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    Clinical Trial Results:
    Randomized, double-blind, placebo-controlled, 3-arm, 36 weeks parallel-group study to evaluate the safety and tolerability of ORY-2001 in patients with Relapsing-Remitting Multiple Sclerosis (RRMS) and Secondary Progressive Multiple Sclerosis (SPMS). SATEEN Study

    Summary
    EudraCT number
    		 2017-002838-23
    Trial protocol
    		 ES  
    Global end of trial date
    01 Sep 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    06 Dec 2023
    First version publication date
    06 Dec 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CL02-ORY-2001MS
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Oryzon Genomics S. A.
    Sponsor organisation address
    Carrer de Sant Ferran, 74, CORNELLA DE LLOBREGAT, Spain, 08940
    Public contact
    Douglas V. Faller, Oryzon Genomics S.A., 34 93 515 1313, dfaller@oryzon.com
    Scientific contact
    Douglas V. Faller, Oryzon Genomics S.A., 34 93 515 13 13, dfaller@oryzon.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
    14 Dec 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    01 Sep 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Sep 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of two doses of vafidemstat (ORY-2001) compared to placebo in multiple sclerosis (MS) patients.
    Protection of trial subjects
    In accordance with European Union RGPD 2016/679 of 27 April, 2016 the data were processed in accordance with the specifications outlined by the local law to ensure that requirements regarding personal data protection are met. If an external organization processed data on behalf of Oryzon, a contractual procedure was signed between Oryzon and the external organization to ensure compliance with the above-mentioned legislation. If applicable, the participation of patients in this study was reported to the appropriate local data protection agencies, in accordance with European Union RGPD 2016/679 of 27 April 2016 and Country-specific guidelines and laws (Spanish Organic Law 3/2018 of 5 December). Patients were free to discontinue their participation in the study at any time. Withdrawal from the study did not affect or prejudice the patient’s further treatment. Patients could be withdrawn from study treatment and assessments at any time, if deemed necessary by the Investigator. In the event that a patient was to withdraw consent to participate in the study, the patient was asked, if possible, to return for a follow-up visit to document his clinical status. The data of this visit were only recorded in the patient's medical history. An independent Data Monitoring Committee (DMC) reviewed unblinded safety data throughout the study.
    Background therapy
    		 -
    Evidence for comparator
    		 Placebo was the comparator in the Treatment period.
    Actual start date of recruitment
    24 Jan 2018
    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
    Spain: 18
    Worldwide total number of subjects
    18
    EEA total number of subjects
    18
    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)
    18
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 8 centers recruited patients in this study. A total of 25 patients were screened and 18 patients were randomized and treated. During the Treatment Period, 7 patients were randomized to and received vafidemstat 0.6 mg, 7 were randomized to and received vafidemstat 1.2 mg and 4 were randomized to and received placebo.

    Pre-assignment
    Screening details
    		 A Screening Period for up to 4 weeks before the Treatment Period was allowed. HIV antibody test and hepatitis testing including HBV surface antigen and HCV antibody were performed at the screening visit (Visit 0) for all patients. These tests were only used to determine patient eligibility for inclusion in the study.

    Period 1
    Period 1 title
    Treatment Period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Subject
    Blinding implementation details
    To guarantee double-blind conditions, all the drugs were presented in identical capsules consisting of special opaque material for clinical studies, and the patients took the same number of capsules daily (one) during 5 consecutive days(total of 5 capsules) followed by 2 days off. The study randomisation was only to be broken for valid medical or safety reasons, for example, a serious adverse event.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Vafidemstat 0.6 mg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vafidemstat 0.6 mg
    Investigational medicinal product code
    Other name
    ORY-2001
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Swedish orange colored size 3 capsules (hydroxypropyl methylcellulose shells) were loaded directly with 0.6 mg vafidemstat (ORY-2001) drug substance without addition of excipients, and by means of Xcelodose® filling technology. Study medication was to be taken orally, after overnight fasting conditions, early in the morning, five times per week, following a 5 days on / 2 days off schedule (i.e., fiw, once daily from Monday to Friday).

    Arm title
    		 Vafidemstat 1.2 mg
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vafidemstat 1.2 mg
    Investigational medicinal product code
    Other name
    ORY-2001
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Swedish orange colored size 3 capsules (hydroxypropyl methylcellulose shells) were loaded directly with 1.2 mg vafidemstat (ORY-2001) drug substance without addition of excipients, and by means of Xcelodose® filling technology. Study medication was to be taken orally, after overnight fasting conditions, early in the morning, five times per week, following a 5 days on / 2 days off schedule (i.e., fiw, once daily from Monday to Friday).

    Arm title
    		 Placebo
    Arm description
    		 -
    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
    Swedish orange colored size 3 capsules (hydroxypropyl methylcellulose shells) were loaded directly with cellulose microcristalline by means of Xcelodose® filling technology. Study medication was to be taken orally, after overnight fasting conditions, early in the morning, five times per week, following a 5 days on / 2 days off schedule (i.e., fiw, once daily from Monday to Friday).

    Number of subjects in period 1
    		Vafidemstat 0.6 mg Vafidemstat 1.2 mg Placebo
    Started
    7
    7
    4
    Completed
    5
    6
    3
    Not completed
    2
    1
    1
         Withdrawal before W36 + No valid MRI assessment
    1
    -
    -
         No valid MRI assessment at W36
    1
    1
    -
         Any eligibility criteria not fulfilled + Major PD
    -
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Vafidemstat 0.6 mg
    Reporting group description
    		 -

    Reporting group title
    Vafidemstat 1.2 mg
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Reporting group values
    		 Vafidemstat 0.6 mg Vafidemstat 1.2 mg Placebo Total
    Number of subjects
    		 7 7 4 18
    Age categorical
    Only the FAS population results are available
    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)
    		 7 7 4 18
        From 65-84 years
    		 0 0 0 0
        85 years and over
    		 0 0 0 0
    Age continuous
    Only the FAS population results are available
    Units: years
        arithmetic mean (standard deviation)
    		 46.7 ( 10.44 ) 50.1 ( 7.76 ) 49.4 ( 9.02 ) -
    Gender categorical
    Units: Subjects
        Female
    		 5 4 4 13
        Male
    		 2 3 0 5

    End points

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    End points reporting groups
    Reporting group title
    Vafidemstat 0.6 mg
    Reporting group description
    		 -

    Reporting group title
    Vafidemstat 1.2 mg
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Primary: Number of TEAEs

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    End point title
    		 Number of TEAEs [1]
    End point description
    End point type
    Primary
    End point timeframe
    Adverse events (AEs) were recorded during the study period from the signing of informed consent to the completion of the follow-up period (Visit FU2). Treatment emergent AEs (TEAEs) were defined as AEs which started after first intake of study drug.
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The primary endpoints were assessments of safety and no statistical testing was performed.
    End point values
    		 Vafidemstat 0.6 mg Vafidemstat 1.2 mg Placebo
    Number of subjects analysed
    7
    7
    4
    Units: Units
        Overall number of TEAEs
    17
    21
    17
    No statistical analyses for this end point

    Primary: Number of Serious TEAEs

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    End point title
    		 Number of Serious TEAEs [2]
    End point description
    End point type
    Primary
    End point timeframe
    Adverse events (AEs) were recorded during the study period from the signing of informed consent to the completion of the follow-up period (Visit FU2). Treatment emergent AEs (TEAEs) were defined as AEs which started after first intake of study drug.
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The primary endpoints were assessments of safety and no statistical testing was performed.
    End point values
    		 Vafidemstat 0.6 mg Vafidemstat 1.2 mg Placebo
    Number of subjects analysed
    7
    7
    4
    Units: Number
        Overall number of serious TEAEs
    0
    0
    0
    No statistical analyses for this end point

    Primary: Incidence of TEAEs

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    End point title
    		 Incidence of TEAEs [3]
    End point description
    End point type
    Primary
    End point timeframe
    Adverse events (AEs) were recorded during the study period from the signing of informed consent to the completion of the follow-up period (Visit FU2). Treatment emergent AEs (TEAEs) were defined as AEs which started after first intake of study drug.
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The primary endpoints were assessments of safety and no statistical testing was performed.
    End point values
    		 Vafidemstat 0.6 mg Vafidemstat 1.2 mg Placebo
    Number of subjects analysed
    7
    7
    4
    Units: unit(s)
    arithmetic mean (standard deviation)
        TEAEs per subject-years of follow-up
    3.64 ( 2.45 )
    4.44 ( 2.67 )
    6.12 ( 4.13 )
    No statistical analyses for this end point

    Primary: Use of concomitant medication (most used)

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    End point title
    		 Use of concomitant medication (most used) [4]
    End point description
    Most used concomitant medications.
    End point type
    Primary
    End point timeframe
    Concomitant medication at week 36.
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The primary endpoints were assessments of safety and no statistical testing was performed.
    End point values
    		 Vafidemstat 0.6 mg Vafidemstat 1.2 mg Placebo
    Number of subjects analysed
    7
    7
    4
    Units: Number
        Anti-inflammatory and Antirheumatic, Non-Steroids
    4
    5
    2
        Other Analgesics And Antipyretics
    2
    3
    3
        Antidepressants
    1
    2
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were recorded during the study period from the signing of informed consent to the completion of the follow-up period (Visit FU2).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    21.0
    Reporting groups
    Reporting group title
    Vafidemstat 0.6 mg
    Reporting group description
    		 -

    Reporting group title
    Vafidemstat 1.2 mg
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Serious adverse events
    		 Vafidemstat 0.6 mg Vafidemstat 1.2 mg Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 7 (0.00%)
    0 / 4 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Vafidemstat 0.6 mg Vafidemstat 1.2 mg Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 7 (85.71%)
    7 / 7 (100.00%)
    3 / 4 (75.00%)
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 7 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    3
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 7 (0.00%)
    3 / 4 (75.00%)
         occurrences all number
    1
    0
    3
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 7 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    0
    0
    2
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 7 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    2
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 7 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    1
    0
    2
    Constipation
         subjects affected / exposed
    2 / 7 (28.57%)
    1 / 7 (14.29%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    0
    Nausea
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 7 (28.57%)
    0 / 4 (0.00%)
         occurrences all number
    0
    2
    0
    Abdominal pain upper
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 7 (14.29%)
    0 / 4 (0.00%)
         occurrences all number
    1
    1
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 7 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    2
    Renal and urinary disorders
    Urinary incontinence
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 7 (14.29%)
    0 / 4 (0.00%)
         occurrences all number
    0
    2
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 7 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    1
    0
    3
    Influenza
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 7 (28.57%)
    0 / 4 (0.00%)
         occurrences all number
    0
    2
    0
    Urinary tract infection
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 7 (14.29%)
    1 / 4 (25.00%)
         occurrences all number
    1
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    14 Dec 2017
    This amendment was regarded as “substantial” as it involved the following objectives: • Clarify and further detail the criteria for clinical observations that would require re-consent of the patient in the event that they appear for the first time, and a withdrawal of the patient in the event that they appear for the second time. • Clarify the duration of the open extension period of the trial. • Correct and / or clarify small inconsistencies between different sections of the protocol and small errors detected in the text. • Modify the information sheet for patients, consequently to the aforementioned changes in the protocol.
    01 Mar 2018
    This amendment was regarded as “substantial” since it involved the following objectives: • Specify some of the inclusion and exclusion criteria for patients in the trial. • Specify the type of antidepressant drugs not allowed in the trial. • Include blood and urine tests in the selection visit, which by mistake was not incorporated into the protocol as it was done for the rest of the follow-up visits. • Determine tuberculosis tests according to routine clinical practice. • Modify the calendar of meetings of the Data Monitoring Committee. • Administrative changes and correction of small errors in the text. • Modify the information sheet for patients, consequently to the aforementioned changes in the protocol.
    09 Jul 2019
    This amendment was considered as “substantial” since the following objectives were involved: • Add a second period of study extension in patients with Secondary Progressive Multiple Sclerosis (SPMS) • Correction of small errors in the text. • New specific patient information sheet for the second extension phase of the study in patients with SPMS.
    16 Oct 2019
    This amendment was considered as “substantial” since the following objectives were involved: • Clarify the period of time in which the concomitant medications established in the protocol would be prohibited in order to avoid possible drug interactions that may exist between vafidemstat and the prohibited concomitant medication. • Management of the patient in case of need for treatment with concomitant prohibited medication during the safety follow-up period.
    12 Nov 2019
    The approval of a substantial amendment to the protocol was requested to include the performance of a pharmacogenetic sub-study in order to evaluate whether individual genetic variability (polymorphisms and associated phenotypes) in the main genes related to ADME pathways in humans was associated with the pharmacokinetic response to vafidemstat.

    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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