Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

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

    The EU Clinical Trials Register currently displays   44356   clinical trials with a EudraCT protocol, of which   7382   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Phase 2 Open-Label Study to Evaluate Etavopivat for the Treatment of Anemia in Patients with Myelodysplastic Syndromes (MDS)

    Summary
    EudraCT number
    		 2022-001253-23
    Trial protocol
    		 FR  
    Global end of trial date
    15 Jul 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    31 Jul 2025
    First version publication date
    31 Jul 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    4202-ONC-203
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05568225
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novo Nordisk A/S
    Sponsor organisation address
    Novo Allé, Bagsvaerd, Denmark, 2880
    Public contact
    Novo Nordisk A/S, Clinical Reporting Office (2834), clinicaltrials@novonordisk.com
    Scientific contact
    Novo Nordisk A/S, Clinical Reporting Office (2834), clinicaltrials@novonordisk.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
    06 Sep 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    15 Jul 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess hematologic improvement based on an erythroid response (HI E) greater than or equal to (≥) 8 weeks duration in subjects with MDS within 24 weeks of etavopivat treatment
    Protection of trial subjects
    The trial was conducted in accordance with the Declaration of Helsinki (Oct 2013) and International Council for Harmonisation (ICH) Good Clinical Practice, including archiving of essential documents (May 1996) and European Standard (EN) International Standard (ISO) 14155 Part 1 and 2 and Food and Drug Administration (FDA) 21 Code of Federal Regulations (CFR) 312.120.
    Background therapy
    		 Etavopivat is a selective, orally bioavailable small-molecule activator of the red blood cell pyruvate kinase (PKR) enzyme. By activating PKR, etavopivat aims to increase the production of adenosine triphosphate (ATP) in red blood cells, which is hypothesized to enhance haemoglobin levels and improve erythroid response. This mechanism is particularly relevant for subjects with myelodysplastic syndromes (MDS), where anaemia and reduced red blood cell production are significant concerns. Subjects in the study were classified into groups based on their risk levels: very low risk, low risk, or intermediate risk MDS. This categorization was important for assessing the therapy's effectiveness and safety in different subject subpopulations, ensuring tailored evaluation of responses to etavopivat and its impact on anaemia management.
    Evidence for comparator
    		 Not applicable
    Actual start date of recruitment
    15 Nov 2022
    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
    United States: 5
    Country: Number of subjects enrolled
    Canada: 6
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    France: 2
    Worldwide total number of subjects
    17
    EEA total number of subjects
    6
    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)
    3
    From 65 to 84 years
    12
    85 years and over
    2

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 The trial was conducted at 10 sites in 4 countries.

    Pre-assignment
    Screening details
    		 Of the 45 subjects targeted, only 24 were screened, and 17 were enrolled into three groups: Non-transfusion dependent (NTD) N=3, Low transfusion burden (LTB) N=5, and High transfusion burden (HTB) N=9. Each subject received 400 mg of etavopivat daily.

    Period 1
    Period 1 title
    Primary treatment period
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Non-transfusion dependent
    Arm description
    		 Non transfusion dependent subjects who had received less than equal to (<=) 2 red blood cell (RBC) transfusions for anaemia within the prior 16 weeks, orally received 400 milligram (mg) of etavopivat once daily for up to 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Etavopivat
    Investigational medicinal product code
    FT-4202
    Other name
    Pharmaceutical forms
    Coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Etavopivat was administrated to subjects as two oral tablets of 200 mg each once daily (daily dosage-400 mg).

    Arm title
    		 Low transfusion burden
    Arm description
    		 Low transfusion burden subjects who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Etavopivat
    Investigational medicinal product code
    FT-4202
    Other name
    Pharmaceutical forms
    Coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Etavopivat was administrated to subjects as two oral tablets of 200 mg each once daily (daily dosage-400 mg).

    Arm title
    		 High transfusion burden
    Arm description
    		 High transfusion burden subjects who had received >= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Etavopivat
    Investigational medicinal product code
    FT-4202
    Other name
    Pharmaceutical forms
    Coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Etavopivat was administrated to subjects as two oral tablets of 200 mg each once daily (daily dosage-400 mg).

    Number of subjects in period 1
    		Non-transfusion dependent Low transfusion burden High transfusion burden
    Started
    3
    5
    9
    Efficacy evaluable set (EES)
    1
    2
    4
    Safety Population
    3
    5
    9
    Full analysis set (FAS)
    3
    5
    9
    Pharmacokinetic Population
    3
    5
    9
    Completed
    1
    2
    4
    Not completed
    2
    3
    5
         Physician decision
    -
    1
    -
         Disease progression
    -
    1
    -
         Adverse event, non-fatal
    1
    -
    -
         Termination of the study by the Sponsor
    -
    -
    1
         Withdrawal of conset
    1
    -
    4
         Lack of efficacy
    -
    1
    -
    Period 2
    Period 2 title
    Extension treatment period
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Non-transfusion dependent
    Arm description
    		 Non transfusion dependent subjects who had received <= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Etavopivat
    Investigational medicinal product code
    FT-4202
    Other name
    Pharmaceutical forms
    Coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Etavopivat was administrated to subjects as two oral tablets of 200 mg each once daily (daily dosage-400 mg).

    Arm title
    		 Low transfusion burden
    Arm description
    		 Low transfusion burden subjects who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Etavopivat
    Investigational medicinal product code
    FT-4202
    Other name
    Pharmaceutical forms
    Coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Etavopivat was administrated to subjects as two oral tablets of 200 mg each once daily (daily dosage-400 mg).

    Arm title
    		 High transfusion burden
    Arm description
    		 High transfusion burden subjects who had received >= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Etavopivat
    Investigational medicinal product code
    FT-4202
    Other name
    Pharmaceutical forms
    Coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Etavopivat was administrated to subjects as two oral tablets of 200 mg each once daily (daily dosage-400 mg).

    Number of subjects in period 2
    		Non-transfusion dependent Low transfusion burden High transfusion burden
    Started
    1
    2
    4
    Completed
    1
    2
    1
    Not completed
    0
    0
    3
         Termination of the study by the Sponsor
    -
    -
    1
         Protocol deviation
    -
    -
    1
         Lack of efficacy
    -
    -
    1

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Non-transfusion dependent
    Reporting group description
    		 Non transfusion dependent subjects who had received less than equal to (<=) 2 red blood cell (RBC) transfusions for anaemia within the prior 16 weeks, orally received 400 milligram (mg) of etavopivat once daily for up to 48 weeks.

    Reporting group title
    Low transfusion burden
    Reporting group description
    		 Low transfusion burden subjects who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.

    Reporting group title
    High transfusion burden
    Reporting group description
    		 High transfusion burden subjects who had received >= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.

    Reporting group values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden Total
    Number of subjects
    		 3 5 9 17
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0
        Adolescents (12-17 years)
    		 0 0 0 0
        Adults (18-64 years)
    		 0 0 3 3
        From 65-84 years
    		 3 4 5 12
        85 years and over
    		 0 1 1 2
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 77.3 ( 5.69 ) 80.2 ( 3.49 ) 71.2 ( 8.11 ) -
    Sex: Female, Male
    Units: Subjects
        Female
    		 0 1 3 4
        Male
    		 3 4 6 13

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Non-transfusion dependent
    Reporting group description
    		 Non transfusion dependent subjects who had received less than equal to (<=) 2 red blood cell (RBC) transfusions for anaemia within the prior 16 weeks, orally received 400 milligram (mg) of etavopivat once daily for up to 48 weeks.

    Reporting group title
    Low transfusion burden
    Reporting group description
    		 Low transfusion burden subjects who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.

    Reporting group title
    High transfusion burden
    Reporting group description
    		 High transfusion burden subjects who had received >= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
    Reporting group title
    Non-transfusion dependent
    Reporting group description
    		 Non transfusion dependent subjects who had received <= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.

    Reporting group title
    Low transfusion burden
    Reporting group description
    		 Low transfusion burden subjects who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.

    Reporting group title
    High transfusion burden
    Reporting group description
    		 High transfusion burden subjects who had received >= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.

    Primary: Proportion of subjects with Hematologic Improvement– Erythroid (HI-E) response for >=8 weeks within 24 weeks of etavopivat treatment

    		 Close Top of page
    End point title
    		 Proportion of subjects with Hematologic Improvement– Erythroid (HI-E) response for >=8 weeks within 24 weeks of etavopivat treatment [1]
    End point description
    This endpoint reported on the combined incidence of NTD, LTB and HTB in terms of (HI-E) response for >=8 weeks duration in subjects with myelodysplastic syndromes (MDS) within 24 weeks. The subjects were allocated to the following arms which were defined as: 1) NTD: >=1.5 grams per deciliter (g/dL) increase in haemoglobin (Hb) from baseline maintained >=8 consecutive weeks and no transfusion of RBC units for anemia over a continuous 8-week treatment period; 2) LTB: absence of any transfusion for >=8 consecutive weeks; and 3) HTB: reduction by >=50 percent (%) of RBC units for >=8 consecutive weeks. EES: All subjects in the FAS who had completed the week 24 response visit and who had a baseline record of the primary endpoint.
    End point type
    Primary
    End point timeframe
    From Baseline to Week 24
    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: Statistical analyses is not required for this endpoint.
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    1
    2
    4
    Units: Proportion of subjects
        number (not applicable)
    0
    50
    25
    No statistical analyses for this end point

    Secondary: Proportion of subjects with Hematologic Improvement–Erythroid (HI-E) response for =>8 weeks within 16 and 48 weeks of etavopivat

    		 Close Top of page
    End point title
    		 Proportion of subjects with Hematologic Improvement–Erythroid (HI-E) response for =>8 weeks within 16 and 48 weeks of etavopivat
    End point description
    This endpoint focused on the combined incidence of NTD, LTB and HTB subjects, evaluating HI-E lasting =>8 weeks in individuals with MDS within 16 weeks and 48 weeks. Subject's response were defined as follows: 1) NTD: an increase of =>1.5 g/dL in Hb maintained for =>8 consecutive weeks without any RBC transfusions for anemia; 2) LTB: no transfusions over =>8 consecutive weeks; and 3) HTB: a reduction of =>50% in RBC units for =>8 consecutive weeks. FAS: All subjects who signed the informed consent and received at least 1 dose of etavopivat. Data for this endpoint were not evaluable due to low subject enrolment and early termination of study.
    End point type
    Secondary
    End point timeframe
    48 weeks
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    0 [2]
    0 [3]
    0 [4]
    Units: Proportion of Subjects
        number (not applicable)
    Notes
    [2] - Data for this endpoint was not analysed due to early termination of study.
    [3] - Data for this endpoint was not analysed due to early termination of study.
    [4] - Data for this endpoint was not analysed due to early termination of study.
    No statistical analyses for this end point

    Secondary: Proportion of subjects with Hematologic Improvement– Erythroid (HI-E) response for =>16 weeks within 24 and 48 weeks of etavopivat

    		 Close Top of page
    End point title
    		 Proportion of subjects with Hematologic Improvement– Erythroid (HI-E) response for =>16 weeks within 24 and 48 weeks of etavopivat
    End point description
    This endpoint focused on the combined incidence of NDT, LTB and HTB subjects, evaluating HI-E response lasting =>16 weeks in individuals with MDS within 24 weeks and 48 weeks. Subject's response were defined as follows: 1) NTD: an increase of =>1.5 g/dL in Hb maintained for =>16 consecutive weeks without any RBC transfusions for anemia; 2) LTB: no transfusions over =>16 consecutive weeks; and 3) HTB: a reduction of =>50% in RBC units for =>16 consecutive weeks. FAS: All subjects who signed the informed consent and received at least 1 dose of etavopivat. Data for this endpoint were not evaluable due to low subject enrolment and early termination of study.
    End point type
    Secondary
    End point timeframe
    48 weeks
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    0 [5]
    0 [6]
    0 [7]
    Units: Proportion of Subjects
        number (not applicable)
    Notes
    [5] - Data for this endpoint was not analysed and calculated due to early termination of study.
    [6] - Data for this endpoint was not analysed due to early termination of study.
    [7] - Data for this endpoint was not analysed due to early termination of study.
    No statistical analyses for this end point

    Secondary: Number of all adverse events (AEs), serious adverse events (SAEs), and AEs related to etavopivat

    		 Close Top of page
    End point title
    		 Number of all adverse events (AEs), serious adverse events (SAEs), and AEs related to etavopivat
    End point description
    This endpoint was assessed for total number of AEs and SAEs in subjects, including AEs related to etavopivat. AEs are any unfavorable medical occurrences in subjects taking the medicinal product, regardless of causality. SAEs are severe AEs that result in death, are life-threatening, require hospitalization, lead to significant disability, or involve congenital anomalies. Important medical events posing risks to the subjects are also classified as SAEs. Treatment Emergent Adverse Events (TEAEs) are AEs occurring after treatment initiation, aiding in the safety assessment of etavopivat. TEAEs will be considered drug-related if assessed by the investigator as possibly related or related, or if relationship is missing. Safety population included all subjects who receive at least one dose of etavopivat (including partial dosing).
    End point type
    Secondary
    End point timeframe
    From baseline up to 48 weeks
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    3
    5
    9
    Units: Events
    number (not applicable)
        AE
    33
    33
    54
        SAE
    1
    0
    6
        AEs related to etavopivat
    0
    0
    1
        AEs possibly related etavopivat
    8
    3
    6
    No statistical analyses for this end point

    Secondary: Number of premature discontinuations, dose interruptions, and dose reductions

    		 Close Top of page
    End point title
    		 Number of premature discontinuations, dose interruptions, and dose reductions
    End point description
    The endpoint was assessed for total number of premature discontinuations, dose interruptions and dose reductions. Premature discontinuation was defined as any discontinuation prior to week 48. A dose interruption is a temporary halt in treatment due to an AE, while a dose reduction involves lowering the dosage of the drug when AEs occur. If a subject tolerates a reduced dose for 14 days, a rechallenge with the original dose may occur after a clinic visit, but any new Grade 3 or higher AEs require treatment discontinuation and consultation with the Global Medical Monitor before resuming. Safety population included all subjects who receive at least one dose of etavopivat (including partial dosing).
    End point type
    Secondary
    End point timeframe
    Within 48 weeks
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    3
    5
    9
    Units: Events
    number (not applicable)
        Premature discontinuations
    1
    0
    0
        Dose interruptions
    0
    0
    2
        Dose reductions
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Overall response rate

    		 Close Top of page
    End point title
    		 Overall response rate
    End point description
    The Overall Response Rate (ORR) is defined as the proportion of subjects who achieve a predefined level of response according to the 2006 International Working Group (IWG) criteria, assessed at each scheduled evaluation. Data for this endpoint were not evaluable due to low subject enrolment and early termination of study.
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    0 [8]
    0 [9]
    0 [10]
    Units: Proportion of subjects
        number (not applicable)
    Notes
    [8] - Data for this endpoint was not analysed due to early termination of study.
    [9] - Data for this endpoint was not analysed due to early termination of study.
    [10] - Data for this endpoint was not analysed due to early termination of study.
    No statistical analyses for this end point

    Secondary: Duration of response

    		 Close Top of page
    End point title
    		 Duration of response
    End point description
    This endpoint reported duration of response which was defined as duration of response will be summarized with quantiles based on product limit estimates (ie, Kaplan-Meier). Duration of response is defined as the time from date of first known incidence of a 2006 IWG criteria response to the most recent date that the 2006 IWG (International Working Group) criteria is not met following the initial response. If the subject has met IWG criteria throughout the period following the initial response, the subject will be censored at the latest date of end of study, loss to follow-up, or death. Data for this endpoint were not evaluable due to low subject enrolment and early termination of study.
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    0 [11]
    0 [12]
    0 [13]
    Units: Days
        number (not applicable)
    Notes
    [11] - Data for this endpoint was not analysed due to early termination of study.
    [12] - Data for this endpoint was not analysed due to early termination of study.
    [13] - Data for this endpoint was not analysed due to early termination of study.
    No statistical analyses for this end point

    Secondary: Reduction in RBC transfusions for >=8 weeks in subjects with LTD or HTB at study entry

    		 Close Top of page
    End point title
    		 Reduction in RBC transfusions for >=8 weeks in subjects with LTD or HTB at study entry [14]
    End point description
    This endpoint reports reduction in RBC transfusion by 8-week interval in subjects with LTB and HTB. EES: All subjects in the FAS who have completed the week 24 response visit and who have a baseline record of the primary endpoint. n (number analysed) = subjects with available data for a specified category.
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    Notes
    [14] - 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: Overall number of subjects analyzed signifies number of subjects evaluable for this outcome measure.
    End point values
    		 Low transfusion burden High transfusion burden
    Number of subjects analysed
    2
    4
    Units: Total RBS units
    arithmetic mean (standard deviation)
        Week 1-8 (n=2,4)
    3.0 ( 2.83 )
    5.8 ( 2.63 )
        Week 9-16 (n=2,4)
    2.5 ( 2.12 )
    6.3 ( 1.71 )
        Week 17-24 (n=2,4)
    3.5 ( 2.12 )
    7.5 ( 3.00 )
        Week 25-32 (n=2,3)
    5.5 ( 0.71 )
    7.3 ( 1.15 )
        Week 33-40 (n=2,3)
    5.0 ( 4.24 )
    6.7 ( 1.15 )
        Week 41-48 (n=2,2)
    4.5 ( 2.12 )
    7.5 ( 2.12 )
    No statistical analyses for this end point

    Secondary: Percent Change from Baseline in RBC transfusion independence for =>8 weeks in subjects with LTB or HTB at study entry

    		 Close Top of page
    End point title
    		 Percent Change from Baseline in RBC transfusion independence for =>8 weeks in subjects with LTB or HTB at study entry [15]
    End point description
    This endpoint is percent change in RBC transfusion by 8-week interval in subjects with LTB and HTB. Percent reduction from baseline in RBC transfusion burden is defined as -100×(Total RBC Units Transfused During Interval)/(Baseline RBC Units Transfused Per 8 Weeks). EES: All subjects in the FAS who have completed the week 24 response visit and who have a baseline record of the primary endpoint. n (number analysed) = subjects with available data for a specified category. 99999 signifies the standard deviation was not evaluable.
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    Notes
    [15] - 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: Overall number of subjects analyzed signifies number of subjects evaluable for this outcome measure.
    End point values
    		 Low transfusion burden High transfusion burden
    Number of subjects analysed
    2
    4
    Units: Percent change of total RBC units
    arithmetic mean (standard deviation)
        Week 1-8 (n=2,4)
    -20.83 ( 64.818 )
    68.75 ( 156.994 )
        Week 9-16 (n=2,4)
    -33.33 ( 47.140 )
    68.75 ( 98.219 )
        Week 17-24 (n=2,4)
    -4.17 ( 41.248 )
    120.83 ( 191.183 )
        Week 25-32 (n=2,3)
    58.33 ( 11.785 )
    127.78 ( 149.381 )
        Week 33-40 (n=2,3)
    33.33 ( 94.281 )
    94.44 ( 91.793 )
        Week 41-48 (n=2,2)
    25.00 ( 35.355 )
    50.00 ( 99999 )
    No statistical analyses for this end point

    Secondary: Decrease in ferritin and transferrin saturation (TSAT)

    		 Close Top of page
    End point title
    		 Decrease in ferritin and transferrin saturation (TSAT)
    End point description
    This endpoint were to report decrease in ferritin and TSAT from baseline to Week 48. Data for this endpoint were not evaluable due to low subject enrolment and early termination of study.
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    0 [16]
    0 [17]
    0 [18]
    Units: ng/mL
        number (not applicable)
    Notes
    [16] - Data for this endpoint was not analysed due to early termination of study.
    [17] - Data for this endpoint was not analysed due to early termination of study.
    [18] - Data for this endpoint was not analysed due to early termination of study.
    No statistical analyses for this end point

    Secondary: Change from baseline in neutrophils and/or platelets counts

    		 Close Top of page
    End point title
    		 Change from baseline in neutrophils and/or platelets counts
    End point description
    This endpoint reports the change from baseline in neutrophils and/or platelets counts from baseline to week 48. The safety set includes all subjects who receive at least one dose of etavopivat (including partial dosing).Here, n (number analysed) = subjects with available data for a specified category. 99999 signifies the standard deviation was not evaluable.
    End point type
    Secondary
    End point timeframe
    Baseline (week 0), week 48
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    3
    5
    9
    Units: Giga per liter (10^9/L)
    arithmetic mean (standard deviation)
        Neutrophils (n=1,2,1)
    -3.200 ( 99999 )
    -0.950 ( 0.0707 )
    -0.100 ( 99999 )
        Platelets counts (n=1,2,1)
    43.0 ( 99999 )
    -51.0 ( 205.06 )
    -8.0 ( 99999 )
    No statistical analyses for this end point

    Secondary: Decrease in iron chelation therapy

    		 Close Top of page
    End point title
    		 Decrease in iron chelation therapy
    End point description
    This endpoint was expected to report decrease of Iron chelation therapy recorded throughout the study. Data for this endpoint were not evaluable due to low subject enrolment and early termination of study.
    End point type
    Secondary
    End point timeframe
    up to 48 weeks
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    0 [19]
    0 [20]
    0 [21]
    Units: Dose unit
        arithmetic mean (standard deviation)
    ( )
    ( )
    ( )
    Notes
    [19] - Data for this endpoint was not analysed due to early termination of study.
    [20] - Data for this endpoint was not analysed due to early termination of study.
    [21] - Data for this endpoint was not analysed due to early termination of study.
    No statistical analyses for this end point

    Secondary: Overall survival

    		 Close Top of page
    End point title
    		 Overall survival
    End point description
    Overall survival is defined as the time from first dose to date of death. If the subject was alive at last contact, the end date will be censored at the latest of either end of study, loss to follow-up, or study discontinuation. Data for this endpoint were not evaluable due to low subject enrolment and early termination of study.
    End point type
    Secondary
    End point timeframe
    Within 48 weeks
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    0 [22]
    0 [23]
    0 [24]
    Units: Months
        number (not applicable)
    Notes
    [22] - Data for this endpoint was not analysed due to early termination of study.
    [23] - Data for this endpoint was not analysed due to early termination of study.
    [24] - Data for this endpoint was not analysed due to early termination of study.
    No statistical analyses for this end point

    Secondary: Etavopivat plasma concentrations

    		 Close Top of page
    End point title
    		 Etavopivat plasma concentrations
    End point description
    This endpoint reported Etavopivat plasma concentrations in order to assess the PK properties of etavopivat in subjects with MDS. PK parameters included but not limited to were: Time to maximum observed plasma concentration, area under the plasma concentration-time curve from time zero until the last quantifiable time point (AUC0-last), from time zero to infinity (AUC0-inf), for a dosing interval (AUCtau/AUC0-24).) However due to early termination of the study, all the PK parameters could not be assessed and only the plasma concentrations was assessed. The PK set includes all safety set subjects who have at least one evaluable concentration for etavopivat at a scheduled PK time point after the start of dosing. Here, n (number analysed) = subjects with available data for a specified category. 99999 signifies the arithmetic mean and standard deviation was not evaluable.
    End point type
    Secondary
    End point timeframe
    Weeks 1 and 4 pre-dose, post-dose 1, 2, 4, 6 hours.Week 2 and EOT (week 48):pre-dose, post-dose 1, 2 hours
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    3
    5
    9
    Units: Nanograms per milliliter (ng/mL)
    arithmetic mean (standard deviation)
        Week 1 predose (n=3,4,9)
    0 ( 99999 )
    0 ( 99999 )
    0 ( 99999 )
        Week 1 post-dose 1 hour (n=3,5,9)
    379.77 ( 292.748 )
    904.80 ( 521.000 )
    1016.78 ( 794.503 )
        Week 1 post-dose 2 hour (n=3,5,9)
    751.67 ( 393.465 )
    579.60 ( 403.593 )
    519.11 ( 273.776 )
        Week 1 post-dose 4 hour (n=2,5,9)
    274.50 ( 86.974 )
    203.80 ( 134.884 )
    165.16 ( 64.895 )
        Week 1 post-dose 6 hour (n=1,5,7)
    63.50 ( 99999 )
    101.02 ( 67.693 )
    83.74 ( 40.170 )
        Week 2 pre-dose (n=3,4,9)
    27.20 ( 17.843 )
    19.08 ( 9.037 )
    19.20 ( 12.240 )
        Week 2 post-dose 1 hour (n=3,5,9)
    1156.00 ( 371.198 )
    899.40 ( 442.555 )
    1076.44 ( 693.907 )
        Week 2 post-dose 2 hour (n=3,4,9)
    927.00 ( 310.140 )
    1024.00 ( 530.102 )
    661.78 ( 392.238 )
        Week 4 predose (n=2,5,9)
    17.04 ( 11.540 )
    24.34 ( 23.333 )
    20.26 ( 9.289 )
        Week 4 post-dose 1 hour (n=2,4,9)
    1050.00 ( 28.284 )
    386.90 ( 214.249 )
    913.20 ( 1013.207 )
        Week 4 post-dose 2 hour (n=2,5,9)
    526.00 ( 134.350 )
    453.40 ( 179.140 )
    678.49 ( 379.918 )
        Week 4 post-dose 4 hour (n=2,4,9)
    151.50 ( 12.021 )
    383.73 ( 261.103 )
    297.90 ( 282.646 )
        Week 4 post-dose 6 hour (n=2,3,9)
    60.75 ( 11.526 )
    148.13 ( 113.650 )
    121.34 ( 115.306 )
        End of treatment pre-dose (n=0,2,1)
    99999 ( 99999 )
    21.60 ( 5.798 )
    24.20 ( 99999 )
        End of treatment post-dose 1 hours (n=0,1,1)
    99999 ( 99999 )
    1620.00 ( 99999 )
    1680.00 ( 99999 )
        End of treatment post-dose 2 hours (n=0,1,1)
    99999 ( 99999 )
    571.00 ( 99999 )
    815.00 ( 99999 )
    No statistical analyses for this end point

    Secondary: RBC 2,3-diphosphoglycerate (2,3-DPG) and adenosine triphosphate (ATP) levels over time

    		 Close Top of page
    End point title
    		 RBC 2,3-diphosphoglycerate (2,3-DPG) and adenosine triphosphate (ATP) levels over time
    End point description
    Etavopivat plasma concentrations were collected in order to assess the pharmacodynamic (PD) properties of etavopivat in subjects with MDS. Data for this endpoint were not evaluable due to low subject enrolment and early termination of study.
    End point type
    Secondary
    End point timeframe
    Within 48 weeks
    End point values
    		 Non-transfusion dependent Low transfusion burden High transfusion burden
    Number of subjects analysed
    0 [25]
    0 [26]
    0 [27]
    Units: Micrograms per milliliter
        number (not applicable)
    Notes
    [25] - Data for this endpoint was not analysed due to early termination of study.
    [26] - Data for this endpoint was not analysed due to early termination of study.
    [27] - Data for this endpoint was not analysed due to early termination of study.
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Up to 48 weeks
    Adverse event reporting additional description
    All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any AE that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all subjects who receive at least one dose of etavopivat (including partial dosing).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25
    Reporting groups
    Reporting group title
    Non-transfusion dependent
    Reporting group description
    		 Non transfusion dependent participants who had received less than equal to <= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.

    Reporting group title
    High transfusion burden
    Reporting group description
    		 High transfusion burden participants who had received greater than equal >= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.

    Reporting group title
    Low transfusion burden
    Reporting group description
    		 Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.

    Serious adverse events
    		 Non-transfusion dependent High transfusion burden Low transfusion burden
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 3 (33.33%)
    3 / 9 (33.33%)
    0 / 5 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Post procedural haemorrhage
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hip fracture
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Arterial rupture
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Peripheral arterial occlusive disease
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac failure
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Staphylococcal infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Non-transfusion dependent High transfusion burden Low transfusion burden
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    9 / 9 (100.00%)
    5 / 5 (100.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Hypotension
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    General disorders and administration site conditions
    Chest discomfort
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Asthenia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Chills
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Fatigue
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    2
    1
    0
    Mucosal dryness
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Influenza like illness
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    2 / 3 (66.67%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    3
    1
    0
    Cough
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    1
    1
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    2
    0
    1
    Depression
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Insomnia
         subjects affected / exposed
    1 / 3 (33.33%)
    2 / 9 (22.22%)
    0 / 5 (0.00%)
         occurrences all number
    1
    3
    0
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Blood bilirubin increased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    3
    0
    Lymphocyte count decreased
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 9 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    3
    0
    0
    Lipase increased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Neutrophil count decreased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    2
    Platelet count decreased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    10
    Troponin I increased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    White blood cell count decreased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    4
    0
    0
    Injury, poisoning and procedural complications
    Hip fracture
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Transfusion reaction
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Cardiac disorders
    Cardiac failure
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    0
    1
    Atrioventricular block second degree
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Palpitations
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 9 (22.22%)
    0 / 5 (0.00%)
         occurrences all number
    0
    3
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    3
    0
    0
    Neutropenia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    0
    Thrombocytopenia
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 9 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    4
    0
    0
    Ear and labyrinth disorders
    External ear inflammation
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Vertigo
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 9 (22.22%)
    0 / 5 (0.00%)
         occurrences all number
    0
    2
    0
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Abdominal pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Haemorrhoids
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Dysphagia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Diarrhoea
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 9 (22.22%)
    1 / 5 (20.00%)
         occurrences all number
    0
    3
    1
    Nausea
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 9 (22.22%)
    1 / 5 (20.00%)
         occurrences all number
    0
    2
    1
    Vomiting
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    1
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Musculoskeletal and connective tissue disorders
    Bone pain
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Arthralgia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Neck pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Muscular weakness
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Pain in extremity
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    COVID-19
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    1
    Herpes zoster
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Eye infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Ear infection
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Lip infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Urinary tract infection
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    2
    1
    0
    Skin infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    0
    1
    Rhinitis
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Pneumonia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    2 / 5 (40.00%)
         occurrences all number
    0
    0
    2
    Metabolism and nutrition disorders
    Haemosiderosis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Decreased appetite
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Metabolic acidosis
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Iron overload
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Hypervolaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Hyperuricaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Vitamin B12 deficiency
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    11 Apr 2022
    Version 1.0: First protocol version
    15 Jun 2022
    Version 2.0: Protocol version 1.0 was not implemented.
    31 Jul 2023
    Version 3.0: 1. Protocol version updated to adjust the treatment period timeframes into a 24-week Primary Treatment period, a 24-week Extension Treatment period, and a Survival Follow-up period. 2. A ‘futility assessment’ was added. 3. Schedule of Events tables and Appendices were updated as needed to reflect the above changes to the protocol. 4. Safety text has been added throughout the document to reflect the Novo Nordisk Safety Surveillance practice.

    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.
    The predefined Quality Tolerance Limit (QTL) that required 5 or more subjects to complete 16 weeks of treatment. However, 7 subjects did not fulfill this requirement, leading to the early termination.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sun Aug 24 08:17:26 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA