Clinical Trial: Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular Dystrophy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset

Brief Summary: The purpose of this study is to assess the safety and biological activity of ATYR1940 in patients with early onset facioscapulohumeral muscular dystrophy (FSHD).

Detailed Summary: A Phase 1b/2 open-label, intrapatient 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 patients with signs or symptoms prior to 10 years of age. In Stage 1, up to 8 patients between the ages of 16 and 25 years will be enrolled. Stage 2 of enrollment will include patients with early onset FSHD between the ages of 12 and 15 years.
Sponsor: aTyr Pharma, Inc.

Current Primary Outcome:

  • Incidences of Treatment-Emergent adverse events and serious adverse events [ Time Frame: 12 weeks ]
    Incidences of adverse events including serious and severe adverse events overall and by intensity
  • Changes from Baseline in safety laboratory test results [ Time Frame: Changes from Baseline after 12 weeks ]
    Changes from Baseline in safety laboratory test results
  • Changes from Baseline in pulmonary evaluation [ Time Frame: Changes from Baseline after 12 week ]
    Change from Baseline in pulmonary evaluation
  • Changes from Baseline in visual assessment [ Time Frame: Changes from Baseline after 12 weeks ]
    Changes in Baseline in visual acuity
  • Changes from Baseline in hearing [ Time Frame: Changes from Baseline after 12 weeks ]
    Safety Primary Outcome Measure - Changes from Baseline in hearing based on audiometry


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Immunogenicity Outcome Measure - Incidence and level of ADA [ Time Frame: 12 weeks ]
    Incidence and level of ADA titers
  • Immunogenicity Outcome Measure - Incidence and level of Jo-1 Ab [ Time Frame: 12 weeks ]
    Incidence and level of Jo-1 Ab titers
  • Immunogenicity Outcome Measure - Incidence of infusion reactions [ Time Frame: 12 weeks ]
    Incidence of infusion reactions


Original Secondary Outcome: Same as current

Information By: aTyr Pharma, Inc.

Dates:
Date Received: November 5, 2015
Date Started: March 30, 2016
Date Completion:
Last Updated: May 15, 2017
Last Verified: May 2017