    No statistical analyses for this end point

    Secondary: Red FST Change from Baseline in Contralateral Eye (CE)

    Close Top of page
    End point title
    Red FST Change from Baseline in Contralateral Eye (CE)
    End point description
    Change from Month 12 to baseline in retinal sensitivity in the untreated contralateral eye, based on red FST. Data from 1 subject is missing. Month 12 data for 1 subject was imputed using LOCF. Smaller values are indicative of better status.
    End point type
    Secondary
    End point timeframe
    From baseline until End of Study (Month 12)
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    4
    Units: log cd/m2
        arithmetic mean (standard deviation)
    0.0 ± 0.42
    -0.5 ± 0.54
    No statistical analyses for this end point

    Secondary: BCVA change from baseline in Contralateral Eye (CE)

    Close Top of page
    End point title
    BCVA change from baseline in Contralateral Eye (CE)
    End point description
    Change of BCVA value at Month 12 versus BCVA value at baseline in the untreated contralateral eye. Baseline defined as the average pre-treatment value (ie, the average of Screening and Day 1 [pre-dose] values). Smaller values are indicative of better status.
    End point type
    Secondary
    End point timeframe
    From baseline to End of Study (12 months)
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    5
    Units: logMAR
        arithmetic mean (standard deviation)
    -0.22 ± 0.264
    0.008 ± 0.0856
    No statistical analyses for this end point

    Secondary: Blue FST Change from Baseline in Contralateral Eye (CE)

    Close Top of page
    End point title
    Blue FST Change from Baseline in Contralateral Eye (CE)
    End point description
    Change from Month 12 to baseline in retinal sensitivity in the untreated contralateral eye, based on blue FST. Data from 1 subject is missing. Month 12 data for 1 subject was imputed using LOCF. Smaller values are indicative of better status.
    End point type
    Secondary
    End point timeframe
    from baseline until End of Study (Month 12)
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    4
    Units: log cd/m2
        arithmetic mean (standard deviation)
    0.1 ± 0.39
    -0.1 ± 0.24
    No statistical analyses for this end point

    Secondary: Mobility Course Composite Score Change from Baseline in Contralateral Eye (CE)

    Close Top of page
    End point title
    Mobility Course Composite Score Change from Baseline in Contralateral Eye (CE)
    End point description
    Change from baseline to Month 12 in Mobility Course Composite Score in the contralateral eye (ie, untreated eyes). The mobility course composite score represents the most difficult course and light level combination passed. Data from 1 subject missing.
    End point type
    Secondary
    End point timeframe
    From baseline until End of Study (Month 12)
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6
    4
    Units: Score
        arithmetic mean (standard deviation)
    2.67 ± 2.714
    0.38 ± 0.750
    No statistical analyses for this end point

    Secondary: Serum pharmacokinetics

    Close Top of page
    End point title
    Serum pharmacokinetics
    End point description
    PK calculations were not performed, since all measurements indicated serum levels were below the LLOQ (1.02 ng/mL) following administration of up to 4 doses, except for one subject who had a QR-110 level of 1.53 ng/mL which is slightly above the LLOQ ; this occurred 3 hours after the first (loading) dose of 320 μg and was not observed at subsequent timepoints for PK sampling.
    End point type
    Secondary
    End point timeframe
    Samples for analysis of QR-110 serum concentration were taken pre-dose and 3 hours post-dose on Day 1 and at the end of the study in all subjects.
    End point values
    Cohort 1: 160/80 μg (loading dose/maintenance dose) Cohort 2: 320/160 μg (loading dose/maintenance dose)
    Number of subjects analysed
    6 [4]
    5 [5]
    Units: ng/mL
        arithmetic mean (standard deviation)
    0 ± 0
    0 ± 0
    Notes
    [4] - All levels were below LLOQ
    [5] - Only 1 subject showed levels above LLOQ (1.53 ng/mL)
    No statistical analyses for this end point

    Adverse events

    Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    (S)AEs were collected from screening, and classed as medical history, non-treatment emergent AE or AE depending on relation to study drug administration.
    Adverse event reporting additional description
    Ten of 11 subjects experienced a total of 61 AEs, of which 33 were treatment related. Reported ocular AEs were mostly mild in severity. The most common AEs were cataract-related (8 of 11 subjects), resulting in an SAE for 6 subjects. Three subjects had an AE indicative of retinal changes. AEs of special interest are depicted.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    20
    Reporting groups
    Reporting group title
    cohort 1: 160/80 μg
    Reporting group description
    Subjects received one 160 μg loading dose, followed by up to three 80 μg maintenance doses.

    Reporting group title
    cohort 2: 320/160 μg
    Reporting group description
    Subjects received one 320 μg loading dose, followed by up to three 160 μg maintenance doses. Following the observed incidence of cataracts and retinal events, the Sponsor decided in consultation with the DMC to stop administration of the 320/160 µg doses, and to prolong the dosing interval for subjects in the 160/80 µg dose group.

    Serious adverse events
    cohort 1: 160/80 μg cohort 2: 320/160 μg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 6 (33.33%)
    4 / 5 (80.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Eye disorders
    Cataract
    Additional description: All SAEs consisted of the PT of cataract (verbatim terms: cataract [4 subjects] and cataract surgery [2 subjects]); each case of cataract was assessed as serious due to lens replacement surgery being performed.
         subjects affected / exposed
    2 / 6 (33.33%)
    4 / 5 (80.00%)
         occurrences causally related to treatment / all
    2 / 2
    4 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    cohort 1: 160/80 μg cohort 2: 320/160 μg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 6 (50.00%)
    5 / 5 (100.00%)
    Eye disorders
    Cystoid macular oedema
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Retinal cyst
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Retinal degeneration
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 5 (40.00%)
         occurrences all number
    0
    2
    Cataract
    Additional description: For 8 subjects >=1 cataract-related AEs were reported reflecting different stages of cataract development. For 6 subjects the cataract resulted in an SAE due to the need for lens replacement surgery."
         subjects affected / exposed
    3 / 6 (50.00%)
    5 / 5 (100.00%)
         occurrences all number
    1
    1

    More information

    Close Top of page

    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    18 May 2017
    Revisions made to historic genotyping and sequencing requirements (non-substantial amendment)
    08 Nov 2017
    Revisions made to add the Cardiff Visual Ability Questionnaire for Children Clarification of Exclusion Criteria 7 through 9 regarding acceptable laboratory values at Screening Revisions to endpoints to enhance nature and quality of data derived from the study (non-substantial amendment)
    04 Jun 2018
    Revised BCVA for Inclusion Criteria (substantial amendment not submitted in Belgium)
    27 Jul 2018
    Revised efficacy assessments and subject disposition, demographics and baseline disease characteristics to indicate that the average of the measures represented a more accurate assessment of the baseline value, considering all collected values and stabilizing the baseline. Revised BCVA for inclusion criteria Allowed for interim analyses to support the design of subsequent QR-110 clinical studies. Revised Schedule of Events This substantial amendment was not submitted in Belgium
    02 Aug 2018
    Added exploratory endpoints that can be done at the investigator’s discretion at any protocol visit (non-substantial).

    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

    Online references

    http://www.ncbi.nlm.nih.gov/pubmed/30559420
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2021 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA