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   44409   clinical trials with a EudraCT protocol, of which   7418   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, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Dose Study to Evaluate the Efficacy and Safety of Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and Chronic Immune Thrombocytopenia (ITP)

    Summary
    EudraCT number
    		 2018-003329-26
    Trial protocol
    		 PL   ES   GR  
    Global end of trial date
    10 Jan 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    17 Sep 2026
    First version publication date
    17 Sep 2026
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    OSCO-P2101
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Oscotec Inc.
    Sponsor organisation address
    Korea Bio-Park, Building A, 9th Floor 700 Daewangpangyo-ro, Seongnam-si, Korea, Republic of, 13488
    Public contact
    Sungsil Lee, Oscotec Inc., 82 31 628 7627, sslee@oscotec.com
    Scientific contact
    Sungsil Lee, Oscotec Inc., 82 31 628 7627, sslee@oscotec.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
    25 Jul 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    10 Jan 2023
    Global end of trial reached?
    Yes
    Global end of trial date
    10 Jan 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and efficacy on the primary endpoint (platelet response) of select (200 mg BID and 400 mg BID) doses of SKI-O-703 compared to placebo in patients with persistent and chronic ITP, with a platelet count <30,000/μL on 2 occasions at least 7 days apart with the confirmatory count on the first day of treatment.
    Protection of trial subjects
    The study was performed in accordance with the ethical principles that have their origin in the Declaration of Helsinki, ICH GCP, the protocol, and all applicable regulations.
    Background therapy
    		 Subjects were allowed to receive stable doses of background medications in line with the current standard of care. The background medications allowed included corticosteroids (<20 mg prednisone equivalent per day) and immunosuppressive drugs such as azathioprine (≤50 mg twice daily), mycophenolate mofetil (≤500 mg twice daily), and cyclosporine (≤100 mg twice daily). The dose of such background medications was fixed for at least 2 weeks before Day 1 and remained unchanged until 12 weeks of treatment was completed or unless rescue therapy was required.
    Evidence for comparator
    		 This study is placebo controlled and placebo is used for comparator.
    Actual start date of recruitment
    06 Dec 2019
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Efficacy, Safety
    Long term follow-up duration
    		 1 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Poland: 12
    Country: Number of subjects enrolled
    Spain: 16
    Country: Number of subjects enrolled
    Greece: 20
    Country: Number of subjects enrolled
    Korea, Republic of: 11
    Country: Number of subjects enrolled
    United States: 1
    Worldwide total number of subjects
    60
    EEA total number of subjects
    48
    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)
    39
    From 65 to 84 years
    19
    85 years and over
    2

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 A total of 61 subjects were randomly assigned to receive the study drugs, of whom 60 subjects were included in the ITT set and the safety set each. Note: 1 subject was randomly assigned to the 400 mg BID group but did not receive any dose of study drug as the subject was withdrawn due to noncompliance with the protocol.

    Pre-assignment
    Screening details
    		 The study included up to 4 weeks of screening period. A total of 85 subjects were screened, of whom 24 subjects were screen failures.

    Period 1
    Period 1 title
    12-Week Treatment Period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 SKI-O-703 200 mg BID
    Arm description
    		 2 capsules of 100 mg SKI-O-703 BID (twice per day)
    Arm type
    		 Experimental

    Investigational medicinal product name
    SKI-O-703 200 mg BID
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    200 mg BID group: 2 capsules of 100 mg SKI-O-703 + 2 capsules of placebo

    Arm title
    		 SKI-O-703 400 mg BID
    Arm description
    		 4 capsules of 100 mg SKI-O-703 BID (twice per day)
    Arm type
    		 Experimental

    Investigational medicinal product name
    SKI-O-703 (100 mg)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    400 mg BID group: 4 capsules of 100 mg SKI-O-703 + 0 capsule of placebo

    Arm title
    		 Placebo
    Arm description
    		 4 capsules of placebo BID (twice per day)
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo group: 4 capsules of placebo (contained only microcrystalline cellulose and magnesium stearate)

    Number of subjects in period 1
    		SKI-O-703 200 mg BID SKI-O-703 400 mg BID Placebo
    Started
    26
    22
    12
    Completed
    22
    20
    10
    Not completed
    4
    2
    2
         Subject withdrew consent
    -
    1
    -
         Adverse event, non-fatal
    2
    -
    1
         Other
    1
    -
    -
         Investigator decision
    1
    1
    1

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    SKI-O-703 200 mg BID
    Reporting group description
    		 2 capsules of 100 mg SKI-O-703 BID (twice per day)

    Reporting group title
    SKI-O-703 400 mg BID
    Reporting group description
    		 4 capsules of 100 mg SKI-O-703 BID (twice per day)

    Reporting group title
    Placebo
    Reporting group description
    		 4 capsules of placebo BID (twice per day)

    Reporting group values
    		 SKI-O-703 200 mg BID SKI-O-703 400 mg BID Placebo Total
    Number of subjects
    		 26 22 12 60
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 18 16 5 39
        From 65-84 years
    		 8 6 5 19
        85 years and over
    		 0 0 2 2
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 56.5 ( 14.98 ) 54.3 ( 16.30 ) 61.1 ( 22.24 ) -
    Gender categorical
    Units: Subjects
        Female
    		 13 16 5 34
        Male
    		 13 6 7 26

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    SKI-O-703 200 mg BID
    Reporting group description
    		 2 capsules of 100 mg SKI-O-703 BID (twice per day)

    Reporting group title
    SKI-O-703 400 mg BID
    Reporting group description
    		 4 capsules of 100 mg SKI-O-703 BID (twice per day)

    Reporting group title
    Placebo
    Reporting group description
    		 4 capsules of placebo BID (twice per day)

    Primary: Platelet count >= 30,000/µL and doubling the baseline (average of 2 previous counts)

    		 Close Top of page
    End point title
    		 Platelet count >= 30,000/µL and doubling the baseline (average of 2 previous counts)
    End point description
    To evaluate the safety and efficacy on the primary endpoint (platelet response) of selected (200 mg BID and 400 mg BID) doses of SKI-O-703 compared to placebo in subjects with persistent and chronic ITP, with a platelet count <30,000/μL on 2 occasions at least 7 days apart with the confirmatory count being taken during screening
    End point type
    Primary
    End point timeframe
    Up to week 12
    End point values
    		 SKI-O-703 200 mg BID SKI-O-703 400 mg BID Placebo
    Number of subjects analysed
    26
    22
    12
    Units: Count of Participants
        number (not applicable)
    12
    14
    4
    Statistical analysis title
    		 Fisher’s Exact test
    Comparison groups
    SKI-O-703 200 mg BID v Placebo
    Number of subjects included in analysis
    38
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.504
    Method
    		 Fisher exact
    Parameter type
    		 ORR difference
    Point estimate
    0.13
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.225
         upper limit
    		 0.433

    Primary: Platelet count >= 30,000/µL and doubling the baseline (average of 2 previous counts)

    		 Close Top of page
    End point title
    		 Platelet count >= 30,000/µL and doubling the baseline (average of 2 previous counts)
    End point description
    End point type
    Primary
    End point timeframe
    Up to week 12
    End point values
    		 SKI-O-703 200 mg BID SKI-O-703 400 mg BID Placebo
    Number of subjects analysed
    26
    22
    12
    Units: percent
    arithmetic mean (confidence interval 95%)
        ORR=overall platelet response rate
    46.2 (26.59 to 66.63)
    63.6 (40.66 to 82.80)
    33.3 (9.92 to 65.11)
    Statistical analysis title
    		 Fisher’s Exact test
    Comparison groups
    SKI-O-703 400 mg BID v Placebo
    Number of subjects included in analysis
    34
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.151
    Method
    		 Fisher exact
    Parameter type
    		 ORR difference
    Point estimate
    0.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.061
         upper limit
    		 0.607

    Primary: Platelet Response Rate based on Rescue Medication with 28-day effective window (NRI)

    		 Close Top of page
    End point title
    		 Platelet Response Rate based on Rescue Medication with 28-day effective window (NRI)
    End point description
    End point type
    Primary
    End point timeframe
    Up to week 12
    End point values
    		 SKI-O-703 200 mg BID SKI-O-703 400 mg BID Placebo
    Number of subjects analysed
    26
    22
    12
    Units: Count of Participants
        number (not applicable)
    12
    14
    5
    Statistical analysis title
    		 Fisher's Exact test
    Comparison groups
    SKI-O-703 200 mg BID v Placebo
    Number of subjects included in analysis
    38
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 > 0.999
    Method
    		 Fisher exact
    Parameter type
    		 ORR difference
    Point estimate
    0.04
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.31
         upper limit
    		 0.369

    Primary: Platelet Response Rate based on Rescue Medication with 28-day effective window (NRI)

    		 Close Top of page
    End point title
    		 Platelet Response Rate based on Rescue Medication with 28-day effective window (NRI)
    End point description
    End point type
    Primary
    End point timeframe
    Up to week 12
    End point values
    		 SKI-O-703 200 mg BID SKI-O-703 400 mg BID Placebo
    Number of subjects analysed
    26
    22
    12
    Units: percent
        arithmetic mean (confidence interval 95%)
    46.2 (26.59 to 66.63)
    63.6 (40.66 to 82.80)
    41.7 (15.17 to 72.33)
    Statistical analysis title
    		 Fisher's Exact test
    Comparison groups
    SKI-O-703 400 mg BID v Placebo
    Number of subjects included in analysis
    34
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.288
    Method
    		 Fisher exact
    Parameter type
    		 ORR difference
    Point estimate
    0.22
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.145
         upper limit
    		 0.54

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Up to 16 weeks
    Adverse event reporting additional description
    Adverse events were assessed and reported from the time the subject signed the ICF until end of study visit (Week 16).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    21.0
    Reporting groups
    Reporting group title
    SKI-O-703 200 mg BID
    Reporting group description
    		 -

    Reporting group title
    SKI-O-703 400 mg BID
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Serious adverse events
    		 SKI-O-703 200 mg BID SKI-O-703 400 mg BID Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 26 (0.00%)
    2 / 22 (9.09%)
    3 / 12 (25.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    Neutrophil count decreased
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 22 (4.55%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Guillain-Barre syndrome
         subjects affected / exposed
    0 / 26 (0.00%)
    0 / 22 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Thrombocytopenia
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 22 (4.55%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 26 (0.00%)
    0 / 22 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Coronavirus infection
         subjects affected / exposed
    0 / 26 (0.00%)
    0 / 22 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         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
    		 SKI-O-703 200 mg BID SKI-O-703 400 mg BID Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 26 (57.69%)
    17 / 22 (77.27%)
    8 / 12 (66.67%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    2 / 26 (7.69%)
    2 / 22 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    2
    2
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 22 (4.55%)
    0 / 12 (0.00%)
         occurrences all number
    2
    1
    0
    Vascular disorders
    Haematoma
         subjects affected / exposed
    1 / 26 (3.85%)
    1 / 22 (4.55%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 22 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    2
    0
    2
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 26 (0.00%)
    2 / 22 (9.09%)
    2 / 12 (16.67%)
         occurrences all number
    0
    3
    2
    Gastrointestinal disorders
    Gingival bleeding
         subjects affected / exposed
    0 / 26 (0.00%)
    3 / 22 (13.64%)
    1 / 12 (8.33%)
         occurrences all number
    0
    3
    1
    Nausea
         subjects affected / exposed
    1 / 26 (3.85%)
    2 / 22 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    2
    0
    Diarrhoea
         subjects affected / exposed
    0 / 26 (0.00%)
    2 / 22 (9.09%)
    2 / 12 (16.67%)
         occurrences all number
    0
    3
    2
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    1 / 26 (3.85%)
    4 / 22 (18.18%)
    0 / 12 (0.00%)
         occurrences all number
    6
    6
    0
    Skin and subcutaneous tissue disorders
    Petechiae
         subjects affected / exposed
    4 / 26 (15.38%)
    3 / 22 (13.64%)
    3 / 12 (25.00%)
         occurrences all number
    4
    3
    5
    Infections and infestations
    Coronavirus infection
         subjects affected / exposed
    3 / 26 (11.54%)
    0 / 22 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    3
    0
    1
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    0 / 26 (0.00%)
    2 / 22 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    0
    2
    0

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 Apr 2019
    Protocol Amendment 1 Version 2.0, dated 03 Apr 2019
    30 Apr 2019
    Protocol Amendment 2 Version 3.0, dated 30 Apr 2019
    22 Apr 2020
    Protocol Amendment 3 Version 4.0, dated 22 Apr 2020

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat Sep 19 16:16:41 CEST 2026 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA