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   38202   clinical trials with a EudraCT protocol, of which   6274   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:
    International Open-Label Extension of the Phase 3 Study CL-503012 with KIACTA™ in Patients with AA Amyloidosis

    Summary
    EudraCT number
    2013-004150-16
    Trial protocol
    LT   GB   PL  
    Global end of trial date
    12 Sep 2016

    Results information
    Results version number
    v1(current)
    This version publication date
    12 Nov 2017
    First version publication date
    12 Nov 2017
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    CL-503015
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    A.T. Development Switzerland SARL
    Sponsor organisation address
    Rue Saint-Perre 2 , Lausanne Vaud, Switzerland, 1003
    Public contact
    Patrick C O'Connor Ph.D, FRCP , Auven Therapeutics, 954 903 0492, Patrick.OConnor@auventx.com
    Scientific contact
    Patrick C O'Connor Ph.D, FRCP , Auven Therapeutics, 954 903 0492, Patrick.OConnor@auventx.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
    19 Dec 2016
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Sep 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this open-label extension (OLE) study is to provide access to Kiacta (eprodisate disodium) for those patients who have completed the pivotal, randomized, placebo-controlled Phase 3 Study CL-503012.
    Protection of trial subjects
    Institutions, investigators, and contract research organizations, etc., associated with this study have abided by all requirements applicable to the use and disclosure of patients’ protected health information (such as the requirements provided for under the Health Insurance Portability and Accountability Act in the United States, the European Union Directive on Data Protection, the Personal Information Protection and Electronic Document Act in Canada and in any other similar regulations or legislation). The study was conducted according to the International Council for Harmonisation harmonised tripartite guideline E6(R1): Good Clinical Practice.
    Background therapy
    There is no standard therapy allowed per protocol (this is an orphan medicine).
    Evidence for comparator
    -
    Actual start date of recruitment
    17 Dec 2014
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Tunisia: 3
    Country: Number of subjects enrolled
    Ukraine: 4
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    Lithuania: 1
    Country: Number of subjects enrolled
    Peru: 9
    Country: Number of subjects enrolled
    Poland: 16
    Country: Number of subjects enrolled
    Russian Federation: 16
    Worldwide total number of subjects
    52
    EEA total number of subjects
    20
    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)
    40
    From 65 to 84 years
    12
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    Patients completing Study CL-503012 who fulfilled the selection criteria were offered the opportunity to participate in Study CL-503015. This study was conducted at 11 study centers in 7 countries.

    Pre-assignment
    Screening details
    Male or nonpregnant females of at least 18 years of age who completed Study CL-503012 and had undergone all study assessments that could affect the primary endpoint of Study CL-503012.

    Period 1
    Period 1 title
    Overall treatment period (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    Kiacta 400 mg BID, 800 mg BID, or 1200 mg BID
    Arm description
    This is an OLE of the Study CL-503012. Therefore, all patients will receive Kiacta 400 mg administered orally as 1 to 3 capsules BID starting at Baseline until EOS.
    Arm type
    Experimental

    Investigational medicinal product name
    Kiacta TM
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    This is an OLE of the Study CL-503012. Therefore, all patients will receive Kiacta 400 mg administered orally as 1 to 3 capsules BID starting at Baseline until EOS. The dose regimen will depend on the patient’s CrCl as calculated with the Cockcroft-Gault formula. At Baseline, the dose regimen will be based on CrCl of the last Study Cl-503012 visit. At subsequent visits the dose regimen will be adjusted based on CrCl. If the dose regimen requires adjustment during the treatment period due to a change in the CrCl range, the change in renal function will be confirmed, and the Investigator will notify the patient of the new dose regimen.

    Number of subjects in period 1
    Kiacta 400 mg BID, 800 mg BID, or 1200 mg BID
    Started
    52
    Completed
    43
    Not completed
    9
         treatment discontinuation
    9

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Overall treatment period
    Reporting group description
    -

    Reporting group values
    Overall treatment period Total
    Number of subjects
    52 52
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    40 40
        From 65-84 years
    12 12
    Gender categorical
    Units: Subjects
        Female
    33 33
        Male
    19 19

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Kiacta 400 mg BID, 800 mg BID, or 1200 mg BID
    Reporting group description
    This is an OLE of the Study CL-503012. Therefore, all patients will receive Kiacta 400 mg administered orally as 1 to 3 capsules BID starting at Baseline until EOS.

    Primary: Collection of data on Kiacta slowing renal function decline

    Close Top of page
    End point title
    Collection of data on Kiacta slowing renal function decline [1]
    End point description
    All patients were to receive the study drug for a maximum of 12 months unless Kiacta became commercially available in the specific country. Study CL-503015 was terminated prematurely on 21 Jun 2016 after the efficacy analysis of Study CL-503012 showed that Kiacta did not meet the primary efficacy endpoint in slowing renal function decline.
    End point type
    Primary
    End point timeframe
    Patients completing Study CL-503012 and fulfilling selection criteria will be offered the opportunity to participate in this OLE study for a maximum 12 months in those countries where the compassionate use program is not applicable.
    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: No statistical analyses were performed. No sample size target was identified. This study planned to include all patients completing Study CL-503012 who fulfilled all selection criteria and who provided their written consent to participate. Data were collected and listings were produced for demography, drug dosage, AEs, clinical laboratory parameters, past and concurrent medical conditions, and concomitant medications. Study CL-503015 was terminated prematurely on 21 Jun 2016.
    End point values
    Kiacta 400 mg BID, 800 mg BID, or 1200 mg BID
    Number of subjects analysed
    52
    Units: not applicable
    0
    No statistical analyses for this end point

    Adverse events

    Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    All patients were to receive Kiacta for a maximum of 12 months unless Kiacta became commercially available in the specific country.
    Adverse event reporting additional description
    Safety was assessed by the incidence of AEs and SAEs. Adverse events included any clinically significant change or new clinically significant occurrence in laboratory tests, and vital signs when compared with Baseline status.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    Kiacta 400 mg BID, 800 mg BID, or 1200 mg BID
    Reporting group description
    This is an OLE of the Study CL-503012. Therefore, all patients will receive Kiacta 400 mg administered orally as 1 to 3 capsules BID starting at Baseline until EOS.

    Serious adverse events
    Kiacta 400 mg BID, 800 mg BID, or 1200 mg BID
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 52 (7.69%)
         number of deaths (all causes)
    1
         number of deaths resulting from adverse events
    0
    Cardiac disorders
    congestive heart failure
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Heart failure
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Meningeal haemorrhage with severe hypertension
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Immune system disorders
    Insufficiency of multiorgan
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Respiratory, thoracic and mediastinal disorders
    infected bronchiectasis
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    pleural effusion worsening
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    Kiacta 400 mg BID, 800 mg BID, or 1200 mg BID
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 52 (13.46%)
    Injury, poisoning and procedural complications
    Calcaneal spur
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Fracture of the left humerus
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Cardiac disorders
    Mitral insufficiency
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Tachicardia
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Hypertension worsening
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Infected brochientasis
         subjects affected / exposed
    2 / 52 (3.85%)
         occurrences all number
    3
    Pleural effusion
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Psychiatric disorders
    Depresion
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Sleep disturbances
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Gastrointestinal disorders
    Acute diarrhea
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Diarrhoea
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Lichen ruber planus
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Restless legs syndrome
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Infections and infestations
    Herpes zoster of right buttock
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Inflammation of skin of left shank
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1
    Common cold
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    1

    More information

    Close Top of page

    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? No

    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
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2020 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA