Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2015-001912-36 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
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, and to additionally explore the pharmacokinetics and biologic activity of ATYR1940 in adult participants with molecularly defined genetic muscular dystrophies. Participants who successfully complete the parent study (NCT02239224) are eligible for enrollment into this long-term extension study.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ATYR1940 | Experimental | Participants will receive ATYR1940 3.0 milligrams per kilograms (mg/kg) intravenous (IV) infusion once weekly for 24 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ATYR1940 | Biological | Concentrate for solution for infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. AEs ware defined as any untoward medical occurrence in a participant administered study drug and that does not necessarily have a causal relationship with the study drug. Worsening of a pre-existing medical condition 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. SAEs were defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required in-participant hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in 'Reported Adverse Events' Section. | Up to End of Study (up to Week 36) |
| Number of Participants With Clinically Significant Physical Examination Abnormality | Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat, cardiovascular system, respiratory system, gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, reflexes, and sensory testing. 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 36) |
| Number of Participants With a Vital Sign-Related Event Resulting in a TEAE | The vital sign parameters that were evaluated included blood pressure, pulse and respiration rates, and body temperature. Vital signs abnormalities that were considered by the Investigator to be clinically significant were reported as AEs if the finding represented a change from baseline. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
Not provided
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| aTyr Pharma Investigative Site | Columbus | Ohio | 43210 | United States | ||
| aTyr Pharma Investigative Site |
Participants who participated in Cohort 2 or 3, completed the double-blind treatment period and were considered to be compliant with the study drug by the Investigator in the Study ATYR1940-C-002 (NCT02239224) were eligible for participation in this study.
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | ATYR1940 | Participants received ATYR1940 3.0 milligrams per kilograms (mg/kg) intravenous (IV) infusion once weekly for 24 weeks. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Intent to treat (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 |
|---|---|---|
| BG000 | ATYR1940 | Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 24 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of 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) and Serious Adverse Events (SAEs) | TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. AEs ware defined as any untoward medical occurrence in a participant administered study drug and that does not necessarily have a causal relationship with the study drug. Worsening of a pre-existing medical condition 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. SAEs were defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required in-participant hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in '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 36) |
Up to End of Study (up to Week 36)
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 | ATYR1940 | Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 24 weeks. |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ear Pain | Ear and labyrinth disorders | MedDRA 18.1 | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | aTyr Pharma | 858 731 8389 | clinicaltrials@atyrpharma.com |
Not provided
| ID | Term |
|---|---|
| D020391 | Muscular Dystrophy, Facioscapulohumeral |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Up to End of Study (up to Week 36) |
| Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE | Pulmonary evaluations included pulmonary function tests. Clinically significant changes were to be reported as adverse events. 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 36) |
| Number of Participants With a Clinical Laboratory Abnormality Resulting in an AE | Laboratory parameters included hematology (hematocrit, hemoglobin, red blood cell count, white blood cell count with differential [neutrophils, lymphocytes, monocytes, eosinophils, basophils], and platelet count); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, alkaline phosphatase, total protein, sodium, potassium, bicarbonate, calcium, chloride, magnesium, inorganic phosphate, creatine kinase, lactate dehydrogenase, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, and cholesterol [nonfasting]); and urinalysis (color, pH, specific gravity, protein, glucose, ketones, and blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. 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 36) |
| Rome |
| 00168 |
| Italy |
| aTyr Pharma Investigative Site | Nijmegen | Netherlands |
| Participants |
|
| Age, Continuous | Mean | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| ID | Title | Description |
|---|---|---|
| OG000 | ATYR1940 | Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 24 weeks. |
|
|
| Primary | Number of Participants With Clinically Significant Physical Examination Abnormality | Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat, cardiovascular system, respiratory system, gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, reflexes, and sensory testing. 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 36) |
|
|
|
| Primary | Number of Participants With a Vital Sign-Related Event Resulting in a TEAE | The vital sign parameters that were evaluated included blood pressure, pulse and respiration rates, and body temperature. Vital signs abnormalities that were considered by the Investigator to be clinically significant were reported as AEs if the finding represented a change from baseline. 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 36) |
|
|
|
| Primary | Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE | Pulmonary evaluations included pulmonary function tests. Clinically significant changes were to be reported as adverse events. 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 36) |
|
|
|
| Primary | Number of Participants With a Clinical Laboratory Abnormality Resulting in an AE | Laboratory parameters included hematology (hematocrit, hemoglobin, red blood cell count, white blood cell count with differential [neutrophils, lymphocytes, monocytes, eosinophils, basophils], and platelet count); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, alkaline phosphatase, total protein, sodium, potassium, bicarbonate, calcium, chloride, magnesium, inorganic phosphate, creatine kinase, lactate dehydrogenase, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, and cholesterol [nonfasting]); and urinalysis (color, pH, specific gravity, protein, glucose, ketones, and blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. 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 36) |
|
|
|
| 0 |
| 9 |
| 9 |
| 9 |
| Vertigo | Ear and labyrinth disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Abdominal Pain Lower | Gastrointestinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Stomatitis | Gastrointestinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Asthenia | General disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Fatigue | General disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Infusion Site Erythema | General disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Non-Cardiac Chest Pain | General disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Oedema Peripheral | General disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Pain | General disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Ear Infection | Infections and infestations | MedDRA 18.1 | Non-systematic Assessment |
|
| Eye Infection | Infections and infestations | MedDRA 18.1 | Non-systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 18.1 | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 18.1 | Non-systematic Assessment |
|
| Infusion Related Reaction | Injury, poisoning and procedural complications | MedDRA 18.1 | Non-systematic Assessment |
|
| Muscle Injury | Injury, poisoning and procedural complications | MedDRA 18.1 | Non-systematic Assessment |
|
| Sunburn | Injury, poisoning and procedural complications | MedDRA 18.1 | Non-systematic Assessment |
|
| Wound | Injury, poisoning and procedural complications | MedDRA 18.1 | Non-systematic Assessment |
|
| Alanine Aminotransferase Increased | Investigations | MedDRA 18.1 | Non-systematic Assessment |
|
| Aspartate Aminotransferase Increased | Investigations | MedDRA 18.1 | Non-systematic Assessment |
|
| Electrocardiogram QT Prolonged | Investigations | MedDRA 18.1 | Non-systematic Assessment |
|
| Hepatic Enzyme Increased | Investigations | MedDRA 18.1 | Non-systematic Assessment |
|
| Oxygen Saturation Decreased | Investigations | MedDRA 18.1 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Jaw Cyst | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Muscle Swelling | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Muscle Tightness | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Pain in Extremity | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Dysstasia | Nervous system disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Hypoaesthesia | Nervous system disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Presyncope | Nervous system disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Blister | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Macule | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Papule | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Flushing | Vascular disorders | MedDRA 18.1 | Non-systematic Assessment |
|
| Haematoma | Vascular disorders | MedDRA 18.1 | Non-systematic Assessment |
|
Not provided
Not provided
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |