Completed
PHASE1, PHASE2
INTERVENTIONAL
NCT02239224
Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy
View on ClinicalTrials.gov
Updated Dec 15, 2025
Brief Summary
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
Detailed Description
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.
Interventions
Name:
Placebo
Type:
BIOLOGICAL
Description:
Concentrate for solution for infusion
Name:
ATYR1940
Type:
BIOLOGICAL
Description:
Concentrate for solution for infusion
Primary Outcomes
Measure:
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
TimeFrame:
Up to End of Study (up to Week 25)
Description:
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.
Measure:
Number of Participants With Positive Anti-Drug Antibodies (ADA)
TimeFrame:
Baseline up to Week 14
Description:
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.
Measure:
Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result
TimeFrame:
Baseline up to Week 14
Description:
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.
Measure:
Number of Participants With a Clinically Significant Laboratory Abnormality
TimeFrame:
Baseline up to Week 14
Description:
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:
Number of Participants With a Physical Examination Abnormality
TimeFrame:
Up to End of Study (up to Week 25)
Description:
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.
Measure:
Number of Participants With a Vital Sign-Related Event Resulting in a TEAE
TimeFrame:
Up to End of Study (Up to Week 25)
Description:
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.
Measure:
Number of Participants With a Pulmonary Function Event Resulting in a TEAE
TimeFrame:
Up to End of Study (Up to Week 25)
Description:
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.
Trial Information
NCT ID
NCT02239224
Status
Completed
Study Type
INTERVENTIONAL
Phases
PHASE1, PHASE2
Sponsor
aTyr Pharma, Inc.
Last Updated
December 15, 2025