Trial Outcomes & Findings for Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension (NCT NCT02531217)
NCT ID: NCT02531217
Last Updated: 2023-08-18
Results Overview
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.
COMPLETED
PHASE1/PHASE2
9 participants
Up to End of Study (up to Week 36)
2023-08-18
Participant Flow
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.
Participant milestones
| Measure |
ATYR1940
Participants received ATYR1940 3.0 milligrams per kilograms (mg/kg) intravenous (IV) infusion once weekly for 24 weeks.
|
|---|---|
|
Overall Study
STARTED
|
9
|
|
Overall Study
Received at Least One Dose of Study Drug
|
9
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension
Baseline characteristics by cohort
| Measure |
ATYR1940
n=9 Participants
Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 24 weeks.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=5 Participants
|
|
Age, Continuous
|
53.2 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to End of Study (up to Week 36)Population: ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.
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.
Outcome measures
| Measure |
ATYR1940
n=9 Participants
Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 24 weeks.
|
|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
TEAEs
|
9 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
SAEs
|
0 Participants
|
PRIMARY outcome
Timeframe: Up to End of Study (up to Week 36)Population: ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.
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.
Outcome measures
| Measure |
ATYR1940
n=9 Participants
Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 24 weeks.
|
|---|---|
|
Number of Participants With Clinically Significant Physical Examination Abnormality
|
4 Participants
|
PRIMARY outcome
Timeframe: Up to End of Study (up to Week 36)Population: ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.
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.
Outcome measures
| Measure |
ATYR1940
n=9 Participants
Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 24 weeks.
|
|---|---|
|
Number of Participants With a Vital Sign-Related Event Resulting in a TEAE
|
0 Participants
|
PRIMARY outcome
Timeframe: Up to End of Study (up to Week 36)Population: ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.
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.
Outcome measures
| Measure |
ATYR1940
n=9 Participants
Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 24 weeks.
|
|---|---|
|
Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE
|
0 Participants
|
PRIMARY outcome
Timeframe: Up to End of Study (Up to Week 36)Population: ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.
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.
Outcome measures
| Measure |
ATYR1940
n=9 Participants
Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 24 weeks.
|
|---|---|
|
Number of Participants With a Clinical Laboratory Abnormality Resulting in an AE
|
3 Participants
|
Adverse Events
ATYR1940
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
ATYR1940
n=9 participants at risk
Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 24 weeks.
|
|---|---|
|
Ear and labyrinth disorders
Ear Pain
|
11.1%
1/9 • 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.
|
|
Ear and labyrinth disorders
Vertigo
|
11.1%
1/9 • 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.
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
22.2%
2/9 • 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.
|
|
Gastrointestinal disorders
Abdominal Pain Lower
|
11.1%
1/9 • 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.
|
|
Gastrointestinal disorders
Constipation
|
11.1%
1/9 • 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.
|
|
Gastrointestinal disorders
Diarrhoea
|
22.2%
2/9 • 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.
|
|
Gastrointestinal disorders
Flatulence
|
11.1%
1/9 • 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.
|
|
Gastrointestinal disorders
Nausea
|
11.1%
1/9 • 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.
|
|
Gastrointestinal disorders
Stomatitis
|
11.1%
1/9 • 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.
|
|
Gastrointestinal disorders
Toothache
|
11.1%
1/9 • 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.
|
|
Gastrointestinal disorders
Vomiting
|
22.2%
2/9 • 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.
|
|
General disorders
Asthenia
|
11.1%
1/9 • 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.
|
|
General disorders
Fatigue
|
22.2%
2/9 • 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.
|
|
General disorders
Infusion Site Erythema
|
11.1%
1/9 • 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.
|
|
General disorders
Non-Cardiac Chest Pain
|
11.1%
1/9 • 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.
|
|
General disorders
Oedema Peripheral
|
11.1%
1/9 • 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.
|
|
General disorders
Pain
|
11.1%
1/9 • 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.
|
|
Infections and infestations
Ear Infection
|
11.1%
1/9 • 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.
|
|
Infections and infestations
Eye Infection
|
11.1%
1/9 • 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.
|
|
Infections and infestations
Influenza
|
33.3%
3/9 • 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.
|
|
Infections and infestations
Nasopharyngitis
|
22.2%
2/9 • 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.
|
|
Injury, poisoning and procedural complications
Infusion Related Reaction
|
22.2%
2/9 • 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.
|
|
Injury, poisoning and procedural complications
Muscle Injury
|
11.1%
1/9 • 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.
|
|
Injury, poisoning and procedural complications
Sunburn
|
11.1%
1/9 • 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.
|
|
Injury, poisoning and procedural complications
Wound
|
22.2%
2/9 • 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.
|
|
Investigations
Alanine Aminotransferase Increased
|
11.1%
1/9 • 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.
|
|
Investigations
Aspartate Aminotransferase Increased
|
11.1%
1/9 • 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.
|
|
Investigations
Electrocardiogram QT Prolonged
|
11.1%
1/9 • 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.
|
|
Investigations
Hepatic Enzyme Increased
|
22.2%
2/9 • 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.
|
|
Investigations
Oxygen Saturation Decreased
|
11.1%
1/9 • 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.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
22.2%
2/9 • 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.
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
33.3%
3/9 • 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.
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
11.1%
1/9 • 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.
|
|
Musculoskeletal and connective tissue disorders
Jaw Cyst
|
11.1%
1/9 • 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.
|
|
Musculoskeletal and connective tissue disorders
Muscle Swelling
|
11.1%
1/9 • 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.
|
|
Musculoskeletal and connective tissue disorders
Muscle Tightness
|
11.1%
1/9 • 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.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
44.4%
4/9 • 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.
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
22.2%
2/9 • 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.
|
|
Nervous system disorders
Dizziness
|
11.1%
1/9 • 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.
|
|
Nervous system disorders
Dysstasia
|
11.1%
1/9 • 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.
|
|
Nervous system disorders
Headache
|
66.7%
6/9 • 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.
|
|
Nervous system disorders
Hypoaesthesia
|
11.1%
1/9 • 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.
|
|
Nervous system disorders
Paraesthesia
|
11.1%
1/9 • 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.
|
|
Nervous system disorders
Presyncope
|
11.1%
1/9 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
11.1%
1/9 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
11.1%
1/9 • 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.
|
|
Skin and subcutaneous tissue disorders
Blister
|
11.1%
1/9 • 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.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
11.1%
1/9 • 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.
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
11.1%
1/9 • 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.
|
|
Skin and subcutaneous tissue disorders
Macule
|
11.1%
1/9 • 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.
|
|
Skin and subcutaneous tissue disorders
Papule
|
11.1%
1/9 • 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.
|
|
Skin and subcutaneous tissue disorders
Rash
|
11.1%
1/9 • 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.
|
|
Vascular disorders
Flushing
|
11.1%
1/9 • 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.
|
|
Vascular disorders
Haematoma
|
11.1%
1/9 • 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.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place