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 were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43865   clinical trials with a EudraCT protocol, of which   7286   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).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    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:
    A Phase 2, randomized, double-blind placebo-controlled study to test the efficacy and safety of KPL-301 in giant cell arteritis

    Summary
    EudraCT number
    		 2018-001003-36
    Trial protocol
    		 EE   GB   DE   ES   NL   BE   SI   IT   HR  
    Global end of trial date
    25 Nov 2020

    Results information
    Results version number
    		 v2(current)
    This version publication date
    22 Jan 2022
    First version publication date
    13 Dec 2021
    Other versions
    		 v1
    Version creation reason
    • Correction of full data set
    Correction of the sponsor contact details and note about statistical analysis.

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    KPL-301-C001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03827018
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Kiniksa Pharmaceuticals, Ltd.
    Sponsor organisation address
    Clarendon House 2 Church Street, Hamilton, Bermuda, HM 11
    Public contact
    Kiniksa Medical Information Group, Kiniksa Pharmaceuticals, Ltd. (Hamilton, Bermuda), 1 7814319100, medinfo@kiniksa.com
    Scientific contact
    Kiniksa Medical Information Group, Kiniksa Pharmaceuticals, Ltd. (Hamilton, Bermuda), 1 7814319100, medinfo@kiniksa.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
    30 Aug 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    25 Nov 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Nov 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the trial was to evaluate the efficacy of KPL-301 versus placebo, coadministered with a 26-week steroid taper, for maintaining sustained remission for 26 weeks in subjects with new-onset or relapsing/refractory giant cell arteritis (GCA). Secondary objectives of the trial were: a) To evaluate the effect of KPL-301 vs placebo on cumulative corticosteroid dose. b) To evaluate the effect of KPL-301 vs placebo on health-related quality of life (HRQoL). c) To evaluate the safety and tolerability of KPL-301. d) To evaluate the pharmacokinetics (PK) of KPL-301.
    Protection of trial subjects
    This study was conducted in accordance with the protocol and with the consensus ethical principles derived from international guidelines including the Declaration of Helsinki and Council for International Organizations of Medical Sciences International Ethical Guidelines, and applicable International Council for/Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines, and applicable country and local laws and regulations. Subjects were informed that their participation was voluntary. Subjects or their legally authorized representative were required to sign a statement of informed consent that met the requirements of 21 Code of Federal Regulations 50, local regulations, ICH guidelines, Health Insurance Portability and Accountability Act requirements, where applicable, and the IRB/IEC or study center.
    Background therapy
    		 All subjects received an unblinded 26-week oral prednisone taper according to a standardized tapering protocol. The oral steroid taper was self-administered by study subjects on a daily basis.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    20 Sep 2018
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety
    Long term follow-up duration
    		 3 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 3
    Country: Number of subjects enrolled
    Slovenia: 2
    Country: Number of subjects enrolled
    Spain: 8
    Country: Number of subjects enrolled
    United Kingdom: 8
    Country: Number of subjects enrolled
    Belgium: 6
    Country: Number of subjects enrolled
    Estonia: 8
    Country: Number of subjects enrolled
    Germany: 7
    Country: Number of subjects enrolled
    Ireland: 2
    Country: Number of subjects enrolled
    Italy: 8
    Country: Number of subjects enrolled
    Australia: 6
    Country: Number of subjects enrolled
    New Zealand: 3
    Country: Number of subjects enrolled
    Serbia: 7
    Country: Number of subjects enrolled
    United States: 2
    Worldwide total number of subjects
    70
    EEA total number of subjects
    44
    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)
    18
    From 65 to 84 years
    51
    85 years and over
    1

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Subjects who met all of the inclusion criteria and none of the exclusion criteria were enrolled into the study. Eligible subjects entered the double-blind treatment period after randomization 3:2 to blinded treatment with KPL-301 150 mg or placebo administered subcutaneously (SC) every other week (every 2 weeks).

    Pre-assignment
    Screening details
    		 The study consisted of a screening period up to 6 weeks. Potential subjects were screened for meeting study-specified diagnostic criteria for GCA. Prior to first administration of KPL-301 or placebo, all subjects were required to have achieved remission.

    Period 1
    Period 1 title
    Base Treatment Period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor
    Blinding implementation details
    This was a double-blind study in which KPL-301 and placebo were identical in appearance/viscosity. Neither the subject nor any of the investigator or sponsor staff who were involved in the treatment or clinical evaluation of the subjects were aware of the treatment received.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 KPL-301 150mg
    Arm description
    		 Treatment with KPL-301 150 mg administered subcutaneously (SC) every other week (every 2 weeks). KPL-301 was coadministered with a 26-week corticosteroid taper.
    Arm type
    		 Experimental

    Investigational medicinal product name
    KPL-301
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects randomized to KPL-301 received 150 mg every other week by SC injection coadministered with a 26-week steroid taper.

    Arm title
    		 Placebo
    Arm description
    		 Treatment with placebo administered subcutaneously (SC) every other week (every 2 weeks). Placebo was coadministered with a 26-week corticosteroid taper.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects randomized to Placebo received sterile, clear, colorless, free from visible particles liquid product identical in appearance/viscosity to the KPL-301 every other week by SC injection coadministered with a 26-week steroid taper.

    Number of subjects in period 1
    		KPL-301 150mg Placebo
    Started
    42
    28
    Completed
    31
    12
    Not completed
    11
    16
         Consent withdrawn by subject
    2
    2
         Adverse event, non-fatal
    1
    1
         Lack of efficacy
    8
    12
         Protocol deviation
    -
    1

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    KPL-301 150mg
    Reporting group description
    		 Treatment with KPL-301 150 mg administered subcutaneously (SC) every other week (every 2 weeks). KPL-301 was coadministered with a 26-week corticosteroid taper.

    Reporting group title
    Placebo
    Reporting group description
    		 Treatment with placebo administered subcutaneously (SC) every other week (every 2 weeks). Placebo was coadministered with a 26-week corticosteroid taper.

    Reporting group values
    		 KPL-301 150mg Placebo Total
    Number of subjects
    		 42 28 70
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 9 9 18
        From 65-84 years
    		 33 18 51
        85 years and over
    		 0 1 1
    Age continuous
    Units: years
        median (full range (min-max))
    		 69.5 (52 to 84) 72 (55 to 85) -
    Gender categorical
    Units: Subjects
        Female
    		 32 18 50
        Male
    		 10 10 20
    Subject analysis sets

    Subject analysis set title
    mITT Analysis Set
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    All randomized subjects who received at least 1 dose of KPL-301 or placebo and at least had 1 assessment in the Double-blind Treatment period. Efficacy analyses were based on the Modified intent-to-treat (mITT) analysis set. All analyses based on mITT were based on each subject’s randomized treatment assignment.

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All randomized subjects who took at least 1 dose of KPL-301 or placebo. Safety analyses were based on the medication that was actually administered to each subject.

    Subject analysis set title
    Per Protocol Analysis Set
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    All mITT subjects without important protocol deviations deemed to impact efficacy or ethical conduct.

    Subject analysis set title
    PK Analysis Set
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All subjects who received at least 1 dose of KPL-301 and had sufficient KPL-301 concentration data for calculation of KPL-301 PK parameters were included in the PK analysis set.

    Subject analysis sets values
    		 mITT Analysis Set Safety Analysis Set Per Protocol Analysis Set PK Analysis Set
    Number of subjects
    70
    70
    67
    70
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    18
    18
    18
        From 65-84 years
    51
    51
    51
        85 years and over
    1
    1
    1
    Age continuous
    Units: years
        median (full range (min-max))
    70 (52 to 85)
    70 (52 to 85)
    70 (52 to 85)
    Gender categorical
    Units: Subjects
        Female
    50
    50
    50
        Male
    20
    20
    20

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    KPL-301 150mg
    Reporting group description
    		 Treatment with KPL-301 150 mg administered subcutaneously (SC) every other week (every 2 weeks). KPL-301 was coadministered with a 26-week corticosteroid taper.

    Reporting group title
    Placebo
    Reporting group description
    		 Treatment with placebo administered subcutaneously (SC) every other week (every 2 weeks). Placebo was coadministered with a 26-week corticosteroid taper.

    Subject analysis set title
    mITT Analysis Set
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    All randomized subjects who received at least 1 dose of KPL-301 or placebo and at least had 1 assessment in the Double-blind Treatment period. Efficacy analyses were based on the Modified intent-to-treat (mITT) analysis set. All analyses based on mITT were based on each subject’s randomized treatment assignment.

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All randomized subjects who took at least 1 dose of KPL-301 or placebo. Safety analyses were based on the medication that was actually administered to each subject.

    Subject analysis set title
    Per Protocol Analysis Set
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    All mITT subjects without important protocol deviations deemed to impact efficacy or ethical conduct.

    Subject analysis set title
    PK Analysis Set
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All subjects who received at least 1 dose of KPL-301 and had sufficient KPL-301 concentration data for calculation of KPL-301 PK parameters were included in the PK analysis set.

