Completed
PHASE1, PHASE2
INTERVENTIONAL
NCT02603562
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
View on ClinicalTrials.gov
Updated Dec 15, 2025
Brief Summary
The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with early onset FSHD.
Detailed Description
A Phase 1b/2 open-label, intraparticipant dose-escalation study aiming to evaluate the safety, tolerability, immunogenicity, biological and pharmacodynamic activity of intravenous ATYR1940, administered once weekly for 12 weeks, in early onset FSHD participants with signs or symptoms prior to 10 years of age.
Interventions
Name:
ATYR1940
Type:
BIOLOGICAL
Description:
Concentrate for solution for infusion
Primary Outcomes
Measure:
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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. AEs were 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.
Measure:
Number of Participants With a Clinical Laboratory Abnormality Leading to an AE
TimeFrame:
Up to End of Study (Up to Week 25)
Description:
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, erythrocyte sedimentation rate, 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.
Measure:
Number of Participants With an Ocular Abnormality Leading to a TEAE
TimeFrame:
Up to End of Study (Up to Week 25)
Description:
Ocular parameters included vitreous, retina, macula, choroid, optic nerve, and optic nerve pallor. 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 an Impact on Hearing From ATYR1940 Treatment
TimeFrame:
Up to End of Study (Up to Week 25)
Description:
Measure:
Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE
TimeFrame:
Up to End of Study (up to Week 25)
Description:
Pulmonary evaluations included pulmonary function tests and pulse oximetry. 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.
Trial Information
NCT ID
NCT02603562
Status
Completed
Study Type
INTERVENTIONAL
Phases
PHASE1, PHASE2
Sponsor
aTyr Pharma, Inc.
Last Updated
December 15, 2025