Clinical Trial Results:
An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)
Summary
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EudraCT number |
2015-001912-36 |
Trial protocol |
NL |
Global end of trial date |
14 Mar 2017
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Results information
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Results version number |
v1(current) |
This version publication date |
23 Dec 2018
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First version publication date |
23 Dec 2018
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Other versions |
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Summary report(s) |
ATYR1940-C-005 CSR synopsis |
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-005
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
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WHO universal trial number (UTN) |
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Other trial identifiers |
IND Number: 122045 | ||
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 |
03 Jan 2018
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
14 Mar 2017
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Global end of trial reached? |
Yes
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Global end of trial date |
14 Mar 2017
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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 |
Evaluate the safety, tolerability, and immunogenicity of long-term treatment with intravenous (IV) ATYR1940 in adult patients with facioscapulohumeral muscular dystrophy (FSHD) previously enrolled in clinical study ATYR1940-C-002.
All clinically significant laboratory abnormalities were reported as adverse events and therefore appear in the Adverse events section of this dataset. As a consequence, the endpoints reported in this dataset are limited to the most relevant safety endpoints.
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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 |
31 Jul 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 |
Netherlands: 5
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Country: Number of subjects enrolled |
Italy: 3
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Country: Number of subjects enrolled |
United States: 1
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Worldwide total number of subjects |
9
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EEA total number of subjects |
8
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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) |
6
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From 65 to 84 years |
3
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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 |
Patients who were enrolled in ≥Cohort 3 and completed the parent study ATYR1940-C-002 through Week 13; demonstrated acceptable tolerability of study drug; were considered by the Investigator to be compliant with study drug and the study procedures; and did not meet any criterion for discontinuation. | ||||||
Period 1
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Period 1 title |
Overall trial (overall period)
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Is this the baseline period? |
Yes | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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All patients - 3 mg/kg | ||||||
Arm description |
- | ||||||
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 |
Study drug has been dispensed for the patient according to their last dose assignment in the parent study.
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Baseline characteristics reporting groups
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Reporting group title |
Overall trial
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
All patients - 3 mg/kg
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Reporting group description |
- |
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End point title |
Incidence of Treatment Emergent Adverse Events (TEAEs) [1] | ||||||
End point description |
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End point type |
Primary
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End point timeframe |
All study visits until the end of the study
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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 |
Anti-drug antibodies (ADA) [2] | ||||||
End point description |
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End point type |
Primary
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End point timeframe |
Eligibility check, Weeks 2 to 4 then every 3 months, 1-week follow-up, 4-week follow-up, 12-week 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 |
Jo-1 antibodies [3] | ||||||
End point description |
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End point type |
Primary
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End point timeframe |
Eligibility check, weekly from Week 2, 1-week follow-up, 4-week follow-up, 12-week 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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Adverse events information
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Timeframe for reporting adverse events |
From informed consent to End of Study visit.
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Adverse event reporting additional description |
Due to the small study size (N=9), 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 |
All patients - 3 mg/kg
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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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04 Nov 2015 |
Version 2.0 (Amendment 1; 04 November 2015) |
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11 Jan 2016 |
Version 3.0 (Amendment 2; 11 January 2016) |
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09 Apr 2016 |
Version 4.0 (Amendment 3; 09 April 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 |