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