    Primary: Time to Flare by Week 26

    		 Close Top of page
    End point title
    		 Time to Flare by Week 26 [1] [2]
    End point description
    End point type
    Primary
    End point timeframe
    By Week 26
    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: Descriptive statistics were included for this primary endpoint. Kaplan-Meier method used to estimate the survival functions for each treatment arm.
    [2] - 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: Placebo hasn't been included in this endpoint as there is no data for Time to Flare - Hazard Ratio.
    End point values
    		 KPL-301 150mg
    Number of subjects analysed
    42
    Units: weeks/percent
    number (confidence interval 95%)
        Time to Flare - Hazard Ratio (KPL-301 vs Placebo)
    0.38 (0.15 to 0.92)
    No statistical analyses for this end point

    Secondary: Sustained Remission at Week 26

    		 Close Top of page
    End point title
    		 Sustained Remission at Week 26
    End point description
    End point type
    Secondary
    End point timeframe
    At Week 26
    End point values
    		 KPL-301 150mg Placebo
    Number of subjects analysed
    42
    28
    Units: percent
    number (confidence interval 95%)
        Sustained Remission at Week 26, %
    83.2 (67.9 to 91.6)
    49.9 (29.6 to 67.3)
    No statistical analyses for this end point

    Secondary: Time to Elevated ESR by Week 26

    		 Close Top of page
    End point title
    		 Time to Elevated ESR by Week 26
    End point description
    End point type
    Secondary
    End point timeframe
    By Week 26
    End point values
    		 KPL-301 150mg Placebo
    Number of subjects analysed
    42
    28
    Units: Weeks
    number (not applicable)
        Time to Elevated ESR by Week 26, median
    26.1
    12.1
    No statistical analyses for this end point

    Secondary: Time to Elevated CRP by Week 26

    		 Close Top of page
    End point title
    		 Time to Elevated CRP by Week 26 [3]
    End point description
    End point type
    Secondary
    End point timeframe
    By Week 26
    Notes
    [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: Placebo hasn't been included in this endpoint as there is no data for Time to Flare - Hazard Ratio.
    End point values
    		 KPL-301 150mg
    Number of subjects analysed
    42
    Units: Weeks/percent
    number (confidence interval 95%)
        Hazard Ratio (KPL-301 vs Placebo)
    0.43 (0.19 to 0.98)
    No statistical analyses for this end point

    Secondary: Time to Signs/Symptoms of GCA or New/Worsening Vasculitis by Imaging by Week 26

    		 Close Top of page
    End point title
    		 Time to Signs/Symptoms of GCA or New/Worsening Vasculitis by Imaging by Week 26 [4]
    End point description
    Time to Symptoms of GCA, New/Worsening Vasculitis
    End point type
    Secondary
    End point timeframe
    By Week 26
    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: Placebo hasn't been included in this endpoint as there is no data for Hazard Ratio (KPL-301 vs Placebo).
    End point values
    		 KPL-301 150mg
    Number of subjects analysed
    42
    Units: Weeks/percent
    number (confidence interval 95%)
        Hazard Ratio (KPL-301 vs Placebo)
    0.49 (0.22 to 1.06)
    No statistical analyses for this end point

    Secondary: Cumulative Steroid Dose at Week 26

    		 Close Top of page
    End point title
    		 Cumulative Steroid Dose at Week 26
    End point description
    End point type
    Secondary
    End point timeframe
    At Week 26
    End point values
    		 KPL-301 150mg Placebo
    Number of subjects analysed
    42
    28
    Units: Dose
    least squares mean (standard error)
        Cumulative Steroid Dose
    2075.11 ± 109.598
    2401.56 ± 134.691
    No statistical analyses for this end point

    Secondary: Percentage of Subjects Completing Corticosteroid Taper and with Normal ESR by Week 26

    		 Close Top of page
    End point title
    		 Percentage of Subjects Completing Corticosteroid Taper and with Normal ESR by Week 26
    End point description
    End point type
    Secondary
    End point timeframe
    By Week 26
    End point values
    		 KPL-301 150mg Placebo
    Number of subjects analysed
    42
    28
    Units: percent
    number (not applicable)
        Subjects Completing Corticosteriod Taper, %
    45.2
    14.3
    No statistical analyses for this end point

    Secondary: Percentage of Subjects Completing Corticosteroid Taper and with Normal CRP by Week 26

    		 Close Top of page
    End point title
    		 Percentage of Subjects Completing Corticosteroid Taper and with Normal CRP by Week 26
    End point description
    End point type
    Secondary
    End point timeframe
    By Week 26
    End point values
    		 KPL-301 150mg Placebo
    Number of subjects analysed
    42
    28
    Units: percent
    number (not applicable)
        Subjects Completing Corticosteroid Taper, %
    23.8
    14.3
    No statistical analyses for this end point

    Secondary: Percentage of Subjects Completing Corticosteroid Taper and with No Signs/Symptoms of GCA by Week 26

    		 Close Top of page
    End point title
    		 Percentage of Subjects Completing Corticosteroid Taper and with No Signs/Symptoms of GCA by Week 26
    End point description
    End point type
    Secondary
    End point timeframe
    By Week 26
    End point values
    		 KPL-301 150mg Placebo
    Number of subjects analysed
    42
    28
    Units: percent
    number (not applicable)
        Subjects Completing Corticosteroid Taper, %
    71.4
    32.1
    No statistical analyses for this end point

    Secondary: Cumulative Steroid Dose at the end of the Washout Safety Follow-up Period

    		 Close Top of page
    End point title
    		 Cumulative Steroid Dose at the end of the Washout Safety Follow-up Period
    End point description
    End point type
    Secondary
    End point timeframe
    At the end of the Washout Safety Follow-up Period
    End point values
    		 KPL-301 150mg Placebo
    Number of subjects analysed
    42
    28
    Units: Dose
    least squares mean (standard error)
        Cumulative Steroid Dose
    2464.81 ± 168.955
    2845.63 ± 207.638
    No statistical analyses for this end point

    Secondary: Sustained Remission at week 26 (Independent Adjudication)

    		 Close Top of page
    End point title
    		 Sustained Remission at week 26 (Independent Adjudication)
    End point description
    End point type
    Secondary
    End point timeframe
    At Week 26
    End point values
    		 KPL-301 150mg Placebo
    Number of subjects analysed
    42
    28
    Units: percent
    number (confidence interval 95%)
        Sustained Remission at Week 26 (%)
    83.2 (67.9 to 91.6)
    49.9 (29.6 to 67.3)
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were monitored from Day 1 of treatment till Week 26 and during the following 12-week safety follow-up period.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    21.0
    Reporting groups
    Reporting group title
    KPL-301 150mg
    Reporting group description
    		 Treatment with KPL-301 150 mg administered subcutaneously (SC) every other week (every 2 weeks). KPL-301 was coadministered with a 26-week corticosteroid taper.

    Reporting group title
    Placebo
    Reporting group description
    		 Treatment with placebo administered subcutaneously (SC) every other week (every 2 weeks). Placebo was coadministered with a 26-week corticosteroid taper.

    Serious adverse events
    		 KPL-301 150mg Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 42 (4.76%)
    3 / 28 (10.71%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Congenital, familial and genetic disorders
    Hypertrophic cardiomyopathy
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 28 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Dementia
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 28 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Oedema peripheral
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 28 (3.57%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Gastrointestinal haemorrhage
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 28 (3.57%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary fibrosis
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 28 (3.57%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 KPL-301 150mg Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    33 / 42 (78.57%)
    25 / 28 (89.29%)
    Investigations
    Carbon monoxide diffusing capacity decreased
         subjects affected / exposed
    4 / 42 (9.52%)
    1 / 28 (3.57%)
         occurrences all number
    4
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    2 / 42 (4.76%)
    5 / 28 (17.86%)
         occurrences all number
    2
    5
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 42 (2.38%)
    4 / 28 (14.29%)
         occurrences all number
    1
    4
    Nervous system disorders
    Headache
         subjects affected / exposed
    6 / 42 (14.29%)
    7 / 28 (25.00%)
         occurrences all number
    6
    7
    Hypoaesthesia
         subjects affected / exposed
    0 / 42 (0.00%)
    2 / 28 (7.14%)
         occurrences all number
    0
    2
    General disorders and administration site conditions
    Oedema peripheral
         subjects affected / exposed
    2 / 42 (4.76%)
    2 / 28 (7.14%)
         occurrences all number
    2
    2
    Pyrexia
         subjects affected / exposed
    0 / 42 (0.00%)
    2 / 28 (7.14%)
         occurrences all number
    0
    2
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    3 / 42 (7.14%)
    0 / 28 (0.00%)
         occurrences all number
    3
    0
    Vomiting
         subjects affected / exposed
    1 / 42 (2.38%)
    2 / 28 (7.14%)
         occurrences all number
    1
    2
    Diarrhoea
         subjects affected / exposed
    0 / 42 (0.00%)
    3 / 28 (10.71%)
         occurrences all number
    0
    3
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    0 / 42 (0.00%)
    2 / 28 (7.14%)
         occurrences all number
    0
    2
    Psychiatric disorders
    Sleep disorder
         subjects affected / exposed
    0 / 42 (0.00%)
    2 / 28 (7.14%)
         occurrences all number
    0
    2
    Musculoskeletal and connective tissue disorders
    Neck pain
         subjects affected / exposed
    4 / 42 (9.52%)
    2 / 28 (7.14%)
         occurrences all number
    4
    2
    Back pain
         subjects affected / exposed
    3 / 42 (7.14%)
    3 / 28 (10.71%)
         occurrences all number
    3
    3
    Muscle spasms
         subjects affected / exposed
    3 / 42 (7.14%)
    3 / 28 (10.71%)
         occurrences all number
    3
    3
    Arthralgia
         subjects affected / exposed
    2 / 42 (4.76%)
    4 / 28 (14.29%)
         occurrences all number
    2
    4
    Osteoarthritis
         subjects affected / exposed
    1 / 42 (2.38%)
    2 / 28 (7.14%)
         occurrences all number
    1
    2
    Tendonitis
         subjects affected / exposed
    1 / 42 (2.38%)
    2 / 28 (7.14%)
         occurrences all number
    1
    2
    Musculoskeletal pain
         subjects affected / exposed
    0 / 42 (0.00%)
    2 / 28 (7.14%)
         occurrences all number
    0
    2
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    5 / 42 (11.90%)
    3 / 28 (10.71%)
         occurrences all number
    5
    3
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 42 (7.14%)
    2 / 28 (7.14%)
         occurrences all number
    3
    2
    Rhinitis
         subjects affected / exposed
    1 / 42 (2.38%)
    2 / 28 (7.14%)
         occurrences all number
    1
    2
    Oral herpes
         subjects affected / exposed
    0 / 42 (0.00%)
    2 / 28 (7.14%)
         occurrences all number
    0
    2
    Respiratory tract infection
         subjects affected / exposed
    0 / 42 (0.00%)
    2 / 28 (7.14%)
         occurrences all number
    0
    2
    Metabolism and nutrition disorders
    Diabetes mellitus
         subjects affected / exposed
    2 / 42 (4.76%)
    2 / 28 (7.14%)
         occurrences all number
    2
    2

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    15 Oct 2018
    Rationale for Amendment – Protocol Amendment v2.0 was completed to clarify aspects of protocol version 1.0 as well as to incorporate feedback from global Regulatory and Ethical bodies. Substantial changes to this version include: • Limiting the double-blind period to 26-weeks total, removing the extended exposure for patients who enroll earlier in the study. • Removal of the Open-Label Extension – Per Regulatory feedback, open-label KPL-301 should only be provided to patients after proof of efficacy has been established • Addition of 12-week Washout Safety Follow-up period to ensure patients have continued access to open-label study supplied prednisone and are adequately followed and weaned back to Standard of Care • Updated inclusion criteria regarding pregnancy and contraception methods to ensure highly effective methods of contraception are reflected • Provided clarification on sample size and event calculations • Addition of Serum Pregnancy test during screening, additional urine pregnancy tests, safety labs, and anti-KPL-301 antibody testing during the study. • Addition of Definition of SUSAR and defined Study Termination criteria
    30 Nov 2018
    Rationale for Amendment – Protocol Amendment v3.0 was completed to clarify aspects of protocol version 2.0 as well as to incorporate feedback from global Regulatory bodies. Substantial changes to this version include: • Discontinuation of study drug (KPL-301 or placebo) in the event of a GCA Flare • Additional PK samples added to Schedule of Activities • Clarification of individual patient and study stopping criteria • Correction of the US Safety contact number
    30 Mar 2020
    Rationale for Amendment – Protocol Amendment v4.0 was completed to clarify aspects of protocol version 3.0 as well as to incorporate feedback from global Regulatory bodies. Substantial changes to this version include: • Updated secondary endpoints and analysis; and inserted study endpoints into the Synopsis Objective section. • The planned number of subjects to be enrolled in the study was updated from approximately 60 to approximately 70 subjects (42 subjects planned to be assigned to the KPL-301 arm and 28 subjects planned to be assigned to the placebo arm) to align with the updated sample size estimation. • Added clarification that SAEs will be recorded in the eCRF within 24 hours of discovery.

    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
    For support, Contact us.
    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.

    European Medicines Agency © 1995-Sat Apr 27 07:17:34 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA