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    Clinical Trial Results:
    A Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group, Multicentre Study on Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763 in Subjects with Non-transfusion Dependent Beta-thalassaemia.

    Summary
    EudraCT number
    		 2019-002221-29
    Trial protocol
    		 GR   IT  
    Global end of trial date
    03 Nov 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    02 Nov 2022
    First version publication date
    02 Nov 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VIT-2763-THAL-201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04364269
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Vifor (International) Inc.
    Sponsor organisation address
    Rechenstrasse 37, St. Gallen, Switzerland, CH-9001
    Public contact
    VIT-2763-THAL-201 Clinical Study Team, Vifor Pharma, Inc., +41 58 852 90 74, VIT2763-THAL-201.clinicaldevelopment@viforpharma.com
    Scientific contact
    VIT-2763-THAL-201 Clinical Study Team, Vifor Pharma, Inc., +41 58 852 90 74, VIT2763-THAL-201.clinicaldevelopment@viforpharma.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
    11 Oct 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 Oct 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    03 Nov 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the safety and tolerability of VIT-2763 versus placebo in adults and adolescents subjects with non-transfusion dependent thalassemia (NTDT) over a 12-week treatment period.
    Protection of trial subjects
    The study was conducted in accordance with the principles of the Declaration of Helsinki including amendments in force up to and including the time the study was conducted. The study was conducted in compliance with the International Council for Harmonisation (ICH) E6 Guideline for Good Clinical Practice (GCP). Prior to the initiation of the study, the protocol, the subject information sheet, and the informed consent form (ICF) were reviewed and approved by Independent Ethics Committees (IECs) or Institutional Review Boards (IRBs), Ethics Committee (EC) operating in accordance with current regulations.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    11 Jun 2020
    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
    Lebanon: 3
    Country: Number of subjects enrolled
    Israel: 3
    Country: Number of subjects enrolled
    Thailand: 9
    Country: Number of subjects enrolled
    Greece: 7
    Country: Number of subjects enrolled
    Italy: 3
    Worldwide total number of subjects
    25
    EEA total number of subjects
    10
    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)
    25
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 From a total of 35 screened participants, 25 were randomized in the study. A total of 10 participants were not randomised, among which 2 participants withdrew their consent, and 8 participants did not meet the inclusion/ exclusion criteria.

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 VIT-2763 QD
    Arm description
    		 Participants who received VIT-2763 once a day (QD).
    Arm type
    		 Experimental

    Investigational medicinal product name
    VIT-2763
    Investigational medicinal product code
    Other name
    Vamifeport
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    VIT-2763 was administered once a day (QD), in a total daily dose of 60 mg or 120 mg, depending on participant's body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. VIT-2763 capsules were administered approximately 1 hour after meals at the same clock time (between 08:00 and 10:00 for the morning dose, and between 20:00 and 22:00 for the evening dose, respectively). Food intake, except for water ad libitum, was to be avoided for at least 1 hour prior to and 1 hour after the morning and evening dose administration.

    Arm title
    		 VIT-2763 BID
    Arm description
    		 Participants who received VIT-2763 twice daily (BID).
    Arm type
    		 Experimental

    Investigational medicinal product name
    VIT-2763
    Investigational medicinal product code
    Other name
    Vamifeport
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    VIT-2763 was administered twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on participant's body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. VIT-2763 capsules were administered approximately 1 hour after meals at the same clock time (between 08:00 and 10:00 for the morning dose, and between 20:00 and 22:00 for the evening dose, respectively). Food intake, except for water ad libitum, was to be avoided for at least 1 hour prior to and 1 hour after the morning and evening dose administration.

    Arm title
    		 Placebo
    Arm description
    		 Participants who received placebo twice daily.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo was administered twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on participant's body weight, during 12 weeks. Participants received placebo at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. Placebo capsules were administered approximately 1 hour after meals at the same clock time (between 08:00 and 10:00 for the morning dose, and between 20:00 and 22:00 for the evening dose, respectively). Food intake, except for water ad libitum, was to be avoided for at least 1 hour prior to and 1 hour after the morning and evening dose administration.

    Number of subjects in period 1
    		VIT-2763 QD VIT-2763 BID Placebo
    Started
    9
    12
    4
    Completed
    8
    11
    4
    Not completed
    1
    1
    0
         Consent withdrawn by subject
    -
    1
    -
         Adverse event, non-fatal
    1
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    VIT-2763 QD
    Reporting group description
    		 Participants who received VIT-2763 once a day (QD).

    Reporting group title
    VIT-2763 BID
    Reporting group description
    		 Participants who received VIT-2763 twice daily (BID).

    Reporting group title
    Placebo
    Reporting group description
    		 Participants who received placebo twice daily.

    Reporting group values
    		 VIT-2763 QD VIT-2763 BID Placebo Total
    Number of subjects
    		 9 12 4 25
    Age categorical
    Units: Subjects
        <=18 years
    		 0 0 0 0
        Between 18 and 65 years
    		 9 12 4 25
        >=65 years
    		 0 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 6 2 1 9
        Male
    		 3 10 3 16

    End points

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    End points reporting groups
    Reporting group title
    VIT-2763 QD
    Reporting group description
    		 Participants who received VIT-2763 once a day (QD).

    Reporting group title
    VIT-2763 BID
    Reporting group description
    		 Participants who received VIT-2763 twice daily (BID).

    Reporting group title
    Placebo
    Reporting group description
    		 Participants who received placebo twice daily.

    Primary: Number of Participants With TEAEs

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    End point title
    		 Number of Participants With TEAEs [1]
    End point description
    Please note that in this section we are presenting just the overview of the adverse events experienced by the trial participants, in particular, the number of participants with at least one TEAE. Please refer to the detailed tables included on the Adverse Event Module for specifics. TEAEs = Treatment-Emergent Adverse Events
    End point type
    Primary
    End point timeframe
    From baseline to Week 16.
    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: The analysis is descriptive. No inferential statistics were performed to compare treatment groups.
    End point values
    		 VIT-2763 QD VIT-2763 BID Placebo
    Number of subjects analysed
    9
    12
    4
    Units: Number
        Number of Participants With TEAEs
    6
    7
    3
    No statistical analyses for this end point

    Primary: Changes in the Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)

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    End point title
    		 Changes in the Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) [2]
    End point description
    Summary of the values by visit from baseline and changes from baseline by post-baseline visit.
    End point type
    Primary
    End point timeframe
    From baseline to Week 12.
    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: The analysis is descriptive. No inferential statistics were performed to compare treatment groups.
    End point values
    		 VIT-2763 QD VIT-2763 BID Placebo
    Number of subjects analysed
    9
    12
    4
    Units: millimeters of mercury (mmHg)
    arithmetic mean (standard deviation)
        SBP Baseline
    114.2 ( 11.62 )
    114.7 ( 13.88 )
    114.8 ( 12.97 )
        SBP Week 1
    115.0 ( 8.35 )
    116.7 ( 10.65 )
    104.0 ( 8.98 )
        SBP Change from Baseline to Week 1
    0.8 ( 8.94 )
    2.0 ( 9.72 )
    -10.8 ( 10.90 )
        SBP Week 2
    113.9 ( 9.41 )
    118.3 ( 14.85 )
    114.8 ( 15.06 )
        SBP Change from Baseline to Week 2
    -0.3 ( 6.60 )
    3.6 ( 8.78 )
    0.0 ( 2.83 )
        SBP Week 4
    112.0 ( 11.20 )
    113.9 ( 12.41 )
    104.8 ( 6.85 )
        SBP Change from Baseline to Week 4
    -2.2 ( 8.77 )
    -0.8 ( 8.51 )
    -10.0 ( 14.72 )
        SBP Week 8
    114.9 ( 7.41 )
    113.8 ( 10.54 )
    109.8 ( 12.61 )
        SBP Change from Baseline to Week 8
    0.7 ( 11.11 )
    0.9 ( 9.63 )
    -5.0 ( 1.63 )
        SBP Week 12
    113.4 ( 11.02 )
    112.8 ( 11.31 )
    107.5 ( 10.08 )
        SBP Change from Baseline to Week 12
    -0.8 ( 13.02 )
    -0.1 ( 10.85 )
    -7.3 ( 3.40 )
        DBP Baseline
    65.94 ( 10.088 )
    65.00 ( 10.189 )
    66.50 ( 7.853 )
        DBP Week 1
    66.44 ( 6.710 )
    69.33 ( 9.921 )
    61.75 ( 4.856 )
        DBP Change from Baseline to Week 1
    0.50 ( 4.886 )
    4.33 ( 6.344 )
    -4.75 ( 6.185 )
        DBP Week 2
    65.89 ( 9.545 )
    68.83 ( 11.535 )
    64.00 ( 6.055 )
        DBP Change from Baseline to Week 2
    -0.06 ( 5.353 )
    3.83 ( 8.726 )
    -2.50 ( 3.000 )
        DBP Week 4
    65.22 ( 11.043 )
    62.33 ( 9.069 )
    62.75 ( 3.775 )
        DBP Change from Baseline to Week 4
    -0.72 ( 9.398 )
    -2.67 ( 9.921 )
    -3.75 ( 5.058 )
        DBP Week 8
    63.44 ( 6.579 )
    67.18 ( 9.174 )
    66.75 ( 7.500 )
        DBP Change from Baseline to Week 8
    -2.50 ( 8.216 )
    3.27 ( 8.439 )
    0.25 ( 7.848 )
        DBP Week 12
    63.67 ( 7.697 )
    67.64 ( 9.657 )
    62.50 ( 3.317 )
        DBP Change from Baseline to Week 12
    -2.28 ( 10.140 )
    3.73 ( 6.310 )
    -4.00 ( 6.880 )
    No statistical analyses for this end point

    Primary: Changes in the Heart Rate

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    End point title
    		 Changes in the Heart Rate [3]
    End point description
    Summary of the values by visit from baseline and changes from baseline by post-baseline visit.
    End point type
    Primary
    End point timeframe
    From baseline to Week 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: The analysis is descriptive. No inferential statistics were performed to compare treatment groups.
    End point values
    		 VIT-2763 QD VIT-2763 BID Placebo
    Number of subjects analysed
    9
    12
    4
    Units: Pulse Rate (bpm)
    arithmetic mean (standard deviation)
        Baseline
    82.00 ( 12.135 )
    77.71 ( 10.208 )
    73.75 ( 5.560 )
        Week 1
    82.33 ( 11.694 )
    80.33 ( 9.875 )
    70.75 ( 6.449 )
        Change from Baseline to Week 1
    0.33 ( 6.042 )
    2.63 ( 5.859 )
    -3.00 ( 5.715 )
        Week 2
    83.56 ( 12.856 )
    75.83 ( 9.953 )
    75.00 ( 8.756 )
        Change from Baseline to Week 2
    1.56 ( 10.442 )
    -1.88 ( 8.263 )
    1.25 ( 3.775 )
        Week 4
    82.33 ( 14.414 )
    77.92 ( 9.830 )
    73.25 ( 3.775 )
        Change from Baseline to Week 4
    0.33 ( 8.818 )
    0.21 ( 5.541 )
    -0.50 ( 1.915 )
        Week 8
    83.22 ( 11.322 )
    80.27 ( 10.992 )
    72.50 ( 5.196 )
        Change from Baseline to Week 8
    1.22 ( 6.648 )
    2.68 ( 9.040 )
    -1.25 ( 7.411 )
    No statistical analyses for this end point

    Primary: Changes in 12-lead Electrocardiogram (ECG) Parameters

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    End point title
    		 Changes in 12-lead Electrocardiogram (ECG) Parameters [4]
    End point description
    Values by visit from baseline and changes from baseline by post-baseline visit for PR interval, QRS duration, QT interval and QTcF interval. PR interval represents the time from the onset of the P wave to the start of the QRS complex. QRS duration represents the time required for a stimulus to spread through the ventricles (ventricular depolarization). QT interval represents the time from the start of the Q wave to the end of the T wave. RR interval represents the time from the onset of one R wave to the onset of the next one, one complete cardiac cycle. QT corrected for heart rate (QTc) interval reflects ventricular repolarization. BAS = Baseline.
    End point type
    Primary
    End point timeframe
    From baseline to Week 12.
    Notes
    [4] - 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: The analysis is descriptive. No inferential statistics were performed to compare treatment groups.
    End point values
    		 VIT-2763 QD VIT-2763 BID Placebo
    Number of subjects analysed
    9
    12
    4
    Units: milliseconds (ms)
    arithmetic mean (standard deviation)
        PR interval - BAS
    158.3 ( 25.14 )
    147.6 ( 20.05 )
    175.8 ( 38.92 )
        PR interval - BAS 2h post-dose
    161.2 ( 21.28 )
    155.1 ( 17.98 )
    175.0 ( 14.07 )
        PR interval - Change from BAS to BAS 2h post-dose
    2.9 ( 25.78 )
    6.3 ( 16.45 )
    -0.8 ( 38.59 )
        PR interval - Week 1
    159.2 ( 22.90 )
    153.0 ( 14.12 )
    189.5 ( 18.72 )
        PR interval - Change from BAS to Week 1
    0.9 ( 16.59 )
    4.7 ( 12.71 )
    13.8 ( 25.67 )
        PR interval - Week 2
    167.6 ( 26.49 )
    153.0 ( 17.87 )
    185.5 ( 4.43 )
        PR interval - Change from BAS to Week 2
    9.2 ( 27.64 )
    5.5 ( 13.56 )
    9.8 ( 37.56 )
        PR interval - Week 4
    154.0 ( 26.49 )
    154.2 ( 21.19 )
    185.0 ( 7.39 )
        PR interval - Change from BAS to Week 4
    -4.3 ( 18.85 )
    8.1 ( 15.48 )
    9.3 ( 33.42 )
        PR interval - Week 8
    161.2 ( 19.34 )
    153.5 ( 20.61 )
    185.8 ( 10.14 )
        PR interval - Change from BAS to Week 8
    2.9 ( 17.05 )
    7.1 ( 12.66 )
    10.0 ( 32.86 )
        PR interval - Week 12
    160.6 ( 19.55 )
    156.6 ( 14.47 )
    182.8 ( 11.98 )
        PR interval - Change from BAS to Week 12
    2.2 ( 19.50 )
    11.5 ( 14.25 )
    7.0 ( 36.09 )
        QRS duration - BAS
    94.0 ( 18.73 )
    88.8 ( 19.17 )
    119.3 ( 50.12 )
        QRS duration - BAS 2h post-dose
    94.3 ( 20.20 )
    95.8 ( 10.96 )
    85.0 ( 11.60 )
        QRS duration - Change from BAS to BAS 2h post-dose
    0.3 ( 4.82 )
    7.0 ( 14.39 )
    -34.3 ( 55.27 )
        QRS duration - Week 1
    98.4 ( 23.16 )
    96.1 ( 11.27 )
    98.0 ( 16.57 )
        QRS duration - Change from BAS to Week 1
    4.4 ( 14.75 )
    7.3 ( 13.45 )
    -21.3 ( 65.76 )
        QRS duration - Week 2
    94.0 ( 29.01 )
    96.3 ( 14.13 )
    91.3 ( 12.09 )
        QRS duration - Change from BAS to Week 2
    0.0 ( 23.04 )
    7.6 ( 17.96 )
    -28.0 ( 61.36 )
        QRS duration - Week 4
    92.3 ( 23.32 )
    92.6 ( 15.11 )
    94.3 ( 8.10 )
        QRS duration - Change from BAS to Week 4
    -1.7 ( 11.16 )
    6.0 ( 16.76 )
    -25.0 ( 54.02 )
        QRS duration - Week 8
    91.0 ( 24.72 )
    94.9 ( 18.27 )
    93.3 ( 10.81 )
        QRS duration - Change from BAS to Week 8
    -3.0 ( 15.99 )
    7.4 ( 28.52 )
    -26.0 ( 60.02 )
        QRS duration - Week 12
    94.3 ( 27.31 )
    84.8 ( 18.66 )
    91.8 ( 10.59 )
        QRS duration - Change from BAS to Week 12
    0.3 ( 20.64 )
    -2.7 ( 23.70 )
    -27.5 ( 60.69 )
        QT interval - BAS
    371.7 ( 76.41 )
    374.2 ( 21.78 )
    420.8 ( 48.75 )
        QT interval - BAS 2h post-dose
    385.7 ( 50.89 )
    376.8 ( 22.25 )
    379.5 ( 30.17 )
        QT interval - Change from BAS to BAS 2h post-dose
    14.0 ( 61.00 )
    2.6 ( 21.49 )
    -41.3 ( 68.30 )
        QT interval - Week 1
    394.1 ( 47.36 )
    374.8 ( 33.12 )
    381.5 ( 32.18 )
        QT interval - Change from BAS to Week 1
    22.4 ( 57.20 )
    0.7 ( 28.96 )
    -39.3 ( 71.63 )
        QT interval - Week 2
    384.0 ( 55.52 )
    367.0 ( 24.68 )
    378.3 ( 27.89 )
        QT interval - Change from BAS to Week 2
    12.3 ( 76.14 )
    -7.2 ( 27.61 )
    -42.5 ( 61.87 )
        QT interval - Week 4
    370.1 ( 67.13 )
    373.6 ( 21.22 )
    378.0 ( 36.51 )
        QT interval - Change from BAS to Week 4
    -1.6 ( 23.07 )
    0.9 ( 24.94 )
    -42.8 ( 61.50 )
        QT interval - Week 8
    389.7 ( 39.20 )
    379.4 ( 25.08 )
    381.8 ( 37.99 )
        QT interval - Change from BAS to Week 8
    18.0 ( 69.60 )
    4.6 ( 22.59 )
    -39.0 ( 64.59 )
        QT interval - Week 12
    380.9 ( 59.97 )
    364.2 ( 36.22 )
    388.3 ( 42.85 )
        QT interval - Change from BAS to Week 12
    9.2 ( 33.53 )
    -10.5 ( 33.69 )
    -32.5 ( 72.06 )
        RR interval - BAS
    789.4 ( 128.89 )
    801.4 ( 95.56 )
    877.5 ( 114.65 )
        RR interval - BAS 2h post-dose
    781.2 ( 155.21 )
    805.5 ( 119.36 )
    812.5 ( 124.38 )
        RR interval - Change from BAS to BAS 2h post-dose
    -8.2 ( 87.78 )
    4.1 ( 112.91 )
    -65.0 ( 196.09 )
        RR interval - Week 1
    793.2 ( 105.35 )
    864.9 ( 149.33 )
    826.5 ( 96.57 )
        RR interval - Change from BAS to Week 1
    3.8 ( 82.82 )
    63.5 ( 137.59 )
    -51.0 ( 98.80 )
        RR interval - Week 2
    757.3 ( 120.25 )
    830.1 ( 138.34 )
    833.8 ( 16.01 )
        RR interval - Change from BAS to Week 2
    -32.1 ( 146.70 )
    28.7 ( 117.34 )
    -43.8 ( 103.92 )
        RR interval - Week 4
    783.7 ( 110.55 )
    829.8 ( 154.72 )
    825.8 ( 96.08 )
        RR interval - Change from BAS to Week 4
    -5.8 ( 62.11 )
    34.1 ( 137.64 )
    -51.8 ( 111.05 )
        RR interval - Week 8
    765.0 ( 91.52 )
    810.1 ( 138.16 )
    844.8 ( 135.47 )
        RR interval - Change from BAS to Week 8
    -24.4 ( 116.42 )
    17.2 ( 108.39 )
    -32.8 ( 152.45 )
        RR interval - Week 12
    780.7 ( 140.88 )
    812.9 ( 93.89 )
    878.3 ( 133.70 )
        RR interval - Change from BAS to Week 12
    -8.8 ( 115.95 )
    20.0 ( 73.46 )
    0.8 ( 172.81 )
        QTcF interval - BAS
    403.3 ( 80.48 )
    400.5 ( 22.76 )
    416.8 ( 17.46 )
        QTcF interval - BAS 2h post-dose
    419.3 ( 36.58 )
    410.1 ( 25.34 )
    407.3 ( 13.05 )
        QTcF interval - Change from BAS to BAS 2h post-dos
    16.0 ( 66.51 )
    9.6 ( 22.60 )
    -9.5 ( 6.76 )
        QTcF interval - Week 1
    425.7 ( 37.25 )
    397.3 ( 30.44 )
    406.8 ( 26.02 )
        QTcF interval - Change from BAS to Week 1
    22.3 ( 65.53 )
    -3.3 ( 24.92 )
    -10.0 ( 20.38 )
        QTcF interval - Week 2
    420.8 ( 42.28 )
    393.3 ( 28.90 )
    402.0 ( 30.30 )
        QTcF interval - Change from BAS to Week 2
    17.4 ( 77.84 )
    -7.2 ( 27.52 )
    -14.8 ( 14.52 )
        QTcF interval - Week 4
    401.3 ( 64.21 )
    401.8 ( 27.41 )
    402.8 ( 23.63 )
        QTcF interval - Change from BAS to Week 4
    -2.0 ( 24.74 )
    2.1 ( 24.41 )
    -14.0 ( 7.07 )
        QTcF interval - Week 8
    426.0 ( 30.12 )
    399.6 ( 27.17 )
    404.3 ( 19.86 )
        QTcF interval - Change from BAS to Week 8
    22.7 ( 80.36 )
    0.3 ( 26.86 )
    -12.5 ( 4.51 )
        QTcF interval - Week 12
    414.1 ( 53.40 )
    391.4 ( 42.91 )
    405.3 ( 25.71 )
        QTcF interval - Change from BAS to Week 12
    10.8 ( 37.94 )
    -8.0 ( 37.96 )
    -11.5 ( 10.63 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Total Serum Iron

