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    Clinical Trial Results:
    A Phase 2a, Double-blind, Randomised, Placebo-controlled, Efficacy, and Safety Study of Multiple Doses of VIT-2763 in Subjects With Sickle Cell Disease (ViSion Serenity)

    Summary
    EudraCT number
    		 2020-005072-34
    Trial protocol
    		 GR  
    Global end of trial date
    07 Mar 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    12 Feb 2025
    First version publication date
    12 Feb 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    VIT-2763-SCD-202
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04817670
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND Number: 147878
    Sponsors
    Sponsor organisation name
    Vifor (International) Inc.
    Sponsor organisation address
    Rechenstrasse 37, St. Gallen, Switzerland, CH-9014
    Public contact
    Study Director, CSL Behring LLC, +1 610 878 4000, clinicaltrials@cslbehring.com
    Scientific contact
    Study Director, CSL Behring LLC, +1 610 878 4000, clinicaltrials@cslbehring.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
    29 Apr 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Mar 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to explore the effect of VIT-2763 on markers of haemolysis.
    Protection of trial subjects
    This study was carried out in accordance with the International Council for Harmonisation (ICH) Good Clinical Practice (GCP) guidelines, the ethical principles that have their origin in the Declaration of Helsinki, all applicable national and local regulations, and standard operating procedures for clinical research and development at Vifor. The study protocol and all amendments were approved by the Independent Ethics Committee (IEC) / Institutional Review Board (IRB) of the participating center. Before any protocol-specific procedures were carried out, participants were informed, in an understandable form, about the nature, scope, and possible consequences of the study. Participant informed consent was obtained and documented according to the provisions of ICH GCP and applicable regulatory requirements. Written informed consent was provided by each participant before any protocol-specific procedures were carried out.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    18 Nov 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Lebanon: 10
    Country: Number of subjects enrolled
    United Kingdom: 5
    Country: Number of subjects enrolled
    United States: 7
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Greece: 2
    Worldwide total number of subjects
    25
    EEA total number of subjects
    3
    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
    		 There were 22 sites initiated for this study in 5 countries (The United Kingdom, Lebanon, Greece, the United States, and France).

    Pre-assignment
    Screening details
    		 A total of 46 participants were screened in this study, of which 28 were screen failures. Out of these 28 participants, 9 were rescreened, and 7 were found eligible for the study. 25 participants were enrolled in the study.

    Period 1
    Period 1 title
    Overall study (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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1: VIT-2763 60 mg BID (120 mg/Day)
    Arm description
    		 Participants received VIT-2763 60 milligrams (mg) (2 x 30 mg capsules), orally, twice daily (BID) for 8 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VIT-2763
    Investigational medicinal product code
    VIT-2763
    Other name
    Vamifeport
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    VIT-2763 capsule for oral administration.

    Arm title
    		 Cohort 2: VIT-2763 120 mg BID (240 mg/Day)
    Arm description
    		 Participants received VIT-2763 120 mg (2 x 60 mg capsules), orally, BID for 8 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VIT-2763
    Investigational medicinal product code
    VIT-2763
    Other name
    Vamifeport
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    VIT-2763 capsule for oral administration.

    Arm title
    		 Cohort 3: VIT-2763 120 mg TID (360 mg/Day)
    Arm description
    		 Participants received VIT-2763 120 mg (2 x 60 mg capsules), orally, three times daily (TID) for 8 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VIT-2763
    Investigational medicinal product code
    VIT-2763
    Other name
    Vamifeport
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    VIT-2763 capsule for oral administration.

    Arm title
    		 Cohort 4: Placebo
    Arm description
    		 Participants received placebo capsules, orally, BID or TID for 8 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo capsule for oral administration.

    Arm title
    		 Cohort 2a: VIT-2763 30 mg BID (60 mg/day)
    Arm description
    		 One participant received VIT-2763 30 mg, orally, BID for 8 weeks under Protocol Version 2.0. However, after the implementation of Protocol Version 3.0 and higher, this participant was excluded from the efficacy analysis and only safety data were reported (as planned).
    Arm type
    		 Experimental

    Investigational medicinal product name
    VIT-2763
    Investigational medicinal product code
    VIT-2763
    Other name
    Vamifeport
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    VIT-2763 capsule for oral administration.

    Number of subjects in period 1
    		Cohort 1: VIT-2763 60 mg BID (120 mg/Day) Cohort 2: VIT-2763 120 mg BID (240 mg/Day) Cohort 3: VIT-2763 120 mg TID (360 mg/Day) Cohort 4: Placebo Cohort 2a: VIT-2763 30 mg BID (60 mg/day)
    Started
    6
    6
    6
    6
    1
    Intent-to-treat (ITT) Population
    6
    6
    6
    6
    0 [1]
    Completed
    5
    6
    5
    6
    1
    Not completed
    1
    0
    1
    0
    0
         Lost to follow-up
    -
    -
    1
    -
    -
         Withdrawal by subject
    1
    -
    -
    -
    -
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: One participant in Cohort 2a arm was enrolled under Protocol Version 2.0. However, after the implementation of Protocol Version 3.0 and higher, this participant was excluded from the efficacy analysis and only safety data were reported (as planned).

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1: VIT-2763 60 mg BID (120 mg/Day)
    Reporting group description
    		 Participants received VIT-2763 60 milligrams (mg) (2 x 30 mg capsules), orally, twice daily (BID) for 8 weeks.

    Reporting group title
    Cohort 2: VIT-2763 120 mg BID (240 mg/Day)
    Reporting group description
    		 Participants received VIT-2763 120 mg (2 x 60 mg capsules), orally, BID for 8 weeks.

    Reporting group title
    Cohort 3: VIT-2763 120 mg TID (360 mg/Day)
    Reporting group description
    		 Participants received VIT-2763 120 mg (2 x 60 mg capsules), orally, three times daily (TID) for 8 weeks.

    Reporting group title
    Cohort 4: Placebo
    Reporting group description
    		 Participants received placebo capsules, orally, BID or TID for 8 weeks.

    Reporting group title
    Cohort 2a: VIT-2763 30 mg BID (60 mg/day)
    Reporting group description
    		 One participant received VIT-2763 30 mg, orally, BID for 8 weeks under Protocol Version 2.0. However, after the implementation of Protocol Version 3.0 and higher, this participant was excluded from the efficacy analysis and only safety data were reported (as planned).

    Reporting group values
    		 Cohort 1: VIT-2763 60 mg BID (120 mg/Day) Cohort 2: VIT-2763 120 mg BID (240 mg/Day) Cohort 3: VIT-2763 120 mg TID (360 mg/Day) Cohort 4: Placebo Cohort 2a: VIT-2763 30 mg BID (60 mg/day) Total
    Number of subjects
    		 6 6 6 6 1 25
    Age categorical
    Units: Subjects
    Age continuous
    Data for Cohort 2a not reported here due to concerns regarding participant privacy as there was only 1 participant in Cohort 2a arm.
    Units: years
        arithmetic mean (standard deviation)
    		 30.2 ( 7.41 ) 36.0 ( 11.87 ) 29.3 ( 9.48 ) 25.3 ( 7.17 ) 0.00 ( 0.00 ) -
    Gender categorical
    Data for Cohort 2a not reported here due to concerns regarding participant privacy as there was only 1 participant in Cohort 2a arm.
    Units: Subjects
        Female
    		 4 3 3 3 0 13
        Male
    		 2 3 3 3 0 11
        Not disclosed
    		 0 0 0 0 1 1
    Ethnicity
    Data for Cohort 2a not reported here due to concerns regarding participant privacy as there was only 1 participant in Cohort 2a arm.
    Units: Subjects
        Hispanic or Latino
    		 0 0 0 0 0 0
        Not Hispanic or Latino
    		 5 6 5 6 0 22
        Unknown or Not Reported
    		 1 0 1 0 0 2
        Not disclosed
    		 0 0 0 0 1 1
    Race
    Data for Cohort 2a not reported here due to concerns regarding participant privacy as there was only 1 participant in Cohort 2a arm.
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0 0 0
        Asian
    		 0 0 0 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0 0 0
        Black or African American
    		 2 3 3 4 0 12
        White
    		 4 3 2 2 0 11
        More than one race
    		 0 0 0 0 0 0
        Unknown or Not Reported
    		 0 0 1 0 0 1
        Not disclosed
    		 0 0 0 0 1 1

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1: VIT-2763 60 mg BID (120 mg/Day)
    Reporting group description
    		 Participants received VIT-2763 60 milligrams (mg) (2 x 30 mg capsules), orally, twice daily (BID) for 8 weeks.

    Reporting group title
    Cohort 2: VIT-2763 120 mg BID (240 mg/Day)
    Reporting group description
    		 Participants received VIT-2763 120 mg (2 x 60 mg capsules), orally, BID for 8 weeks.

    Reporting group title
    Cohort 3: VIT-2763 120 mg TID (360 mg/Day)
    Reporting group description
    		 Participants received VIT-2763 120 mg (2 x 60 mg capsules), orally, three times daily (TID) for 8 weeks.

    Reporting group title
    Cohort 4: Placebo
    Reporting group description
    		 Participants received placebo capsules, orally, BID or TID for 8 weeks.

    Reporting group title
    Cohort 2a: VIT-2763 30 mg BID (60 mg/day)
    Reporting group description
    		 One participant received VIT-2763 30 mg, orally, BID for 8 weeks under Protocol Version 2.0. However, after the implementation of Protocol Version 3.0 and higher, this participant was excluded from the efficacy analysis and only safety data were reported (as planned).

    Primary: Mean Change From Baseline in Haemolysis Marker (Indirect Bilirubin)

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    End point title
    		 Mean Change From Baseline in Haemolysis Marker (Indirect Bilirubin) [1] [2]
    End point description
    Mean change from baseline in haemolysis markers was measured by reduction of indirect bilirubin. This analysis was performed on intent-to-treat (ITT) population. The ITT population consisted of all participants who were randomly assigned to a treatment group under Protocol Version 3.0 or higher. Here, the 'overall number of participants analyzed' (N) signifies the number of participants with evaluable data for this outcome measure. The 'number analyzed' (n) signifies the number of participants with evaluable data for each specified timepoint.
    End point type
    Primary
    End point timeframe
    Baseline and after 8 weeks of treatment
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: As the endpoint was descriptive, no statistical hypothesis testing was planned or conducted.
    [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: Data for all applicable arms is reported for this endpoint (except Cohort2a after implementation of Protocol Version 3 and higher [as planned]).
    End point values
    		 Cohort 1: VIT-2763 60 mg BID (120 mg/Day) Cohort 2: VIT-2763 120 mg BID (240 mg/Day) Cohort 3: VIT-2763 120 mg TID (360 mg/Day) Cohort 4: Placebo
    Number of subjects analysed
    5
    5
    5
    6
    Units: micromoles per liter (umol/L)
    arithmetic mean (standard deviation)
        Baseline (n=5,5,5,6)
    24.0 ( 13.51 )
    56.4 ( 43.71 )
    44.0 ( 24.48 )
    31.2 ( 12.95 )
        Change at 8 weeks (n=4,4,4,5)
    -4.0 ( 4.69 )
    -5.8 ( 11.35 )
    -21.8 ( 11.62 )
    0.6 ( 7.30 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline in Haemolysis Marker (Direct and Total Bilirubin)

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    End point title
    		 Mean Change From Baseline in Haemolysis Marker (Direct and Total Bilirubin) [3]
    End point description
    Mean change from Baseline in haemolysis markers was measured by direct and total bilirubin. This analysis was performed on ITT population. The ITT population consisted of all participants who were randomly assigned to a treatment group under Protocol Version 3.0 or higher. Here, the 'overall number of participants analyzed' (N) signifies the number of participants with evaluable data for this outcome measure. The 'number analyzed' (n) signifies the number of participants with evaluable data for each specified category.
    End point type
    Secondary
    End point timeframe
    Baseline and after 8 weeks of treatment
    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: Data for all applicable arms is reported for this endpoint (except Cohort2a after implementation of Protocol Version 3 and higher [as planned]).
    End point values
    		 Cohort 1: VIT-2763 60 mg BID (120 mg/Day) Cohort 2: VIT-2763 120 mg BID (240 mg/Day) Cohort 3: VIT-2763 120 mg TID (360 mg/Day) Cohort 4: Placebo
    Number of subjects analysed
    5
    6
    5
    6
    Units: umol/L
    arithmetic mean (standard deviation)
        Direct Bilirubin (n=4,4,4,5)
    -0.5 ( 1.29 )
    -2.0 ( 2.83 )
    -2.5 ( 7.05 )
    -0.4 ( 1.52 )
        Total Bilirubin (n=5,6,5,6)
    -4.2 ( 4.76 )
    -13.2 ( 16.57 )
    -26.0 ( 16.00 )
    -0.2 ( 7.08 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline in Haemolysis Marker (Lactate Dehydrogenase)

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    End point title
    		 Mean Change From Baseline in Haemolysis Marker (Lactate Dehydrogenase) [4]
    End point description
    Mean change from Baseline in haemolysis markers was measured by lactate dehydrogenase. This analysis was performed on ITT population. The ITT population consisted of all participants who were randomly assigned to a treatment group under Protocol Version 3.0 or higher. Here, the 'overall number of participants analyzed' (N) signifies the number of participants with evaluable data for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline and after 8 weeks of treatment
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data for all applicable arms is reported for this endpoint (except Cohort2a after implementation of Protocol Version 3 and higher [as planned]).
    End point values
    		 Cohort 1: VIT-2763 60 mg BID (120 mg/Day) Cohort 2: VIT-2763 120 mg BID (240 mg/Day) Cohort 3: VIT-2763 120 mg TID (360 mg/Day) Cohort 4: Placebo
    Number of subjects analysed
    4
    3
    3
    5
    Units: units per liter (U/L)
        arithmetic mean (standard deviation)
    -45.8 ( 47.56 )
    -24.0 ( 6.08 )
    7.7 ( 197.50 )
    47.8 ( 60.06 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline in Haemolysis Marker (Potassium)

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    End point title
    		 Mean Change From Baseline in Haemolysis Marker (Potassium) [5]
    End point description
    Mean change from Baseline in haemolysis markers was measured by potassium. This analysis was performed on ITT population. The ITT population consisted of all participants who were randomly assigned to a treatment group under Protocol Version 3.0 or higher. Here, the 'overall number of participants analyzed' (N) signifies the number of participants with evaluable data for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline and after 8 weeks of treatment
    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: Data for all applicable arms is reported for this endpoint (except Cohort2a after implementation of Protocol Version 3 and higher [as planned]).
    End point values
    		 Cohort 1: VIT-2763 60 mg BID (120 mg/Day) Cohort 2: VIT-2763 120 mg BID (240 mg/Day) Cohort 3: VIT-2763 120 mg TID (360 mg/Day) Cohort 4: Placebo
    Number of subjects analysed
    5
    5
    5
    6
    Units: millimoles per liter (mmol/L)
        arithmetic mean (standard deviation)
    0.08 ( 0.396 )
    0.08 ( 0.192 )
    -0.22 ( 0.319 )
    -0.05 ( 0.295 )
    No statistical analyses for this end point

    Secondary: Mean Change From Baseline in Haemolysis Marker (Hemoglobin and Haptoglobin)

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    End point title
    		 Mean Change From Baseline in Haemolysis Marker (Hemoglobin and Haptoglobin) [6]
    End point description
    Mean change from Baseline in haemolysis markers was measured by hemoglobin and haptoglobin. This analysis was performed on ITT population. The ITT population consisted of all participants who were randomly assigned to a treatment group under Protocol Version 3.0 or higher. Here, the 'overall number of participants analyzed' (N) signifies the number of participants with evaluable data for this outcome measure. The 'number analyzed' (n) signifies the number of participants with evaluable data for each specified category.
    End point type
    Secondary
    End point timeframe
    Baseline and after 8 weeks of treatment
    Notes
    [6] - 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: Data for all applicable arms is reported for this endpoint (except Cohort2a after implementation of Protocol Version 3 and higher [as planned]).
    End point values
    		 Cohort 1: VIT-2763 60 mg BID (120 mg/Day) Cohort 2: VIT-2763 120 mg BID (240 mg/Day) Cohort 3: VIT-2763 120 mg TID (360 mg/Day) Cohort 4: Placebo
    Number of subjects analysed
    5
    6
    5
    6
    Units: grams per liter (g/L)
    arithmetic mean (standard deviation)
        Hemoglobin (n=5,6,4,6)
    -3.400 ( 1.4748 )
    -2.067 ( 5.5142 )
    -1.575 ( 8.4017 )
    2.733 ( 9.9933 )
        Haptoglobin (n=5,6,5,6)
    0.102 ( 0.2281 )
    -0.012 ( 0.0286 )
    0.528 ( 0.8312 )
    0.000 ( 0.0000 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Treatment-emergent Adverse Events (TEAEs), TEAEs Related to IMP and by Severity of TEAEs

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    End point title
    		 Number of Participants With Treatment-emergent Adverse Events (TEAEs), TEAEs Related to IMP and by Severity of TEAEs
    End point description
    TEAEs were defined as adverse events (AEs) with an onset date later or on the same date as the first investigational medicinal product (IMP) intake. The severity grading was determined according to the Common Terminology Criteria for AEs, where the Common Terminology Criteria grades relate to severity as follows: Grade 1: Mild, Grade 2: Moderate, Grade 3: Severe, Grade 4: Life-threatening and Grade 5: Death. Analysis was performed on the safety set. The safety set consists of all randomized participants (under Protocol Version 3.0 or higher) who had taken at least one dose of IMP. The participants in the safety set were analyzed based on the treatment they received, regardless of randomization. Data are separately reported for the one participant in the Cohort 2a arm (randomized under Protocol Version 2.0).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to 12 weeks
    End point values
    		 Cohort 1: VIT-2763 60 mg BID (120 mg/Day) Cohort 2: VIT-2763 120 mg BID (240 mg/Day) Cohort 3: VIT-2763 120 mg TID (360 mg/Day) Cohort 4: Placebo Cohort 2a: VIT-2763 30 mg BID (60 mg/day)
    Number of subjects analysed
    6
    6
    6
    6
    1
    Units: participants
        Any TEAEs
    4
    4
    5
    5
    1
        TEAEs related to IMP
    0
    0
    0
    1
    0
        TEAEs with severity: Mild
    0
    3
    2
    3
    1
        TEAEs with severity: Moderate
    3
    1
    1
    2
    1
        TEAEs with severity: Severe
    1
    0
    2
    0
    0
        TEAEs with severity: Life threatening
    0
    0
    0
    0
    0
        TEAEs with severity: Death
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the first dose of study drug up to 12 weeks
    Adverse event reporting additional description
    Safety set consists of all randomized participants (under Protocol Version 3.0 or higher) who had taken at least one dose of IMP. Participants in safety set were analyzed based on the treatment they received, regardless of randomization. Data are separately reported for the 1 participant in the Cohort 2a arm (randomized under Protocol Version 2.0)
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    Cohort 1: VIT-2763 60 mg BID (120 mg/Day)
    Reporting group description
    		 Participants received VIT-2763 60 mg (2 x 30 mg capsules), orally, BID for 8 weeks.

    Reporting group title
    Cohort 2: VIT-2763 120 mg BID (240 mg/Day)
    Reporting group description
    		 Participants received VIT-2763 120 mg (2 x 60 mg capsules), orally, BID for 8 weeks.

    Reporting group title
    Cohort 3: VIT-2763 120 mg TID (360 mg/Day)
    Reporting group description
    		 Participants received VIT-2763 120 mg (2 x 60 mg capsules), orally, TID for 8 weeks.

    Reporting group title
    Cohort 4: Placebo
    Reporting group description
    		 Participants received placebo capsules, orally, BID or TID for 8 weeks.

    Reporting group title
    Cohort 2a: VIT-2763 30 mg BID (60 mg/day)
    Reporting group description
    		 One participant received VIT-2763 30 mg, orally, BID for 8 weeks under Protocol Version 2.0. However, after the implementation of Protocol Version 3.0 and higher, this participant was excluded from the efficacy analysis and only safety data were reported (as planned).

    Serious adverse events
    		 Cohort 1: VIT-2763 60 mg BID (120 mg/Day) Cohort 2: VIT-2763 120 mg BID (240 mg/Day) Cohort 3: VIT-2763 120 mg TID (360 mg/Day) Cohort 4: Placebo Cohort 2a: VIT-2763 30 mg BID (60 mg/day)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    1 / 1 (100.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Sickle cell anaemia with crisis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    1 / 1 (100.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 1 (100.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Cohort 1: VIT-2763 60 mg BID (120 mg/Day) Cohort 2: VIT-2763 120 mg BID (240 mg/Day) Cohort 3: VIT-2763 120 mg TID (360 mg/Day) Cohort 4: Placebo Cohort 2a: VIT-2763 30 mg BID (60 mg/day)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 6 (66.67%)
    4 / 6 (66.67%)
    5 / 6 (83.33%)
    5 / 6 (83.33%)
    1 / 1 (100.00%)
    Vascular disorders
    Pallor
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    Pain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Pyrexia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    0
    0
    1
    Cough
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    0
    1
    1
    Investigations
    White blood cell count increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Congenital, familial and genetic disorders
    Sickle cell disease
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    3
    1
    0
    0
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 6 (16.67%)
    2 / 6 (33.33%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    2
    0
    0
    0
    Migraine
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Blood and lymphatic system disorders
    Sickle cell anaemia with crisis
         subjects affected / exposed
    3 / 6 (50.00%)
    1 / 6 (16.67%)
    2 / 6 (33.33%)
    2 / 6 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    4
    1
    3
    4
    0
    Anaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    Thrombocytosis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Eye disorders
    Dry eye
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Eye pruritus
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Toothache
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Dry skin
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Skin depigmentation
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 6 (16.67%)
    2 / 6 (33.33%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    2
    0
    0
    0
    Back pain
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    Joint stiffness
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Joint swelling
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Musculoskeletal chest pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Musculoskeletal stiffness
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Myalgia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    Infections and infestations
    Influenza
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    Pneumonia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Viral infection
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 1 (0.00%)
         occurrences all number
    0
    0
    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
    03 Mar 2021
    • Dosing was changed from QD to BID, 12 hours apart. • The contraceptive advice was updated to state that abstinence should only be used as a contraceptive method if it is in line with the participants’ usual and preferred lifestyle and that periodic abstinence is not an acceptable method of contraception. • Inclusion Criterion 4 was added to exclude participants with low ferritin levels. • Exclusion Criterion 11 was added to exclude participants with evidence of pulmonary hypertension. • Exclusion Criteria 13 was revised to exclude participants with QTc interval is greater than (>) 450 milliseconds (msec). • Added primary and secondary endpoints. • Added the definition of the end of study. • Added criteria for stopping IP based on ferritin level and Unexpected clinically relevant worsening of complications related to SCD. • Updated the unblinding procedure in case of emergency. • Added iron chelation therapy and RBC transfusion as prohibited therapy and concomitant treatment. • Added total iron binding capacity to the list of central laboratory assays. • Specified that signs and symptoms of SCD that have unexpectedly worsened in severity or frequency or changed in nature during the study should be recorded as AEs/SAEs. • Specified the study population to be analyzed.
    09 Mar 2022
    • Updated Sponsor contacts and Medical Monitor. • Revised study design and cohorts. • Revised expected participant duration to a maximum of 16 weeks. • Updated Nonclinical Safety Data. • Updated Summary of Completed Clinical Studies to include recent data for Studies VIT-2763-101 and VIT-2763-THAL-201. • Added Justification of Safety and Dose. • Revised criterion for withdrawal of participants from IP. • Updated treatment arms and dosing and administration guidelines. • Clarified procedures for overdose of vamifeport. • Updated risks/precautions. • Updated assessments before randomization to add total serum iron, serum ferritin, serum transferrin, calculated TSAT, hepcidin, and erythropoietin (central laboratory assessment) and clarified that full haematology panel/RBC indices (to determine baseline value) was to be done before randomization. Deleted vamifeport PK sample before randomization. • Revised study procedures for administration of first dose of IP. • Deleted 12-lead ECG assessment 3 hours post-morning dose at Visits 3, 4, and 6, and added 12-lead ECG assessment at Visit 3 at 2 hours (±30 minutes) post-morning dose. • Added total serum iron, serum ferritin, serum transferrin, calculated TSAT, hepcidin, and erythropoietin (central laboratory assessment) at Visits 3 and 5. • Added collection of additional PK samples, samples for total serum iron, serum ferritin, serum transferrin, calculated TSAT, hepcidin, and erythropoietin (central laboratory assessment), and a 2-hour post-morning dose 12-lead ECG to Visit 4. • Added 2-hour post-morning dose 12-lead ECG to Visit 5 and Visit 6 and revised the timing of sample collection for total serum iron, serum ferritin, serum transferrin, calculated TSAT, hepcidin, and erythropoietin (central laboratory assessment) to 2 hours post-morning dose. • Deleted PK sample collection at Visit 6 and added collection of samples for total serum iron, serum ferritin, serum transferrin, calculated TSAT.
    16 Mar 2023
    • Updated Co-ordinating Investigator and Sponsor contacts and Medical Expert. • Added assessment of safety and tolerance of vamifeport in patients with SCD as a secondary study objective. • Clarified the administration schedule for the vamifeport 120 mg total daily dose. • Clarified Inclusion Criterion 3 and 7. • Updated Inclusion Criterion 9. • Clarified Exclusion Criterion 1. • Updated Exclusion Criterion 5,12, 18, and 19. • Clarified BID dosing schedule. • Added exploratory PK parameters. • Clarified Morning IP dose needs to be taken at the site. • Added procedure for missed or delayed dose, allowed adaptations for assessments, operational management at the sites for Visit 4, optional blood sample for SCD genotyping, and clarify hydroxyurea administration together with contraception. • Revised the purpose of 2 blood samples at the screening visit and timing of dose administration. • Revised timing of dose administration. • Added optional blood sample for SCD genotyping. • Minor editorial and document formatting revisions were made throughout.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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