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
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EudraCT number |
2018-004740-36 |
Trial protocol |
DE FR DK |
Global end of trial date |
01 Jan 2021
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Results information
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Results version number |
v1(current) |
This version publication date |
01 Aug 2022
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First version publication date |
01 Aug 2022
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
PTCEMF-GD-004
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT03783923 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
PTC Therapeutics, Inc.
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Sponsor organisation address |
100 Corporate Court, South Plainfield, United States, NJ 07080
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Public contact |
Medical Information, PTC Therapeutics, Inc., +011 44 1-866-562-4620, medinfo@ptcbio.com
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Scientific contact |
Medical Information, PTC Therapeutics International Limited, +353 19068700, medinfo@ptcbio.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
01 Jan 2021
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
01 Jan 2021
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Global end of trial reached? |
Yes
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Global end of trial date |
01 Jan 2021
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Was the trial ended prematurely? |
Yes
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General information about the trial
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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.
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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.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
31 Oct 2019
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
United States: 11
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Worldwide total number of subjects |
11
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
10
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From 65 to 84 years |
1
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||||||||||||||||||
Pre-assignment
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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
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Period 1 title |
Placebo-Controlled Period (26 Weeks)
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Is this the baseline period? |
Yes | ||||||||||||||||||||||||
Allocation method |
Randomised - controlled
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Blinding used |
Double blind | ||||||||||||||||||||||||
Roles blinded |
Investigator, Subject | ||||||||||||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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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
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Investigational medicinal product code |
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Other name |
Emflaza®
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Deflazacort was administered as per the dose and schedule specified in the arm description.
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Arm title
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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
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Placebo matched to deflazacort was administered as per schedule specified in the arm description.
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Period 2
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Period 2 title |
Open-Label Extension (26 Weeks)
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Is this the baseline period? |
No | ||||||||||||||||||||||||
Allocation method |
Non-randomised - controlled
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Blinding used |
Not blinded | ||||||||||||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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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
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Investigational medicinal product code |
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Other name |
Emflaza®
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Deflazacort was administered as per the dose and schedule specified in the arm description.
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Arm title
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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
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Investigational medicinal product code |
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Other name |
Emflaza®
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Deflazacort was administered as per the dose and schedule specified in the arm description.
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Baseline characteristics reporting groups
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Reporting group title |
Deflazacort
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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
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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. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Deflazacort
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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
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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
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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
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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
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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.
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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.
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End point type |
Primary
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End point timeframe |
Baseline, Week 26
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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. |
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 26
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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. |
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Notes [3] - Due to early termination of study, data was not collected or analyzed. |
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 26
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 26
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 26
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 26
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 26
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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. |
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Notes [5] - Due to early termination of the study, data was not collected or analyzed. |
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 26
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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. |
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Notes [7] - Due to early termination of the study, data was not collected or analyzed. |
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 26
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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. |
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Notes [9] - Due to early termination of the study, data was not collected or analyzed. |
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Baseline up to Week 52
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
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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. |
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
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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. |
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
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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. |
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
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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. |
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No statistical analyses for this end point |
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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.
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End point type |
Secondary
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End point timeframe |
Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
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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. |
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No statistical analyses for this end point |
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Adverse events information
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Timeframe for reporting adverse events |
Baseline up to Week 52
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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.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
23.1
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Reporting groups
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Reporting group title |
Deflazacort
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Reporting group description |
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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. |
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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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. |
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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. |
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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. |
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Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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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. |