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    Clinical Trial Results:
    A Multicenter Open-Label Study on the Safety and Efficacy of Deflazacort (Emflaza) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

    Summary
    EudraCT number
    		 2018-004740-36
    Trial protocol
    		 DE   FR   DK  
    Global end of trial date
    01 Jan 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    01 Aug 2022
    First version publication date
    01 Aug 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    PTCEMF-GD-004
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03783923
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    PTC Therapeutics, Inc.
    Sponsor organisation address
    100 Corporate Court, South Plainfield, United States, NJ 07080
    Public contact
    Medical Information, PTC Therapeutics, Inc., +011 44 1-866-562-4620, medinfo@ptcbio.com
    Scientific contact
    Medical Information, PTC Therapeutics International Limited, +353 19068700, medinfo@ptcbio.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
    01 Jan 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    01 Jan 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Jan 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of the study was to evaluate the efficacy of deflazacort as measured by muscle function in participants with LGMD2I.
    Protection of trial subjects
    This trial was conducted in accordance with the ethical principles of Good Clinical Practice, according to the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Harmonized Tripartite Guideline.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    31 Oct 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 11
    Worldwide total number of subjects
    11
    EEA total number of subjects
    0
    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)
    10
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Participants enrolled under protocol v3.0 were 1:1 randomized to get placebo or deflazacort. After protocol v4.0, the study became an open-label study and all participants received deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety and efficacy summaries.

    Period 1
    Period 1 title
    Placebo-Controlled Period (26 Weeks)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Deflazacort
    Arm description
    		 Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 milligrams (mg)/kilograms (kg)/day for 26 weeks in placebo-controlled period and for 26 weeks in open-label extension period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deflazacort
    Investigational medicinal product code
    Other name
    Emflaza®
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Deflazacort was administered as per the dose and schedule specified in the arm description.

    Arm title
    		 Placebo
    Arm description
    		 Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo matched to deflazacort was administered as per schedule specified in the arm description.

    Number of subjects in period 1
    		Deflazacort Placebo
    Started
    5
    6
    Safety population
    5
    2
    Completed
    1
    2
    Not completed
    4
    4
         Consent withdrawn by subject
    -
    2
         Adverse event, non-fatal
    1
    -
         Study Terminated
    3
    2
    Period 2
    Period 2 title
    Open-Label Extension (26 Weeks)
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Deflazacort
    Arm description
    		 Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 milligrams (mg)/kilograms (kg)/day for 26 weeks in placebo-controlled period and for 26 weeks in open-label extension period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deflazacort
    Investigational medicinal product code
    Other name
    Emflaza®
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Deflazacort was administered as per the dose and schedule specified in the arm description.

    Arm title
    		 Placebo
    Arm description
    		 Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Deflazacort
    Investigational medicinal product code
    Other name
    Emflaza®
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Deflazacort was administered as per the dose and schedule specified in the arm description.

    Number of subjects in period 2
    		Deflazacort Placebo
    Started
    1
    2
    Completed
    0
    0
    Not completed
    1
    2
         Consent withdrawn by subject
    1
    1
         Study Terminated
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Deflazacort
    Reporting group description
    		 Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 milligrams (mg)/kilograms (kg)/day for 26 weeks in placebo-controlled period and for 26 weeks in open-label extension period.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.

    Reporting group values
    		 Deflazacort Placebo Total
    Number of subjects
    		 5 6 11
    Age Categorical
    Units: participants
        <=18 years
    		 0 0 0
        Between 18 and 65 years
    		 5 5 10
        >=65 years
    		 0 1 1
    Sex: Female, Male
    Units: participants
        Female
    		 4 4 8
        Male
    		 1 2 3
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 0 0 0
        White
    		 5 6 11
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    Deflazacort
    Reporting group description
    		 Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 milligrams (mg)/kilograms (kg)/day for 26 weeks in placebo-controlled period and for 26 weeks in open-label extension period.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.
    Reporting group title
    Deflazacort
    Reporting group description
    		 Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 milligrams (mg)/kilograms (kg)/day for 26 weeks in placebo-controlled period and for 26 weeks in open-label extension period.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.

    Subject analysis set title
    Deflazacort
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.

    Primary: Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort

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    End point title
    		 Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort [1]
    End point description
    Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'n' = participants evaluable at specified timepoint. 99999 = Single participant was analyzed; hence, standard deviation (SD) could not be calculated.
    End point type
    Primary
    End point timeframe
    Baseline, Week 26
    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 endpoint is descriptive in nature.
    End point values
    		 Deflazacort
    Number of subjects analysed
    7
    Units: seconds
    arithmetic mean (standard deviation)
        Baseline (n = 7)
    5.476 ( 2.0178 )
        Change at Week 26 (n = 1)
    -0.200 ( 99999 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Forced Vital Capacity (FVC) After 26 Weeks of Treatment With Deflazacort

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    End point title
    		 Change From Baseline in Forced Vital Capacity (FVC) After 26 Weeks of Treatment With Deflazacort [2]
    End point description
    Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    Notes
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The end point is reporting statistics for specified arm only.
    End point values
    		 Deflazacort
    Number of subjects analysed
    0 [3]
    Units: liters
        arithmetic mean (standard deviation)
    ( )
    Notes
    [3] - Due to early termination of study, data was not collected or analyzed.
    No statistical analyses for this end point

    Secondary: Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort

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    End point title
    		 Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort
    End point description
    Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'n' = participants evaluable at specified timepoint. 99999 = Single participant was analyzed; hence, SD could not be calculated.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 Deflazacort
    Number of subjects analysed
    7
    Units: meters
    arithmetic mean (standard deviation)
        Baseline (n = 7)
    135.4 ( 26.75 )
        Change at Week 26 (n = 1)
    2.0 ( 99999 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort

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    End point title
    		 Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort
    End point description
    Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'n' = participants evaluable at specified timepoint. 99999 = Single participant was analyzed; hence, SD could not be calculated.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 Deflazacort
    Number of subjects analysed
    7
    Units: seconds
    arithmetic mean (standard deviation)
        Baseline (n = 7)
    11.93 ( 4.743 )
        Change at Week 26 (n = 1)
    9.70 ( 99999 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort

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    End point title
    		 Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort
    End point description
    Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'n' = participants evaluable at specified timepoint. 99999 = Single participant was analyzed; hence, SD could not be calculated.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 Deflazacort
    Number of subjects analysed
    7
    Units: seconds
    arithmetic mean (standard deviation)
        Baseline (n = 7)
    3.66 ( 1.707 )
        Change at Week 26 (n =1)
    0.10 ( 99999 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort

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    End point title
    		 Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort
    End point description
    Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'n' = participants evaluable at specified timepoint. 99999 = Single participant was analyzed; hence, SD could not be calculated.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 Deflazacort
    Number of subjects analysed
    7
    Units: seconds
    arithmetic mean (standard deviation)
        Baseline (n = 7)
    8.53 ( 1.897 )
        Change at Week 26 (n = 1)
    -0.40 ( 99999 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) After 26 Weeks of Treatment With Deflazacort

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    End point title
    		 Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) After 26 Weeks of Treatment With Deflazacort [4]
    End point description
    Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The endpoint is descriptive in nature.
    End point values
    		 Deflazacort
    Number of subjects analysed
    0 [5]
    Units: centimeters
        arithmetic mean (standard deviation)
    ( )
    Notes
    [5] - Due to early termination of the study, data was not collected or analyzed.
    No statistical analyses for this end point

    Secondary: Change From Baseline in Hand-Held Myometry After 26 Weeks of Treatment With Deflazacort

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    End point title
    		 Change From Baseline in Hand-Held Myometry After 26 Weeks of Treatment With Deflazacort [6]
    End point description
    Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    Notes
    [6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The endpoint is descriptive in nature.
    End point values
    		 Deflazacort
    Number of subjects analysed
    0 [7]
    Units: newtons
        arithmetic mean (standard deviation)
    ( )
    Notes
    [7] - Due to early termination of the study, data was not collected or analyzed.
    No statistical analyses for this end point

    Secondary: Change From Baseline in Global T2 Relaxation Time of Selected Upper and Lower Limb Muscles After 26 Weeks of Treatment With Deflazacort

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    End point title
    		 Change From Baseline in Global T2 Relaxation Time of Selected Upper and Lower Limb Muscles After 26 Weeks of Treatment With Deflazacort [8]
    End point description
    Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    Notes
    [8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The endpoint is descriptive in nature.
    End point values
    		 Deflazacort
    Number of subjects analysed
    0 [9]
    Units: seconds
        arithmetic mean (standard deviation)
    ( )
    Notes
    [9] - Due to early termination of the study, data was not collected or analyzed.
    No statistical analyses for this end point

    Secondary: Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort

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    End point title
    		 Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [10]
    End point description
    The pharmacokinetic (PK) population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'n' = participants evaluable for specified category. 99999 = Due to concentration below the limit of quantitation (BLQ) of 0.50 ng/mL, data not calculated.
    End point type
    Secondary
    End point timeframe
    Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
    Notes
    [10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The end point is reporting statistics for specified arm only.
    End point values
    		 Deflazacort
    Number of subjects analysed
    4
    Units: nanograms (ng)*hour (hr)/milliliter (mL)
    arithmetic mean (standard deviation)
        21-desacetyl deflazacort: Baseline (n=4)
    401.1 ( 139.62 )
        21-desacetyl deflazacort: Week 13 (n=2)
    365.6 ( 99999 )
        6β-hydroxy-21-desacetyl deflazacort: Week 1 (n=4)
    418.2 ( 58.569 )
        6β-hydroxy-21-desacetyl deflazacort: Week 13 (n=2)
    515.3 ( 99999 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Adverse Events (AEs)

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    End point title
    		 Number of Participants With Adverse Events (AEs)
    End point description
    An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. AEs were summarized separately for Stage 1 and for the overall ataluren experience. Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo during the study were not included for safety analysis.
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 52
    End point values
    		 Deflazacort
    Number of subjects analysed
    7
    Units: participants
    5
    No statistical analyses for this end point

    Secondary: Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort

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    End point title
    		 Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [11]
    End point description
    The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'n' = participants evaluable for specified category. 99999 = No participant was analyzed; hence, data not available.
    End point type
    Secondary
    End point timeframe
    Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
    Notes
    [11] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The end point is reporting statistics for specified arm only.
    End point values
    		 Deflazacort
    Number of subjects analysed
    4
    Units: ng*hr/mL
    arithmetic mean (standard deviation)
        21-desacetyl deflazacort: Baseline (n=4)
    423.6 ( 150.16 )
        21-desacetyl deflazacort: Week 13 (n=0)
    99999 ( 99999 )
        6β-hydroxy-21-desacetyl deflazacort: Week 1 (n=3)
    520.3 ( 31.508 )
        6β-hydroxy-21-desacetyl deflazacort: Week 13 (n=0)
    99999 ( 99999 )
    No statistical analyses for this end point

    Secondary: Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort

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    End point title
    		 Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [12]
    End point description
    The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'n' = participants evaluable for specified category. 99999 = Due to concentration BLQ of 0.50 ng/mL, data not calculated.
    End point type
    Secondary
    End point timeframe
    Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
    Notes
    [12] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The end point is reporting statistics for specified arm only.
    End point values
    		 Deflazacort
    Number of subjects analysed
    4
    Units: ng/mL
    arithmetic mean (standard deviation)
        21-desacetyl deflazacort: Baseline (n=4)
    184.8 ( 49.054 )
        21-desacetyl deflazacort: Week 13 (n=2)
    171.0 ( 99999 )
        6β-hydroxy-21-desacetyl deflazacort: Week 1 (n=4)
    135.8 ( 29.205 )
        6β-hydroxy-21-desacetyl deflazacort: Week 13 (n=2)
    162.0 ( 99999 )
    No statistical analyses for this end point

    Secondary: Time to Reach Cmax (Tmax) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort

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    End point title
    		 Time to Reach Cmax (Tmax) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [13]
    End point description
    The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'n' = participants evaluable for specified category.
    End point type
    Secondary
    End point timeframe
    Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
    Notes
    [13] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The end point is reporting statistics for specified arm only.
    End point values
    		 Deflazacort
    Number of subjects analysed
    4
    Units: hr
    median (full range (min-max))
        21-desacetyl deflazacort: Baseline (n=4)
    0.992 (0.50 to 1.00)
        21-desacetyl deflazacort: Week 13 (n=2)
    0.525 (0.50 to 0.55)
        6β-hydroxy-21-desacetyl deflazacort: Week 1 (n=4)
    1.000 (1.00 to 1.90)
        6β-hydroxy-21-desacetyl deflazacort: Week 13 (n=2)
    1.550 (1.10 to 2.00)
    No statistical analyses for this end point

    Secondary: Half-Life (t1/2) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort

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    End point title
    		 Half-Life (t1/2) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [14]
    End point description
    The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category. 99999 = Due to concentration BLQ of 0.50 ng/mL, data not calculated. 9999 = Single participant was analyzed; hence, SD could not be calculated.
    End point type
    Secondary
    End point timeframe
    Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
    Notes
    [14] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The end point is reporting statistics for specified arm only.
    End point values
    		 Deflazacort
    Number of subjects analysed
    4
    Units: hr
    arithmetic mean (standard deviation)
        21-desacetyl deflazacort: Baseline (n=4)
    1.174 ( 0.0849 )
        21-desacetyl deflazacort: Week 13 (n=2)
    1.235 ( 99999 )
        6β-hydroxy-21-desacetyl deflazacort: Week 1 (n=3)
    2.358 ( 0.5825 )
        6β-hydroxy-21-desacetyl deflazacort: Week 13 (n=1)
    1.95 ( 9999 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to Week 52
    Adverse event reporting additional description
    Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. Participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments per planned analysis.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.1
    Reporting groups
    Reporting group title
    Deflazacort
    Reporting group description
    		 Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.

    Serious adverse events
    		 Deflazacort
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 7 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Deflazacort
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 7 (71.43%)
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Reproductive system and breast disorders
    Amenorrhoea
    Additional description: This is a gender-specific AE. Only female participants were at risk.
         subjects affected / exposed [1]
    1 / 5 (20.00%)
         occurrences all number
    1
    Menstruation irregular
    Additional description: This is a gender-specific AE. Only female participants were at risk.
         subjects affected / exposed [2]
    1 / 5 (20.00%)
         occurrences all number
    1
    Psychiatric disorders
    Apathy
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Depressed mood
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Sleep disorder
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Investigations
    Weight increased
         subjects affected / exposed
    2 / 7 (28.57%)
         occurrences all number
    3
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Skin laceration
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Post-traumatic pain
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Spinal compression fracture
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Fall
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    2
    Hypoaesthesia
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Tension headache
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Visual field defect
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Dyspepsia
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    2
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    2 / 7 (28.57%)
         occurrences all number
    2
    Abnormal hair growth
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Hirsutism
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Endocrine disorders
    Cushingoid
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    2
    Back pain
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Muscle tightness
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Myalgia
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Infections and infestations
    Influenza
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Increased appetite
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    2
    Notes
    [1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: This is a gender-specific AE. Only female participants were at risk.
    [2] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: This is a gender-specific AE. Only female participants were at risk.

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    28 Feb 2019
    The overall reasons for this amendment were to include DEXA as an additional safety assessment in participants with LGMD2I. In addition, several editorial changes were made to clarify and correct the text.
    26 Sep 2019
    The overall reasons for this amendment were to address regulatory feedback and to provide updates, including adding an exploratory objective and endpoint, to add study visits, to clarify options for participants at the end of the study and if their blind is broken, and to clarify statistical analysis for the key secondary endpoints. In addition, several editorial changes were made to clarify and correct the text.
    24 Mar 2020
    The overall reason for this amendment was the closure of the study. The protocol was amended to allow continuation of treatment for those participants already on study and to allow further enrollment up to approximately 30 participants.

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    The study was terminated early due to low enrollment and missing efficacy assessment data due to missed visits related to COVID-19.
    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.

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