Clinical Trial Results:
An Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies
Summary
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EudraCT number |
2015-001910-88 |
Trial protocol |
DK |
Global end of trial date |
05 Oct 2016
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Results information
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Results version number |
v1(current) |
This version publication date |
13 Apr 2018
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First version publication date |
13 Apr 2018
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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 |
ATYR1940-C-004
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
- | ||
Other trial identifiers |
IND number: 122045, IND number: 132865 | ||
Sponsors
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Sponsor organisation name |
aTyr Pharma, Inc.
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Sponsor organisation address |
3545 John Hopkins Court, Suite #250, San Diego, CA, United States, 92121
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Public contact |
Clinical Trial Operations, Voisin Consulting, clinicaltrialinformation@voisinconsulting.com
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Scientific contact |
Clinical Trial Operations, Voisin Consulting, clinicaltrialinformation@voisinconsulting.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 |
30 Mar 2017
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
05 Oct 2016
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Global end of trial reached? |
Yes
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Global end of trial date |
05 Oct 2016
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
To evaluate the safety, tolerability, and immunogenicity of the weekly and twice weekly intravenous (IV) administration of ATYR1940, at doses of 0.3, 1.0, and 3.0 mg/kg, to patients with Limb Girdle Muscular Dystrophy 2B (LGMD2B, dysferlinopathy) and Facioscapulohumeral Muscular Dystrophy (FSHD).
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Protection of trial subjects |
The study process, benefits and risks of participating in the study were explained to each subject. In addition, if the study drug needed to be stopped for safety, the doctor, his/her staff along with the medical monitor, were to continue to monitor participant's health and determine what treatment should be given (if any) until the symptoms or findings had resolved or until a satisfactory conclusion was reached.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
02 Oct 2015
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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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 |
France: 8
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Country: Number of subjects enrolled |
Denmark: 5
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Country: Number of subjects enrolled |
United States: 5
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Worldwide total number of subjects |
18
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EEA total number of subjects |
13
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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) |
18
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From 65 to 84 years |
0
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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 |
- | |||||||||||||||||||||
Pre-assignment period milestones
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Number of subjects started |
26 [1] | |||||||||||||||||||||
Number of subjects completed |
18 | |||||||||||||||||||||
Pre-assignment subject non-completion reasons
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Reason: Number of subjects |
Screen failures: 8 | |||||||||||||||||||||
Notes [1] - The number of subjects reported to have started the pre-assignment period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same. Justification: The worldwide number corresponds to the number of patients randomized (18) and not to the number of patients screened (26). |
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Period 1
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Period 1 title |
Treatment period (overall period)
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Is this the baseline period? |
Yes | |||||||||||||||||||||
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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Group A – FSHD | |||||||||||||||||||||
Arm description |
Starting at a dose of 0.3 mg/kg, with the potential for intrapatient dose escalation to 1.0 mg/kg over the dosing period | |||||||||||||||||||||
Arm type |
Experimental | |||||||||||||||||||||
Investigational medicinal product name |
ATYR1940
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Concentrate for solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Patients were to receive a single, 90-minute IV placebo infusion followed by administration of ATYR1940 as a 90-minute IV infusion once a week for 8 weeks, then twice weekly for 4 weeks, for a total of 12 weeks
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Arm title
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Group B – LGMD2B and FSHD | |||||||||||||||||||||
Arm description |
Starting at a dose of 0.3 mg/kg/week, with the potential for intrapatient dose escalation to a dose of 3.0 mg/kg and up to 3.0 mg/kg twice a week over the dosing period | |||||||||||||||||||||
Arm type |
Experimental | |||||||||||||||||||||
Investigational medicinal product name |
ATYR1940
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Concentrate for solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Patients were to receive a single, 90-minute IV placebo infusion followed by administration of ATYR1940 as a 90-minute IV infusion once a week for 8 weeks, then twice weekly for 4 weeks, for a total of 12 weeks
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Baseline characteristics reporting groups
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Reporting group title |
Group A – FSHD
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Reporting group description |
Starting at a dose of 0.3 mg/kg, with the potential for intrapatient dose escalation to 1.0 mg/kg over the dosing period | ||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Group B – LGMD2B and FSHD
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Reporting group description |
Starting at a dose of 0.3 mg/kg/week, with the potential for intrapatient dose escalation to a dose of 3.0 mg/kg and up to 3.0 mg/kg twice a week over the dosing period | ||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Group A – FSHD
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Reporting group description |
Starting at a dose of 0.3 mg/kg, with the potential for intrapatient dose escalation to 1.0 mg/kg over the dosing period | ||
Reporting group title |
Group B – LGMD2B and FSHD
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Reporting group description |
Starting at a dose of 0.3 mg/kg/week, with the potential for intrapatient dose escalation to a dose of 3.0 mg/kg and up to 3.0 mg/kg twice a week over the dosing period |
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End point title |
Hematocrit [1] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Hemoglobin [2] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Erythrocytes [3] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Leukocytes [4] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Platelets [5] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [5] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Neutrophils [6] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [6] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Lymphocytes [7] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [7] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Monocytes [8] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [8] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Eosinophils [9] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [9] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Basophils [10] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [10] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Aspartate Aminotransferase [11] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [11] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Alanine Aminotransferase [12] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [12] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Bilirubin [13] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [13] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Blood Urea Nitrogen [14] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [14] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Creatinine [15] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [15] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Creatine Kinase [16] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [16] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Cholesterol [17] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [17] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Sodium [18] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [18] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Potassium [19] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [19] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Bicarbonate [20] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [20] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Calcium [21] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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Notes [21] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Electrocardiogram - Heart rate [22] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline (Week 2 preinfusion) to 1-week post-treatment follow-up
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Notes [22] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Electrocardiogran - PR duration [23] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline (Week 2 preinfusion) to 1-week post-treatment follow-up
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Notes [23] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Electrocardiogram - QRS duration [24] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline (Week 2 preinfusion) to 1-week post-treatment follow-up
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Notes [24] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Electrocardiogram - QTcF Interval [25] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline (Week 2 preinfusion) to 1-week post-treatment follow-up
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Notes [25] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
PFT - FEV1/FVC Ratio [26] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Change from baseline to 4-week post-treatment follow-up
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Notes [26] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Anti-drug antibodies [27] | |||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Level of ADA titers: Screening, weeks 4, 6, 8 and 10 to 25 (follow-up period)
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Notes [27] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Anti-Jo1 antibodies [28] | |||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Screening, weeks 3 to 13, 14, 17 and 25 (follow-up period)
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Notes [28] - 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: No statistical analysis was performed for any of the primary/safety endpoints. |
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No statistical analyses for this end point |
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End point title |
Manual Muscle Testing - Overall Total Score | ||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Percent change from baseline to 1-week post-treatment follow-up
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No statistical analyses for this end point |
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End point title |
INQoL - QoL | ||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Change from baseline to 1-week post-treatment follow-up
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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 |
All AEs were to be collected and recorded from the time written informed consent is obtained through the EOS visit, or after the end of the study, if thought to be related to study drug.
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Adverse event reporting additional description |
TEAEs reported for ≥ 2 patients treated with ATYR1940 are listed in the section below.
The number of occurrences per TEAE is not available in the source data, the field "Occurrences all number" therefore corresponds to the number of subjects affected per TEAE.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
18.1
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Reporting groups
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Reporting group title |
Group A – FSHD
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Reporting group description |
Starting at a dose of 0.3 mg/kg, with the potential for intrapatient dose escalation to 1.0 mg/kg over the dosing period | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Group B – LGMD2B and FSHD
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Reporting group description |
Starting at a dose of 0.3 mg/kg/week, with the potential for intrapatient dose escalation to a dose of 3.0 mg/kg and up to 3.0 mg/kg twice a week over the dosing period | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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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08 Jan 2016 |
Protocol ATYR1940-C-004, Version 2.0, 08 January 2016 |
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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. | |||
None reported |