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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-001753-17 | EudraCT Number |
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The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies
Study ATYR1940-C-002 is a multi-national, multi-center, double-blind, randomized, placebo-controlled, ascending dose study designed to evaluate the safety, tolerability, PK, immunogenicity, and pharmacodynamic effects of ATYR1940 in participants with FSHD. Up to 44 participants are planned to be enrolled at multiple study centers in the United States and Europe; the actual number of participants enrolled will depend on the number of cohorts initiated.
Participants will be screened for study eligibility during the Screening period within 3 weeks before Baseline (that is, Day 1, the first day of Study Drug administration). Eligible participants, based on Screening assessments, will be randomly assigned to treatment with ATYR1940 or placebo. Participants who are randomized will be considered enrolled in the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1: ATYR1940 0.3 mg/kg | Experimental | Participants will receive ATYR1940 0.3 milligrams/kilograms (mg/kg) intravenous (IV) infusion once weekly for 4 weeks. |
|
| Cohort 2: ATYR1940 1.0 mg/kg | Experimental | Participants will receive ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks. |
|
| Cohort 3: ATYR1940 3.0 mg/kg | Experimental | Participants will receive ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks. |
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| Placebo | Placebo Comparator | Participants will receive placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Biological | Concentrate for solution for infusion |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with study drug. Worsening of a pre-existing medical condition, (that is, diabetes, migraine headaches, gout) should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. Classification of AEs was to be done by the Investigator according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Up to End of Study (up to Week 25) |
| Number of Participants With Positive Anti-Drug Antibodies (ADA) | Titers through Week 14 are summarized. For Cohorts 1 and 2, samples that screened positive but did not confirm on confirmatory assay were not titered. | Baseline up to Week 14 |
| Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result | Criterion for a positive Jo-1 Ab test result: >10.0 units/milliliter (U/mL), a cut-point associated with anti-synthetase syndrome. Criterion for an equivocal Jo-1 Ab test result: 7.0 to 10.0 U/mL. Participants were required to have a negative Jo-1 Ab test result (defined as <7 U/mL) for inclusion in the study as well as to continue dosing with study drug during the study. | Baseline up to Week 14 |
| Number of Participants With a Clinically Significant Laboratory Abnormality | Laboratory parameters included hematology (hematocrit, hemoglobin, platelet count, red blood cell count, white blood cell count, neutrophils, lymphocytes, monocytes, eosinophils, basophils); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase (GGT), alkaline phosphatase, sodium, total protein, bicarbonate, potassium, calcium, chloride, magnesium, inorganic phosphate, creatine phosphokinase [will be fractionated if elevated], lactic dehydrogenase, erythrocyte sedimentation rate, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, cholesterol [non-fasting]); Urinalysis (Color, pH, specific gravity, protein, glucose, ketones, blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious AEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 6 and Week 14 | MMT was graded on a scale from 0 (no movement) to 10 (normal movement). Each side of the body and the position in which each muscle was tested were recorded for each participant. The total MMT score were calculated by averaging a converted MMT scores across all tested muscle groups. Decreased motor function was indicated by decreased individual muscle or composite MMT score. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Sanjay Shukla, MD | aTyr Pharma | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| aTyr Pharma Investigative Site | Rochester | New York | 14642 | United States | ||
| aTyr Pharma Investigative Site |
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1: ATYR1940 0.3 mg/kg | Participants received ATYR1940 0.3 milligrams/kilograms (mg/kg) intravenous (IV) infusion once weekly for 4 weeks. |
| FG001 | Cohort 2: ATYR1940 1.0 mg/kg |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ATYR1940 |
| Biological |
Concentrate for solution for infusion |
|
| Baseline up to Week 14 |
| Number of Participants With a Physical Examination Abnormality | Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat (HEENT), cardiovascular system, respiratory system, chest (breasts), gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, and reflexes, and sensory testing. The body systems with at least 1 physical abnormality is presented. One participant could be represented in more than 1 body system category. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Up to End of Study (up to Week 25) |
| Number of Participants With a Vital Sign-Related Event Resulting in a TEAE | The vital sign parameters that were evaluated included heart rate, systolic and diastolic blood pressure, and respiration rate as well as temperature. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Up to End of Study (Up to Week 25) |
| Number of Participants With a Pulmonary Function Event Resulting in a TEAE | Pulmonary function testing included measurements of forced vital capacity (FVC) and forced expiratory volume in 1 second (FEV1). A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Up to End of Study (Up to Week 25) |
| Baseline, Week 6 and Week 14 |
| Change From Baseline in Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL at Week 6 and Week 14 | The INQoL is a validated muscle disease-specific measure of quality of life. The self-administered questionnaire consisted of 45 questions with 4 domains measuring the impact of common muscle disease symptoms (weakness, locking [or myotonia], pain, and fatigue); 5 domains measuring the influence of the muscle disease on particular areas of life (activities, independence, relationships, emotions, and body image); and the last domain focused on the positive and negative effects of treatment and was divided into 2 scores. All responses were given in a 7-point Likert scale, with improved QoL indicated by decreased scores. Overall QoL score was calculated from preselected 5 domains (activities, independence, relationships, emotions, and body image) by adding the scores from each domain. In summary, INQoL yielded 11 scores and 1 QoL score. The Overall scoring used a scale of 0-100, with improved QoL indicated by decreased scores. | Baseline, Week 6 and Week 14 |
| Maximum Observed Plasma Concentration (Cmax) of ATYR1940 | Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only) |
| Time to Reach Maximum Observed Plasma Concentration (Tmax) of ATYR1940 | Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only) |
| Area Under the Plasma Concentration Time Curve From Time 0 to Time t (AUC 0-t) of ATYR1940 | Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only) |
| Average of Half-life (T1/2) of ATYR1940 | Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only) |
| Columbus |
| Ohio |
| 43210 |
| United States |
| aTyr Pharma Investigative Site | Marseille | France |
| aTyr Pharma Investigative Site | Rome | Italy |
| aTyr Pharma Investigative Site | Nijmegen | Netherlands |
Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.
| FG002 | Cohort 3: ATYR1940 3.0 mg/kg | Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks. |
| FG003 | Placebo | Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. |
| Received at Least One Dose of Study Drug |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
Intent to treat (ITT) analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1: ATYR1940 0.3 mg/kg | Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks. |
| BG001 | Cohort 2: ATYR1940 1.0 mg/kg | Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks. |
| BG002 | Cohort 3: ATYR1940 3.0 mg/kg | Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks. |
| BG003 | Placebo | Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Full Range | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with study drug. Worsening of a pre-existing medical condition, (that is, diabetes, migraine headaches, gout) should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. Classification of AEs was to be done by the Investigator according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Posted | Count of Participants | Participants | Up to End of Study (up to Week 25) |
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| |||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With Positive Anti-Drug Antibodies (ADA) | Titers through Week 14 are summarized. For Cohorts 1 and 2, samples that screened positive but did not confirm on confirmatory assay were not titered. | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Posted | Count of Participants | Participants | Baseline up to Week 14 |
| |||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result | Criterion for a positive Jo-1 Ab test result: >10.0 units/milliliter (U/mL), a cut-point associated with anti-synthetase syndrome. Criterion for an equivocal Jo-1 Ab test result: 7.0 to 10.0 U/mL. Participants were required to have a negative Jo-1 Ab test result (defined as <7 U/mL) for inclusion in the study as well as to continue dosing with study drug during the study. | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Posted | Count of Participants | Participants | Baseline up to Week 14 |
| |||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With a Clinically Significant Laboratory Abnormality | Laboratory parameters included hematology (hematocrit, hemoglobin, platelet count, red blood cell count, white blood cell count, neutrophils, lymphocytes, monocytes, eosinophils, basophils); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase (GGT), alkaline phosphatase, sodium, total protein, bicarbonate, potassium, calcium, chloride, magnesium, inorganic phosphate, creatine phosphokinase [will be fractionated if elevated], lactic dehydrogenase, erythrocyte sedimentation rate, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, cholesterol [non-fasting]); Urinalysis (Color, pH, specific gravity, protein, glucose, ketones, blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious AEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Posted | Count of Participants | Participants | Baseline up to Week 14 |
| |||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With a Physical Examination Abnormality | Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat (HEENT), cardiovascular system, respiratory system, chest (breasts), gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, and reflexes, and sensory testing. The body systems with at least 1 physical abnormality is presented. One participant could be represented in more than 1 body system category. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. Here, 'Overall Number of Participants Analyzed' (N) signifies number of participants evaluable for this outcome measure. | Posted | Count of Participants | Participants | Up to End of Study (up to Week 25) |
| |||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With a Vital Sign-Related Event Resulting in a TEAE | The vital sign parameters that were evaluated included heart rate, systolic and diastolic blood pressure, and respiration rate as well as temperature. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Posted | Count of Participants | Participants | Up to End of Study (Up to Week 25) |
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| Primary | Number of Participants With a Pulmonary Function Event Resulting in a TEAE | Pulmonary function testing included measurements of forced vital capacity (FVC) and forced expiratory volume in 1 second (FEV1). A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Posted | Count of Participants | Participants | Up to End of Study (Up to Week 25) |
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| Secondary | Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 6 and Week 14 | MMT was graded on a scale from 0 (no movement) to 10 (normal movement). Each side of the body and the position in which each muscle was tested were recorded for each participant. The total MMT score were calculated by averaging a converted MMT scores across all tested muscle groups. Decreased motor function was indicated by decreased individual muscle or composite MMT score. | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints. | Posted | Mean | Full Range | percent change | Baseline, Week 6 and Week 14 |
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| Secondary | Change From Baseline in Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL at Week 6 and Week 14 | The INQoL is a validated muscle disease-specific measure of quality of life. The self-administered questionnaire consisted of 45 questions with 4 domains measuring the impact of common muscle disease symptoms (weakness, locking [or myotonia], pain, and fatigue); 5 domains measuring the influence of the muscle disease on particular areas of life (activities, independence, relationships, emotions, and body image); and the last domain focused on the positive and negative effects of treatment and was divided into 2 scores. All responses were given in a 7-point Likert scale, with improved QoL indicated by decreased scores. Overall QoL score was calculated from preselected 5 domains (activities, independence, relationships, emotions, and body image) by adding the scores from each domain. In summary, INQoL yielded 11 scores and 1 QoL score. The Overall scoring used a scale of 0-100, with improved QoL indicated by decreased scores. | Per-Protocol (PP) analysis set included all participants in the ITT analysis set with no major protocol deviations and ≥1 post-baseline pharmacodynamic (PD) or clinical parameter assessed. Change from baseline included only those participants with both a baseline value and a value for the summarized time period. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints. | Posted | Mean | Full Range | score on a scale | Baseline, Week 6 and Week 14 |
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| Secondary | Maximum Observed Plasma Concentration (Cmax) of ATYR1940 | Pharmacokinetic (PK) analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints. | Posted | Mean | Standard Deviation | nanogram/milliliter | Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only) |
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| Secondary | Time to Reach Maximum Observed Plasma Concentration (Tmax) of ATYR1940 | PK analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints. | Posted | Mean | Standard Deviation | hours | Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only) |
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| Secondary | Area Under the Plasma Concentration Time Curve From Time 0 to Time t (AUC 0-t) of ATYR1940 | PK analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints. | Posted | Mean | Standard Deviation | nanogram*hour/milliliter | Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only) |
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| Secondary | Average of Half-life (T1/2) of ATYR1940 | PK analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints. | Posted | Mean | Standard Deviation | hours | Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only) |
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Up to End of Study (up to Week 25)
ITT Analysis Set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1: ATYR1940 0.3 mg/kg | Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks. | 0 | 3 | 3 | 3 | ||
| EG001 | Cohort 2: ATYR1940 1.0 mg/kg | Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks. | 0 | 6 | 6 | 6 | ||
| EG002 | Cohort 3: ATYR1940 3.0 mg/kg | Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks. | 1 | 6 | 6 | 6 | ||
| EG003 | Placebo | Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. | 0 | 5 | 5 | 5 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infusion-related Reaction | General disorders | MedDRA 18.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eyelid Irritation | Eye disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Scintillating Scotoma | Eye disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Chills | General disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Infusion Site Erythema | General disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Malaise | General disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Non-Cardiac Chest Pain | General disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Seasonal Allergy | Immune system disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 18.0 | Non-systematic Assessment |
| |
| Skin Abrasion | Injury, poisoning and procedural complications | MedDRA 18.0 | Non-systematic Assessment |
| |
| Thermal Burn | Injury, poisoning and procedural complications | MedDRA 18.0 | Non-systematic Assessment |
| |
| Blood Pressure Increased | Investigations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Electrocardiogram PR Shortened | Investigations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Electrocardiogram QT Prolonged | Investigations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Electrocardiogram T Wave Inversion | Investigations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Neutrophil Count Decreased | Investigations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Protein Urine Present | Investigations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Pulmonary Function Test Abnormal | Investigations | MedDRA 18.0 | Non-systematic Assessment |
| |
| White Blood Cell Count Decreased | Investigations | MedDRA 18.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Muscular Weakness | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Musculoskeletal Pain | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Pain in Extremity | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Polymenorrhoea | Reproductive system and breast disorders | MedDRA 18.0 | Non-systematic Assessment | This Adverse event is gender specific. |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Skin Lesion | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Hot Flush | Vascular disorders | MedDRA 18.0 | Non-systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 18.0 | Non-systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Gennyne Walker | aTyR Pharma | 858 731 8389 | clinicaltrials@atyrpharma.com |
| ID | Term |
|---|---|
| D020391 | Muscular Dystrophy, Facioscapulohumeral |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| Male |
|
|
|
Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. |
|
|
| OG002 | Cohort 3: ATYR1940 3.0 mg/kg | Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks. |
| OG003 | Placebo | Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. |
|
|
| OG002 |
| Cohort 3: ATYR1940 3.0 mg/kg |
Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks. |
| OG003 | Placebo | Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. |
|
|
Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.
|
|
Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.
|
|
| OG003 |
| Placebo |
Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. |
|
|
| OG001 | Cohort 2: ATYR1940 1.0 mg/kg | Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks. |
| OG002 | Cohort 3: ATYR1940 3.0 mg/kg | Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks. |
| OG003 | Placebo | Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. |
|
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