Clinical Trial: Safety, Tolerability, PK, and Activity of ATYR1940 in Patients With Muscular Dystrophy - Study Extension

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients With Fascioscapulohumeral M

Brief Summary: The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult patients with molecularly defined genetic muscular dystrophies.

Detailed Summary: The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult patients with molecularly defined genetic muscular dystrophies, and to additionally explore the pharmacokinetics and biologic activity of ATYR1940 in adult patients with molecularly defined genetic muscular dystrophies. Patients who successfully complete a parent study (NCT02239224) are eligible for enrollment into this long-term extension study.
Sponsor: aTyr Pharma, Inc.

Current Primary Outcome:

  • Safety Primary Outcome Measure - Change from baseline in physical examination [ Time Frame: 6 months ]
    Change from baseline in physical examination
  • Safety Primary Outcome Measure - Change from Baseline in vital sign measurements (blood pressure, pulse, respiratory rate, and body temperature) and pulmonary evaluation [ Time Frame: 6 months ]
    Change from Baseline in vital sign measurements (blood pressure, pulse, respiratory rate, and body temperature) and pulmonary evaluation
  • Tolerability Primary Outcome Measure - Incidences of Adverse Events (AEs), including serious and severe AEs [ Time Frame: 6 months ]
    Incidences of Adverse Events (AEs), including serious and severe AEs
  • Safety Primary Outcome Measure - Change from Baseline in safety laboratory test results (clinical chemistry, hematology, and urinalysis) [ Time Frame: 6 months ]
    Change from Baseline in safety laboratory test results (clinical chemistry, hematology, and urinalysis)


Original Primary Outcome: Same as current

Current Secondary Outcome: Immunogenicity Additional Outcome Measure - Serum-based muscle biomarkers [ Time Frame: 6 months ]

Serum-based muscle biomarkers


Original Secondary Outcome: Same as current

Information By: aTyr Pharma, Inc.

Dates:
Date Received: June 25, 2015
Date Started: September 2015
Date Completion: August 2018
Last Updated: May 15, 2017
Last Verified: May 2017