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    Clinical Trial Results:
    A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial to Evaluate Efficacy and Safety of Lenabasum in Dermatomyositis

    Summary
    EudraCT number
    		 2018-003273-10
    Trial protocol
    		 GB   DE   HU   CZ   BG   SE   ES   IT  
    Global end of trial date
    05 Oct 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    30 Jul 2022
    First version publication date
    30 Jul 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    JBT101-DM-002
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03813160
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Corbus Pharmaceuticals, Inc.
    Sponsor organisation address
    500 River Ridge Drive, Second Floor, Norwood, Massachusetts, United States, 02062
    Public contact
    Brian Walsh, Corbus Pharmaceuticals, Inc., brian.walsh@corbuspharma.com
    Scientific contact
    Rachael Brake, Corbus Pharmaceuticals, Inc., rachael.brakeBrian.Walsh@corbuspharma.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
    18 Mar 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    05 Oct 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    05 Oct 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of lenabasum compared to placebo in subjects with dermatomyositis (DM) as measured by Total Improvement Score (TIS). To evaluate the safety and tolerability of lenabasum in subjects with DM. To evaluate the pharmacokinetics (PK) of lenabasum and investigate its metabolites in subjects with DM. To evaluate the effect of lenabasum compared to placebo on blood biomarkers of inflammation in subjects with DM. To evaluate the effect of lenabasum compared to placebo in skin biopsies in involved skin in subjects with DM (optional at selected sites). To evaluate the effect of lenabasum compared to placebo in involved skin as evaluated by skin photography in subjects with DM (optional at selected sites). To evaluate the long-term efficacy and safety of lenabasum in subjects who completed the study treatment phase and continued to receive treatment in an optional open-label extension (OLE).
    Protection of trial subjects
    Oversight of subject safety was provided by an independent unblinded Data Monitoring Committee (DMC), which advised the Sponsor and the investigators. The independent DMC reviewed the accumulated safety and operational data about every 6 months through the last subject/last visit or more frequently, if necessary. The DMC reviewed interim/cumulative data for evidence of study-related AEs and factors external to the study such as scientific or therapeutic developments that could impact subject safety. The DMC also reviewed progress of the study and efficacy outcomes. The DMC made recommendations to the Sponsor about any of the items it reviewed.
    Background therapy
    		 Subjects were allowed to continue their standard-of-care treatment (stable dose of immunosuppressive medication) while participating in the study, in order to reduce the risk of disease flare precipitated by having to discontinue medication to meet entry criteria. To avoid confounding efficacy and safety evaluations, changes in ongoing treatments and introduction of new therapies were kept to a minimum.
    Evidence for comparator
    		 Placebo was a powder-in-capsule containing microcrystalline cellulose and magnesium stearate (no active ingredient).
    Actual start date of recruitment
    30 Dec 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
    Poland: 7
    Country: Number of subjects enrolled
    Spain: 8
    Country: Number of subjects enrolled
    Sweden: 2
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    Bulgaria: 7
    Country: Number of subjects enrolled
    Czechia: 6
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    Hungary: 7
    Country: Number of subjects enrolled
    Italy: 7
    Country: Number of subjects enrolled
    Canada: 1
    Country: Number of subjects enrolled
    Japan: 26
    Country: Number of subjects enrolled
    Korea, Republic of: 8
    Country: Number of subjects enrolled
    United States: 92
    Worldwide total number of subjects
    178
    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)
    153
    From 65 to 84 years
    25
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 178 subjects were randomized to receive lenabasum 5 mg BID, lenabasum 20 mg BID or placebo at multiple study sites in the United States, Bulgaria, Canada, Czech Republic, Germany, Hungary, Italy, Japan, Poland, Republic of Korea, Spain, Sweden, and United Kingdom. Subjects were randomized between 17 Dec 2018 and 26 Apr 2021.

    Pre-assignment
    Screening details
    		 Of the 178 randomized subjects, 23 subjects were screen failures. The most common reasons for screen failure were failure to satisfy inclusion/exclusion criteria (15) and other (8). Three (1.7%) subjects discontinued prior to dosing, and 175 (98.3%) subjects were included in the modified intent-to-treat (mITT) population.

    Period 1
    Period 1 title
    Part A
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor
    Blinding implementation details
    Lenabasum and placebo capsules had a similar physical appearance and were packaged, labeled, and handled so that subjects and study staff were not able to distinguish between the two. Identical assessments and procedures were followed during the study for subjects assigned to lenabasum or placebo.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Lenabasum 20 mg
    Arm description
    		 Lenabasum 20mg was given orally, twice daily as a hard capsule.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lenabasum 20mg BID
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    Lenabasum 20mg was given twice daily as a hard capsule for 52 weeks.

    Arm title
    		 Lenabasum 5mg
    Arm description
    		 Lenabasum 5mg was given orally, twice daily as a hard capsule.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lenabasum 5mg BID
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    Lenabasum 5mg was given twice daily as a hard capsule for 52 weeks.

    Arm title
    		 Placebo
    Arm description
    		 Placebo was given as a powder-in-capsule containing microcrystalline cellulose and magnesium stearate.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo was given as a hard capsule twice daily for 52 weeks.

    Number of subjects in period 1
    		Lenabasum 20 mg Lenabasum 5mg Placebo
    Started
    71
    35
    72
    Completed
    35
    22
    38
    Not completed
    36
    13
    34
         Physician decision
    1
    -
    -
         Consent withdrawn by subject
    6
    1
    1
         unknown
    1
    -
    -
         Study terminated by Sponsor
    -
    -
    25
         Adverse event, non-fatal
    2
    1
    4
         Not specified
    -
    -
    1
         Non-compliance with the study
    -
    -
    1
         Lost to follow-up
    -
    1
    1
         Study terminated by Sponsor.
    26
    -
    -
         Study terminated by the Sponsor
    -
    10
    -
         Lack of efficacy
    -
    -
    1
    Period 2
    Period 2 title
    Part B
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Lenabasum 20mg
    Arm description
    		 Lenabasum 20mg given orally, twice daily as a hard capsule.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Lenabasum 20mg BID
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    Lenabasum 20mg BID was given orally as a hard-gelatin capsule in the open-label extension period (Part B).

    Number of subjects in period 2 [1]
    		Lenabasum 20mg
    Started
    49
    Completed
    9
    Not completed
    40
         Sponsor terminated the study
    27
         Adverse event, non-fatal
    1
         Not specified
    1
         Lost to follow-up
    1
         Missing
    10
    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: Out of 95 subjects who completed Part A of the study, 81 subjects entered the open-label extension phase of the study (Part B).

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Lenabasum 20 mg
    Reporting group description
    		 Lenabasum 20mg was given orally, twice daily as a hard capsule.

    Reporting group title
    Lenabasum 5mg
    Reporting group description
    		 Lenabasum 5mg was given orally, twice daily as a hard capsule.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo was given as a powder-in-capsule containing microcrystalline cellulose and magnesium stearate.

    Reporting group values
    		 Lenabasum 20 mg Lenabasum 5mg Placebo Total
    Number of subjects
    		 71 35 72 178
    Age categorical
    < 65 Years
    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)
    		 59 30 63 152
        From 65-84 years
    		 12 5 9 26
        85 years and over
    		 0 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 52.7 ± 12.76 51.5 ± 11.34 51.5 ± 12.19 -
    Gender categorical
    Female Male
    Units: Subjects
        Female
    		 56 30 59 145
        Male
    		 15 5 13 33
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 3 3
        Asian
    		 15 7 13 35
        Black or African American
    		 3 0 0 3
        White
    		 53 26 56 135
        Other
    		 0 2 0 2

    End points

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    End points reporting groups
    Reporting group title
    Lenabasum 20 mg
    Reporting group description
    		 Lenabasum 20mg was given orally, twice daily as a hard capsule.

    Reporting group title
    Lenabasum 5mg
    Reporting group description
    		 Lenabasum 5mg was given orally, twice daily as a hard capsule.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo was given as a powder-in-capsule containing microcrystalline cellulose and magnesium stearate.
    Reporting group title
    Lenabasum 20mg
    Reporting group description
    		 Lenabasum 20mg given orally, twice daily as a hard capsule.

    Primary: Total Improvement Score

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    End point title
    		 Total Improvement Score
    End point description
    Modified Intent-to-Treat Population
    End point type
    Primary
    End point timeframe
    Total Improvement Score (TIS) for lenabasum 20 mg BID compared to placebo at Week 28.
    End point values
    		 Lenabasum 20 mg Lenabasum 5mg Placebo
    Number of subjects analysed
    69
    28
    71
    Units: LS Mean (SE)
        least squares mean (standard error)
    26.8 ± 2.85
    22.7 ± 3.81
    23.7 ± 2.79
    Statistical analysis title
    		 Lenabasum 20 mg BID versus placebo
    Statistical analysis description
    The analysis of the primary endpoint was performed using an mixed model for repeated measures (MMRM) using data after missing data or visits due to COVID-19 using LOCF. The primary efficacy endpoint analysis compared TIS (by the 2016 ACR/EULAR Myositis Response Criteria) of lenabasum 20 mg to placebo at Week 28.
    Comparison groups
    Lenabasum 20 mg v Placebo
    Number of subjects included in analysis
    140
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.3311 [1]
    Method
    		 Mixed models analysis
    Parameter type
    		 Mean difference (final values)
    Point estimate
    3.2
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -3.3
         upper limit
    		 9.6
    Notes
    [1] - Based on MMRM with region, Sex, Baseline MMT-8 score, Baseline immunosuppressive use, visit, treatment and Baseline immunosuppressive use by-visit and treatment-by-visit interaction as fixed effects. Covariance structure type = un.
    Statistical analysis title
    		 Lenabasum 5mg BID versus Placebo
    Statistical analysis description
    Secondary analyses compared lenabasum 5 mg BID and all lenabasum vs. placebo at Week 28.
    Comparison groups
    Lenabasum 5mg v Placebo
    Number of subjects included in analysis
    99
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [2]
    P-value
    		 = 0.8161
    Method
    		 Mixed models analysis
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -0.9
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -8.9
         upper limit
    		 7
    Notes
    [2] - Based on MMRM with region, Sex, Baseline MMT-8 score, Baseline immunosuppressive use, visit, treatment and Baseline immunosuppressive use by-visit and treatment-by-visit interaction as fixed effects. Covariance structure type = un.

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Assessment of Treatment emergent adverse events from Day 1 through Week 52.
    Adverse event reporting additional description
    Part A: The Safety Follow-up Visit was to occur 4±1 weeks after the last visit (Visit 10/ET) and was to be completed by subjects who did not rollover into Part B. Part B: The Safety Follow-up Visit was to occur 4 ± 1 weeks after the last visit (Visit B8 or Visit C8 if OLE was extended).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.1
    Reporting groups
    Reporting group title
    Lenabasum 20 mg BID, Part A
    Reporting group description
    		 Adverse events in Part A.

    Reporting group title
    Lenabasum 5 mg BID, Part A
    Reporting group description
    		 Adverse events in Part A.

    Reporting group title
    Placebo, Part A
    Reporting group description
    		 Adverse events in Part A.

    Reporting group title
    Lenabasum 20 mg BID, Part B
    Reporting group description
    		 Adverse events in Part B.

    Serious adverse events
    		 Lenabasum 20 mg BID, Part A Lenabasum 5 mg BID, Part A Placebo, Part A Lenabasum 20 mg BID, Part B
    Total subjects affected by serious adverse events
         subjects affected / exposed
    8 / 69 (11.59%)
    3 / 35 (8.57%)
    3 / 71 (4.23%)
    3 / 49 (6.12%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Epstein-Barr virus associated lymphoma
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 35 (0.00%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Malignant melanoma
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 35 (0.00%)
    1 / 71 (1.41%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Papillary thyroid cancer
         subjects affected / exposed
    0 / 69 (0.00%)
    1 / 35 (2.86%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Gastrointestinal procedural complication
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 35 (0.00%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Aortic thrombosis
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 35 (0.00%)
    1 / 71 (1.41%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Deep vein thrombosis
         subjects affected / exposed
    0 / 69 (0.00%)
    1 / 35 (2.86%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 35 (0.00%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Accelerated idioventricular rhythm
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 35 (0.00%)
    1 / 71 (1.41%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Coronary artery dissection
         subjects affected / exposed
    0 / 69 (0.00%)
    1 / 35 (2.86%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Vertebral artery dissection
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 35 (0.00%)
    1 / 71 (1.41%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Headache
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 35 (0.00%)
    1 / 71 (1.41%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 35 (0.00%)
    1 / 71 (1.41%)
    0 / 49 (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
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 35 (0.00%)
    1 / 71 (1.41%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pneumonia aspiration
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 35 (0.00%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Dermatomyositis
         subjects affected / exposed
    1 / 69 (1.45%)
    1 / 35 (2.86%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Spinal stenosis
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 35 (0.00%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis perforated
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 35 (0.00%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis viral
         subjects affected / exposed
    0 / 69 (0.00%)
    1 / 35 (2.86%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia respiratory syncytial viral
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 35 (0.00%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 35 (0.00%)
    0 / 71 (0.00%)
    2 / 49 (4.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cellulitis
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 35 (0.00%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         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
    		 Lenabasum 20 mg BID, Part A Lenabasum 5 mg BID, Part A Placebo, Part A Lenabasum 20 mg BID, Part B
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    60 / 69 (86.96%)
    30 / 35 (85.71%)
    62 / 71 (87.32%)
    37 / 49 (75.51%)
    Investigations
    Blood pressure increased
         subjects affected / exposed
    0 / 69 (0.00%)
    2 / 35 (5.71%)
    1 / 71 (1.41%)
    0 / 49 (0.00%)
         occurrences all number
    0
    2
    1
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    9 / 69 (13.04%)
    2 / 35 (5.71%)
    3 / 71 (4.23%)
    2 / 49 (4.08%)
         occurrences all number
    10
    2
    3
    2
    Headache
         subjects affected / exposed
    7 / 69 (10.14%)
    5 / 35 (14.29%)
    10 / 71 (14.08%)
    10 / 49 (20.41%)
         occurrences all number
    8
    6
    10
    10
    Somnolence
         subjects affected / exposed
    2 / 69 (2.90%)
    2 / 35 (5.71%)
    0 / 71 (0.00%)
    3 / 49 (6.12%)
         occurrences all number
    2
    2
    0
    3
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    6 / 69 (8.70%)
    1 / 35 (2.86%)
    2 / 71 (2.82%)
    2 / 49 (4.08%)
         occurrences all number
    6
    1
    2
    2
    Adverse drug reaction
         subjects affected / exposed
    2 / 69 (2.90%)
    2 / 35 (5.71%)
    3 / 71 (4.23%)
    2 / 49 (4.08%)
         occurrences all number
    2
    2
    4
    2
    Chills
         subjects affected / exposed
    0 / 69 (0.00%)
    2 / 35 (5.71%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Oedema peripheral
         subjects affected / exposed
    2 / 69 (2.90%)
    2 / 35 (5.71%)
    1 / 71 (1.41%)
    0 / 49 (0.00%)
         occurrences all number
    3
    2
    1
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    10 / 69 (14.49%)
    4 / 35 (11.43%)
    6 / 71 (8.45%)
    7 / 49 (14.29%)
         occurrences all number
    12
    7
    7
    8
    Dry mouth
         subjects affected / exposed
    5 / 69 (7.25%)
    2 / 35 (5.71%)
    2 / 71 (2.82%)
    3 / 49 (6.12%)
         occurrences all number
    5
    2
    2
    3
    Nausea
         subjects affected / exposed
    8 / 69 (11.59%)
    4 / 35 (11.43%)
    3 / 71 (4.23%)
    2 / 49 (4.08%)
         occurrences all number
    10
    5
    3
    2
    Abdominal pain upper
         subjects affected / exposed
    1 / 69 (1.45%)
    3 / 35 (8.57%)
    0 / 71 (0.00%)
    0 / 49 (0.00%)
         occurrences all number
    1
    3
    0
    0
    Constipation
         subjects affected / exposed
    1 / 69 (1.45%)
    2 / 35 (5.71%)
    1 / 71 (1.41%)
    0 / 49 (0.00%)
         occurrences all number
    1
    2
    1
    0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    0 / 69 (0.00%)
    2 / 35 (5.71%)
    0 / 71 (0.00%)
    4 / 49 (8.16%)
         occurrences all number
    0
    2
    0
    4
    Dermatomyositis
         subjects affected / exposed
    19 / 69 (27.54%)
    11 / 35 (31.43%)
    29 / 71 (40.85%)
    35 / 49 (71.43%)
         occurrences all number
    27
    17
    45
    42
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 69 (0.00%)
    2 / 35 (5.71%)
    4 / 71 (5.63%)
    1 / 49 (2.04%)
         occurrences all number
    0
    2
    4
    1
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    0 / 69 (0.00%)
    2 / 35 (5.71%)
    0 / 71 (0.00%)
    1 / 49 (2.04%)
         occurrences all number
    0
    2
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    7 / 69 (10.14%)
    0 / 35 (0.00%)
    2 / 71 (2.82%)
    7 / 49 (14.29%)
         occurrences all number
    8
    0
    4
    9
    Back pain
         subjects affected / exposed
    2 / 69 (2.90%)
    0 / 35 (0.00%)
    4 / 71 (5.63%)
    3 / 49 (6.12%)
         occurrences all number
    2
    0
    4
    3
    Pain in extremity
         subjects affected / exposed
    2 / 69 (2.90%)
    2 / 35 (5.71%)
    4 / 71 (5.63%)
    4 / 49 (8.16%)
         occurrences all number
    3
    2
    4
    5
    Neck pain
         subjects affected / exposed
    1 / 69 (1.45%)
    2 / 35 (5.71%)
    0 / 71 (0.00%)
    1 / 49 (2.04%)
         occurrences all number
    1
    2
    0
    1
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    4 / 69 (5.80%)
    1 / 35 (2.86%)
    3 / 71 (4.23%)
    1 / 49 (2.04%)
         occurrences all number
    4
    1
    5
    1
    Nasopharyngitis
         subjects affected / exposed
    5 / 69 (7.25%)
    3 / 35 (8.57%)
    2 / 71 (2.82%)
    2 / 49 (4.08%)
         occurrences all number
    5
    3
    2
    2
    Urinary tract infection
         subjects affected / exposed
    4 / 69 (5.80%)
    2 / 35 (5.71%)
    3 / 71 (4.23%)
    1 / 49 (2.04%)
         occurrences all number
    5
    2
    4
    2
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    4 / 69 (5.80%)
    0 / 35 (0.00%)
    0 / 71 (0.00%)
    1 / 49 (2.04%)
         occurrences all number
    4
    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
    03 Oct 2018
    Version 1.1
    17 Oct 2018
    Version 1.2
    05 Dec 2019
    Version 2.0: Secondary efficacy endpoint was revised to include each component of the TIS (MDGA, PtGA, HAQ, muscle enzymes, and EMGA) at the request of Regulatory Authorities. Evaluation of long-term efficacy and safety as an additional objective in an optional open-label extension (OLE) was added. Subjects received lenabasum 20 mg BID. A schedule of assessments was provided for one year with the potential for the OLE to be extended beyond 1 year. Due to a continued favorable safety profile and efficacy signals for lenabasum, an optional OLE was added for further evaluation of long-term safety and efficacy. Added key inclusion criteria including DM diagnosis by Bohan and Peter or ACR/EULAR criteria at the request of Regulatory Authorities. No major changes were made to eligibility criteria other than providing additional clarity to address questions from sites/ IRB/EC/Regulatory Authorities.
    13 Jan 2021
    Version 2.2

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    26 Apr 2021
    Sponsor made the decision to terminate the double-blind study once all subjects had completed at least Visit 6 allowing for analysis of the amended primary endpoint. Sponsor made the decision to discontinue development of lenabasum for DM.
    -

    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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