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    Clinical Trial Results:
    Double-Blind, Randomized, 8-Week Placebo-Controlled, and 16-Week Open Label Extension Study Investigating the Safety, Pharmacokinetics and Pharmacodynamics of SAR100842 Given Orally to Patients with Diffuse Cutaneous Systemic Sclerosis

    Summary
    EudraCT number
    2012-001369-34
    Trial protocol
    GB   DE   IT  
    Global end of trial date
    02 Apr 2014

    Results information
    Results version number
    v1(current)
    This version publication date
    27 Apr 2016
    First version publication date
    28 Jun 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ACT12339
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01651143
    WHO universal trial number (UTN)
    U1111-1127-2854
    Sponsors
    Sponsor organisation name
    Sanofi aventis recherche & développement
    Sponsor organisation address
    1 avenue Pierre, Brossolette, Chilly-Mazarin, France, 91380
    Public contact
    Trial Transparency Team, Sanofi-aventis recherche & développement, Contact-US@sanofi.com
    Scientific contact
    Trial Transparency Team, Sanofi-aventis recherche & développement, Contact-US@sanofi.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
    28 May 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Apr 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate safety and tolerability of 8-week oral administration of SAR100842 in subjects with diffuse cutaneous systemic sclerosis/scleroderma (dcSSc).
    Protection of trial subjects
    Subjects were fully informed of all pertinent aspects of the clinical trial as well as the possibility to discontinue at any time in language and terms appropriate for the subject and considering the local culture. During the course of the trial, subjects were provided with individual subject cards indicating the nature of the trial the subject is participating, contact details and any information needed in the event of a medical emergency. Collected personal data and human biological samples were processed in compliance with the Sanofi-Aventis Group Personal Data Protection Charter ensuring that the Group abides by the laws governing personal data protection in force in all countries in which it operates.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    04 Jan 2013
    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
    France: 6
    Country: Number of subjects enrolled
    United Kingdom: 5
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Switzerland: 4
    Country: Number of subjects enrolled
    United States: 16
    Worldwide total number of subjects
    32
    EEA total number of subjects
    12
    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)
    29
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study was conducted at 12 sites in 5 countries. A total of 48 subjects were screened between 4 January 2013 and 12 September 2013.

    Pre-assignment
    Screening details
    Of 48 screened subjects, 16 were screen failure and 32 subjects were randomized and treated.

    Period 1
    Period 1 title
    Core Part
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo-Core Part
    Arm description
    Placebo matched to SAR100842 for 8 weeks.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo- Core Part
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo matched to SAR100842 300 mg (100 mg +200 mg tablets), given twice daily (2 tablets in the morning and 2 tablets in the evening) for a total daily dose of 600 mg for 8 weeks.

    Arm title
    SAR100842-Core Part
    Arm description
    SAR100842 300 mg BID for 8 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    SAR100842
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    SAR100842 300 mg (100 mg + 200 mg tablets), given twice daily (2 tablets in the morning and 2 tablets in the evening) for a total daily dose of 600 mg for 8 weeks.

    Number of subjects in period 1
    Placebo-Core Part SAR100842-Core Part
    Started
    17
    15
    Completed
    17
    14
    Not completed
    0
    1
         Personal request
             -
             1
    Period 2
    Period 2 title
    Extension Part
    Is this the baseline period?
    No
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo/SAR100842-Extension Part
    Arm description
    Subjects who received placebo in core part were treated with SAR100842 for further 16 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    SAR100842
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    SAR100842 300 mg (100 mg + 200 mg tablets), given twice daily (2 tablets in the morning and 2 tablets in the evening) for a total daily dose of 600 mg for 16 weeks.

    Arm title
    SAR100842-Extension Part
    Arm description
    Subjects who received SAR100842 in core part were treated with SAR100842 for further 16 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    SAR100842
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    SAR100842 300 mg (100 mg + 200 mg tablets), given twice daily (2 tablets in the morning and 2 tablets in the evening) for a total daily dose of 600 mg for 16 weeks.

    Number of subjects in period 2 [1]
    Placebo/SAR100842-Extension Part SAR100842-Extension Part
    Started
    16
    14
    Completed
    15
    13
    Not completed
    1
    1
         Adverse event
             1
             1
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: One subject from placebo core part group did not continue in Extension part.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo-Core Part
    Reporting group description
    Placebo matched to SAR100842 for 8 weeks.

    Reporting group title
    SAR100842-Core Part
    Reporting group description
    SAR100842 300 mg BID for 8 weeks.

    Reporting group values
    Placebo-Core Part SAR100842-Core Part Total
    Number of subjects
    17 15 32
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    50.6 ± 11.3 48.8 ± 10.3 -
    Gender categorical
    Units: Subjects
        Female
    12 9 21
        Male
    5 6 11

    End points

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    End points reporting groups
    Reporting group title
    Placebo-Core Part
    Reporting group description
    Placebo matched to SAR100842 for 8 weeks.

    Reporting group title
    SAR100842-Core Part
    Reporting group description
    SAR100842 300 mg BID for 8 weeks.
    Reporting group title
    Placebo/SAR100842-Extension Part
    Reporting group description
    Subjects who received placebo in core part were treated with SAR100842 for further 16 weeks.

    Reporting group title
    SAR100842-Extension Part
    Reporting group description
    Subjects who received SAR100842 in core part were treated with SAR100842 for further 16 weeks.

    Primary: Treatment Emergent Adverse Event - Safety Population

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    End point title
    Treatment Emergent Adverse Event - Safety Population [1]
    End point description
    Adverse Event (AE) was defined as any untoward medical occurrence in a subject or clinical investigation subject administered a pharmaceutical product and which did not necessarily have to have a causal relationship with this treatment. Analysis was performed on safety population defined as randomized and treated subjects in the core part (respectively extension part, for extension part analysis), analyzed according to the treatment actually received.
    End point type
    Primary
    End point timeframe
    Baseline Up to Week 8 - Core part, Week 8 to Week 24 - Extension part
    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 data reported are qualitative, hence, no statistical analysis is provided.
    End point values
    Placebo-Core Part SAR100842-Core Part Placebo/SAR100842-Extension Part SAR100842-Extension Part
    Number of subjects analysed
    17
    15
    16
    14
    Units: subjects
        TEAE
    12
    12
    13
    10
        Treatment emergent SAE
    0
    1
    1
    1
        TEAE leading permanent treatment discontinuation
    0
    0
    1
    1
    No statistical analyses for this end point

    Secondary: Change From Baseline in Total Modified Rodnan Skin Score (mRSS) score Up to Week 24: mITT Population

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    End point title
    Change From Baseline in Total Modified Rodnan Skin Score (mRSS) score Up to Week 24: mITT Population
    End point description
    mRSS (measurement of skin thickening) was assessed by palpation of the skin in 17 areas of the body using 0 - 3 scale (0 = normal, 1 = mild thickness, 2 = moderate thickness and 3 = severe thickness). Total skin score can range from 0 (no thickening) to 51 (severe thickening in all 17 areas).  Modified intent­to­treat (mITT) population was defined as randomized and treated subjects with at least 1 post­ Investigational medicinal product (IMP)­administration measurement during the core part (respectively extension part, for extension part analysis).
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8 - Core part, Week 8 to Week 24 - Extension part
    End point values
    Placebo-Core Part SAR100842-Core Part Placebo/SAR100842-Extension Part SAR100842-Extension Part
    Number of subjects analysed
    17
    15
    13
    14
    Units: units on a scale
        arithmetic mean (standard deviation)
    -2.76 ± 4.85
    -3.4 ± 4.08
    -7.31 ± 4.59
    -7.36 ± 4.24
    No statistical analyses for this end point

    Secondary: Change From Baseline in Scleroderma Health Assessment Questionnaire (SHAQ) Total Score Up to Week 24: mITT Population

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    End point title
    Change From Baseline in Scleroderma Health Assessment Questionnaire (SHAQ) Total Score Up to Week 24: mITT Population
    End point description
    The SHAQ included a standard Health Assessment Questionnaire Disability Index (HAQ-DI) subscore and 5 visual analog scales (VAS) for severity assessments of Raynaud’s phenomenon, breathing, digital ulcers, gastrointestinal disease and overall disease. The HAQ-DI was scored 0 to 3 from no disability to the most severe one. Analysis was performed on mITT population (core part and extension part).
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8 - Core part, Week 8 to Week 24 - Extension part
    End point values
    Placebo-Core Part SAR100842-Core Part Placebo/SAR100842-Extension Part SAR100842-Extension Part
    Number of subjects analysed
    17
    15
    13
    14
    Units: units on a scale
        arithmetic mean (standard deviation)
    0 ± 0.33
    -0.14 ± 0.3
    -0.23 ± 0.3
    -0.15 ± 0.33
    No statistical analyses for this end point

    Secondary: Percentage of Responder Based on 4 Skin Biomarkers At Week 8: PD Population

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    End point title
    Percentage of Responder Based on 4 Skin Biomarkers At Week 8: PD Population
    End point description
    A subjects was considered as a responder if at least 20% reduction from baseline was demonstrated in 2 of these 4 biomarkers (cartilage oligomeric matrix protein [COMP], Collagen 1A1 [COL1A1] messenger ribonucleic acid [mRNA], thrombospondin 1 [TSP1] and alpha-smooth muscle actin [α-SMA] positive cells. Pharmacodynamics (PD) population, included randomized and treated subjects who received at least 4 weeks of IMP with at least a baseline and a post-baseline assessment.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8
    End point values
    Placebo-Core Part SAR100842-Core Part
    Number of subjects analysed
    17
    14
    Units: percentage of subjects
        number (not applicable)
    47.1
    64.3
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All Adverse Events (AE) were collected from signature of the informed consent form up to the final visit (core & extension part: Week 8, 24) regardless of seriousness or relationship to investigational product. Analysis was performed on safety population.
    Adverse event reporting additional description
    Reported adverse events are treatment-emergent adverse events that is AEs that developed/worsened during the ‘on treatment period’ [from the first administration of the study drug to the last administration of the study drug during the core/extension part + 5 days (5 half-lives)].
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    17.0
    Reporting groups
    Reporting group title
    Placebo-Core Part
    Reporting group description
    Placebo matched to SAR100842 for 8 weeks.

    Reporting group title
    SAR100842-Core Part
    Reporting group description
    SAR100842 300 mg BID for 8 weeks.

    Reporting group title
    Placebo/SAR100842-Extension Part
    Reporting group description
    Subjects who received placebo in core part were treated with SAR100842 for further 16 weeks.

    Reporting group title
    SAR100842-Extension Part
    Reporting group description
    Subjects who received SAR100842 in core part were treated with SAR100842 for further 16 weeks.

    Serious adverse events
    Placebo-Core Part SAR100842-Core Part Placebo/SAR100842-Extension Part SAR100842-Extension Part
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    1 / 16 (6.25%)
    1 / 14 (7.14%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Syncope
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Infected Skin Ulcer
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Placebo-Core Part SAR100842-Core Part Placebo/SAR100842-Extension Part SAR100842-Extension Part
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 17 (70.59%)
    11 / 15 (73.33%)
    13 / 16 (81.25%)
    9 / 14 (64.29%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Hot Flush
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Orthostatic Hypotension
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Hypotension
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Surgical and medical procedures
    Sinus Operation
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin Papilloma
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    2
    0
    1
    0
    Device Leakage
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Fatigue
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    2 / 16 (12.50%)
    1 / 14 (7.14%)
         occurrences all number
    1
    0
    2
    1
    Non-Cardiac Chest Pain
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Feeling Abnormal
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Oedema Peripheral
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    0
    1
    Pain
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Ulcer
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Psychiatric disorders
    Depression
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Insomnia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Reproductive system and breast disorders
    Menstruation Irregular
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Vaginal Haemorrhage
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Vaginal Discharge
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Injury, poisoning and procedural complications
    Accidental Overdose
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Post Procedural Discomfort
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Fall
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Procedural Pain
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Thermal Burn
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Investigations
    Lymphocyte Count Decreased
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Prothrombin Time Prolonged
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    1
    Cardiac disorders
    Cardiac Failure
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Palpitations
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    1
    1
    Dyspnoea
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    2 / 16 (12.50%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    2
    0
    Nasal Dryness
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Oropharyngeal Pain
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Pleural Effusion
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Rales
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pulmonary Mass
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Nervous system disorders
    Amnesia
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Dizziness
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Headache
         subjects affected / exposed
    0 / 17 (0.00%)
    3 / 15 (20.00%)
    3 / 16 (18.75%)
    1 / 14 (7.14%)
         occurrences all number
    0
    3
    3
    1
    Hyperaesthesia
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Migraine
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    1
    Paraesthesia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Muscle Contractions Involuntary
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Eye disorders
    Lacrimation Increased
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Pterygium
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Vitreous Detachment
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    1
    Gastrointestinal disorders
    Abdominal Pain
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Abdominal Pain Upper
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    1 / 14 (7.14%)
         occurrences all number
    1
    0
    1
    1
    Constipation
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    2
    1
    0
    0
    Diarrhoea
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 15 (13.33%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    1
    2
    1
    0
    Dyspepsia
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    2 / 16 (12.50%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Gastrooesophageal Reflux Disease
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 15 (6.67%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    1
    1
    1
    0
    Mouth Ulceration
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nausea
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 15 (13.33%)
    2 / 16 (12.50%)
    0 / 14 (0.00%)
         occurrences all number
    1
    2
    2
    0
    Oral Pain
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Tongue Ulceration
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Toothache
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Vomiting
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 15 (6.67%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    1
    1
    1
    0
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Keloid Scar
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Night Sweats
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Palmar Erythema
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Photosensitivity Reaction
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Pruritus
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pruritus Generalised
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Skin Depigmentation
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Skin Discolouration
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Skin Discomfort
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Skin Induration
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Skin Lesion
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Skin Ulcer
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    2 / 16 (12.50%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    2
    0
    Swelling Face
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    3 / 16 (18.75%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    3
    0
    Arthritis
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    1
    Back Pain
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    0
    1
    Limb Discomfort
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Muscle Spasms
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Musculoskeletal Discomfort
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Musculoskeletal Stiffness
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Myalgia
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Pain In Extremity
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Temporomandibular Joint Syndrome
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    1
    Metabolism and nutrition disorders
    Decreased Appetite
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Infections and infestations
    Application Site Cellulitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastroenteritis Viral
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Herpes Zoster
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    2 / 14 (14.29%)
         occurrences all number
    0
    0
    0
    2
    Sinusitis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 15 (6.67%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    0
    1
    Upper Respiratory Tract Infection
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Urinary Tract Infection
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 Feb 2013
    A 16 week extension was proposed to all subjects willing to continue on SAR100842, after they had completed the 8 week treatment and procedures. This longer exposure would provide additional long term safety data, and additional guidance for the selection and monitoring of subjects in longer further studies. Since SAR100842 was to be given in open label, subjects initially treated with placebo had the opportunity to receive the drug. The following sections was updated: A summary of the 6-month toxicology data supporting this extension was added, and the rationale of this extension was provided. A secondary objective and endpoint on safety and tolerability during the extension part was added. The inclusion/exclusion criteria for the extension were described. Only subjects who had completed the treatment and all procedures of the core part were included. All events described as reason for discontinuation in the core part were listed as exclusion criteria of the extension part, details of the volume of blood samples for the extension were provided (50ml to 65 ml depending on the number of blood samples taken). Clinical and laboratory safety were to be assessed through vital signs, physical examination, blood and urine samples. Depending on the duration of the window, additional physical examination, body weight and blood samples were to be performed at inclusion in the extension part. An optional skin biopsy was to be proposed at the end of the open label extension. Two informed consents were added, one for the extension part and another for the skin biopsy at the end of open label treatment, subjects who stopped the study treatment prematurely had the procedures listed in the end of treatment visit except that the last IMP was not to be given. Any procedure related to this intake such as PK sampling, and blood pressure measurement 4 hours after the intake, was not applicable. Mycophenolate mofetil greater than 2gm a day was added in exclusion criteria.
    03 Dec 2013
    One of the secondary objective of this study was to explore the effect of SAR100842 in subjects with diffuse cutaneous systemic sclerosis/scleroderma (dcSSc) as measured by disease related biomarkers.

    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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