Clinical Trial: The Safety and Biological Activity of ATYR1940 in Patients With Limb Girdle or Facioscapulohumeral Muscular Dystrophies

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 Limb Girdle and Facioscapulohumeral Muscula

Brief Summary: The purpose of this study is to assess the safety and biological activity of ATYR1940 in patients with Limb Girdle (LGMD2B) or Facioscapulohumeral Muscular Dystrophy (FSHD).

Detailed Summary: ATYR1940-C-004 is a Phase1b/2 open-label intrapatient study dose escalation study aiming to evaluate the safety, tolerability, immunogenicity, biological activity of intravenous ATYR1940, administered once weekly for 8 weeks, then twice a weekly for 4 weeks in adult patients with LGMD2B and FSHD. Approximately 8 LGMD2B and 8 FHSD patients will be enrolled.
Sponsor: aTyr Pharma, Inc.

Current Primary Outcome:

• Tolerability Primary Outcome Measure - incidence of treatment-emergent adverse events (AEs) and serious adverse events overal (SAEs) overall and by intensity [ Time Frame: 12 weeks ]
  Incidences of Adverse Events (AEs), including serious and severe AEs
• Safety Primary Outcome Measure - change from Baseline in safety laboratory test results [ Time Frame: 12 weeks ]
  Change from Baseline in safety laboratory test results (clinical chemistry, hematology, and urinalysis)
• Safety Primary Outcome Measure - change from Baseline in ECG [ Time Frame: 12 weeks ]
  Change from Baseline in 12-lead ECG findings
• Safety Primary Outcome Measure - change from Baseline in vital signs measurements and pulmonary evaluations [ Time Frame: 12 week ]
  Change from Baseline in vital signs measurements (blood pressure, pulse, respiratory rate, and body temperature) and pulmonary evaluations (PFTs and pulse oximetry)

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Pharmacodynamic Additional Outcome Measure - changes in muscular dystrophy-related inflammatory immune state [ Time Frame: 12 weeks ]
  Effects assessed by changes in muscular dystrophy-related inflammatory immune state in peripheral blood
• Pharmacodynamic Additional Outcome Measure - changes in serum- and/or plasma-based muscle biomarkers. [ Time Frame: 12 weeks ]
  Serum-based muscle biomarkers

Original Secondary Outcome: Same as current

Information By: aTyr Pharma, Inc.

Dates:
Date Received: September 28, 2015
Date Started: November 5, 2015
Date Completion:
Last Updated: May 15, 2017
Last Verified: May 2017