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    End point title
    		 Change From Baseline in Total Serum Iron
    End point description
    Assessment of total serum Iron from baseline over a 12-week period (absolute and change from baseline). For the serum iron parameter, the 'Baseline' was collected during the screening period within the biochemistry sample.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 12.
    End point values
    		 VIT-2763 QD VIT-2763 BID Placebo
    Number of subjects analysed
    9
    12
    4
    Units: Micromoles per Litre (umol/L)
    arithmetic mean (standard deviation)
        Baseline
    23.81 ( 13.438 )
    28.42 ( 9.796 )
    30.88 ( 13.329 )
        Week 1
    12.54 ( 9.159 )
    11.38 ( 7.693 )
    28.20 ( 17.919 )
        Change from Baselines to Week 1
    -11.27 ( 7.158 )
    -17.03 ( 9.611 )
    -2.68 ( 12.365 )
        Week 2
    11.71 ( 8.622 )
    12.99 ( 8.978 )
    32.48 ( 15.541 )
        Change from Baseline to Week 2
    -9.74 ( 4.707 )
    -15.43 ( 12.784 )
    1.60 ( 3.996 )
        Week 4
    13.90 ( 12.222 )
    12.85 ( 7.583 )
    31.08 ( 13.721 )
        Change from Baseline to Week 4
    -9.33 ( 4.659 )
    -14.03 ( 8.432 )
    0.20 ( 2.902 )
        Week 8
    11.35 ( 7.822 )
    11.55 ( 9.651 )
    26.70 ( 12.040 )
        Change from Baseline to Week 8
    -11.88 ( 9.627 )
    -17.05 ( 11.936 )
    -0.37 ( 3.550 )
        Week 12
    13.72 ( 11.031 )
    11.62 ( 6.442 )
    27.10 ( 12.856 )
        Change from Baseline to Week 12
    -10.54 ( 6.782 )
    -16.22 ( 10.892 )
    0.03 ( 2.701 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Serum Ferritin

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    End point title
    		 Change From Baseline in Serum Ferritin
    End point description
    Assessment of serum ferritin from baseline over a 12-week period (absolute and change from baseline). For the serum ferritin parameter, the 'Baseline' was collected during the screening period within the biochemistry sample.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 12.
    End point values
    		 VIT-2763 QD VIT-2763 BID Placebo
    Number of subjects analysed
    9
    12
    4
    Units: Microgrammes per Litre (ug/L)
    arithmetic mean (standard deviation)
        Baseline
    403.14 ( 226.315 )
    1133.23 ( 2119.115 )
    440.03 ( 321.453 )
        Week 1
    416.76 ( 220.567 )
    471.53 ( 209.800 )
    445.80 ( 228.244 )
        Change from Baseline to Week 1
    -10.14 ( 31.509 )
    40.89 ( 164.466 )
    5.77 ( 93.946 )
        Week 2
    441.34 ( 269.599 )
    500.79 ( 230.076 )
    411.35 ( 207.477 )
        Change from Baseline to Week 2
    3.29 ( 60.579 )
    23.80 ( 149.147 )
    -49.80 ( 76.867 )
        Week 4
    476.60 ( 282.457 )
    585.71 ( 311.224 )
    389.83 ( 240.553 )
        Change from Baseline to Week 4
    1.05 ( 61.638 )
    89.98 ( 249.397 )
    -50.20 ( 80.958 )
        Week 8
    463.67 ( 206.646 )
    508.56 ( 211.366 )
    340.23 ( 165.356 )
        Change from Baseline to Week 8
    -11.88 ( 43.063 )
    52.84 ( 157.952 )
    -9.45 ( 20.577 )
        Week 12
    465.72 ( 227.378 )
    927.42 ( 1478.471 )
    261.70 ( 53.033 )
        Change from Baseline to Week 12
    -15.84 ( 66.208 )
    -139.23 ( 626.380 )
    7.25 ( 57.205 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Serum Transferrin

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    End point title
    		 Change From Baseline in Serum Transferrin
    End point description
    Assessment of serum transferrin from baseline over a 12-week period (absolute and change from baseline). For the serum transferrin parameter, the "Baseline 2h post-dose" was defined as the value at Visit 3 2h post-dose.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 12.
    End point values
    		 VIT-2763 QD VIT-2763 BID Placebo
    Number of subjects analysed
    9
    12
    4
    Units: Grammes per Litre (g/L)
    arithmetic mean (standard deviation)
        Baseline 2h post-dose
    1.601 ( 0.4377 )
    1.673 ( 0.3177 )
    1.655 ( 0.3003 )
        Week 1
    1.610 ( 0.4557 )
    1.822 ( 0.3903 )
    1.683 ( 0.2210 )
        Change from Baseline to Week 1
    0.009 ( 0.1184 )
    0.149 ( 0.1334 )
    0.028 ( 0.2419 )
        Week 2
    1.601 ( 0.5043 )
    1.759 ( 0.3569 )
    1.655 ( 0.2408 )
        Change from Baseline to Week 2
    -0.014 ( 0.1516 )
    0.087 ( 0.1429 )
    0.000 ( 0.1846 )
        Week 4
    1.708 ( 0.2672 )
    1.705 ( 0.3496 )
    1.745 ( 0.3756 )
        Change from Baseline to Week 4
    0.053 ( 0.2014 )
    0.054 ( 0.1535 )
    0.090 ( 0.2740 )
        Week 8
    1.640 ( 0.2745 )
    1.725 ( 0.3194 )
    1.553 ( 0.2836 )
        Change from Baseline to Week 8
    -0.015 ( 0.0675 )
    0.093 ( 0.1618 )
    -0.120 ( 0.0854 )
        Week 12
    1.706 ( 0.1954 )
    1.656 ( 0.2974 )
    1.610 ( 0.4784 )
        Change from Baseline to Week 12
    -0.040 ( 0.1116 )
    0.100 ( 0.1044 )
    -0.063 ( 0.1266 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Calculated Transferrin Saturation (TSAT)

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    End point title
    		 Change From Baseline in Calculated Transferrin Saturation (TSAT)
    End point description
    Assessment of TSAT from baseline over a 12-week period (absolute and change from baseline). For the calculated transferrin saturation parameter, the 'Baseline' was collected during the screening period within the biochemistry sample. Transferrin Saturation (TSAT) was calculated as Total Iron /Total Iron Binding Capacity (TIBC) X 100.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 12.
    End point values
    		 VIT-2763 QD VIT-2763 BID Placebo
    Number of subjects analysed
    9
    12
    4
    Units: Percentage (%)
    arithmetic mean (standard deviation)
        Baseline
    69.3 ( 31.16 )
    79.0 ( 24.05 )
    83.3 ( 33.50 )
        Week 1
    36.8 ( 24.44 )
    32.2 ( 21.44 )
    56.5 ( 30.56 )
        Change from Baselone to Week 1
    -32.6 ( 19.55 )
    -46.8 ( 22.55 )
    -26.8 ( 28.77 )
        Week 2
    36.0 ( 22.81 )
    35.8 ( 24.13 )
    71.5 ( 33.91 )
        Change from Baseline to Week 2
    -29.5 ( 14.57 )
    -43.2 ( 33.23 )
    -11.8 ( 23.50 )
        Week 4
    41.0 ( 32.27 )
    38.4 ( 24.85 )
    82.5 ( 35.00 )
        Change from Baseline to Week 4
    -24.0 ( 18.21 )
    -38.7 ( 27.57 )
    -0.8 ( 1.50 )
        Week 8
    32.3 ( 17.48 )
    33.5 ( 27.08 )
    77.3 ( 39.26 )
        Change from Baseline to Week 8
    -32.7 ( 25.80 )
    -47.9 ( 29.70 )
    -0.3 ( 0.58 )
        Week 12
    36.0 ( 25.93 )
    34.8 ( 17.76 )
    76.3 ( 40.99 )
        Change from Baseline to Week 12
    -27.6 ( 20.37 )
    -46.8 ( 23.61 )
    -1.3 ( 2.31 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetics Parameters - VIT-2763 Plasma Concentration Over Time

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    End point title
    		 Pharmacokinetics Parameters - VIT-2763 Plasma Concentration Over Time [5]
    End point description
    Sparse sampling for determination of VIT-2763 plasma concentration following multiple dosing was obtained from predose trough to 3 hours or 4 hours post-dose at selected study visits. Pharmacokinetics parameters (Cmax, clearance, distribution volume, area under the curve (AUC) were not calculated for the study.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 4, Week 8 and Week 12.
    Notes
    [5] - 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: There are no results of PK parameters applicable for the Placebo arm.
    End point values
    		 VIT-2763 QD VIT-2763 BID
    Number of subjects analysed
    9
    12
    Units: Nanograms per milliliter (ng/mL)
    arithmetic mean (standard deviation)
        Visit 3 1 hour post-dose
    796.5 ( 508.2 )
    609.6 ( 455.2 )
        Visit 3 4 hours post-dose
    222.3 ( 73.1 )
    411.0 ( 269.5 )
        Visit 6 pre-dose
    17.2 ( 10.2 )
    114.4 ( 78.2 )
        Visit 6 1 hour post-dose
    986.2 ( 796.7 )
    483.5 ( 247.3 )
        Visit 6 3 hours post-dose
    578.2 ( 494.5 )
    544.3 ( 337.5 )
        Visit 7 pre-dose
    15.5 ( 6.6 )
    111.0 ( 61.9 )
        Visit 7 1 hour post-dose
    751.7 ( 682.3 )
    692.2 ( 470.2 )
        Visit 7 4 hours post-dose
    295.5 ( 209.5 )
    384.3 ( 200.9 )
        Visit 8 pre-dose
    361.5 ( 579.2 )
    136.0 ( 76.8 )
        Visit 8 1 hour post-dose
    894.1 ( 670.3 )
    650.6 ( 474.5 )
        Visit 8 3 hours post-dose
    684.0 ( 405.5 )
    569.9 ( 343.1 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Overall study
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    VIT-2763 QD
    Reporting group description
    		 Participants who received VIT-2763 once a day (QD).

    Reporting group title
    VIT-2763 BID
    Reporting group description
    		 Participants who received VIT-2763 twice daily (BID).

    Reporting group title
    Placebo
    Reporting group description
    		 Participants who received placebo twice daily.

    Serious adverse events
    		 VIT-2763 QD VIT-2763 BID Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 12 (0.00%)
    0 / 4 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 VIT-2763 QD VIT-2763 BID Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 9 (66.67%)
    7 / 12 (58.33%)
    3 / 4 (75.00%)
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 9 (22.22%)
    2 / 12 (16.67%)
    0 / 4 (0.00%)
         occurrences all number
    2
    2
    0
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 12 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 12 (16.67%)
    0 / 4 (0.00%)
         occurrences all number
    0
    2
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Respiratory symptom
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 12 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Palpitations
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    1
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    1
    Presyncope
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    1
    1
    0
    Haemolysis
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 12 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Ear and labyrinth disorders
    Hypoacusis
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 12 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Eye disorders
    Photopsia
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 12 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Vitreous floaters
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 12 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 12 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Dyspepsia
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 12 (16.67%)
    0 / 4 (0.00%)
         occurrences all number
    0
    2
    0
    Faeces discoloured
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Nausea
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Vomiting
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Hepatobiliary disorders
    Jaundice
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 12 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 12 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Skin ulcer
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Bone pain
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Infections and infestations
    Rhinitis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Urinary tract infection
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 12 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 12 (8.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Dec 2019
    Version 2.0
    10 Dec 2020
    Version 3.0
    12 Apr 2021
    Version 4.0 (final)

    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
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