Clinical Trial: Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy

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

Official Title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dy

Brief Summary: ATYR1940-C-006 is a multi-national, multicenter study being conducted at centers in the United States (US) and Europe who participated in the Study ATYR1940-C-003 (Stage 1 only) or ATYR1940-C-004 (i.e., the parent studies).

Detailed Summary:

Study ATYR1940-C-006 is a multi-national, multi-center, open-label extension study designed to evaluate the long-term safety, effects on muscle, and PD of ATYR1940 in patients with LGMD2B or FSHD previously treated in the Protocol ATYR1940-C-003 (Stage 1 only) or ATYR1940-C-004 (i.e., the parent studies). This study will be conducted at the same study centers at which patients were enrolled in the parent studies.

Patients who completed the treatment period in the parent study; in the Investigator's opinion, demonstrated acceptable tolerability of ATYR1940, are considered by the Investigator to be compliant with ATYR1940 and the study procedures, and do not meet any criterion for ATYR1940 discontinuation are eligible for participation in the current study, contingent upon Investigator and patient agreement to continue ATYR1940 treatment.

For the first 12 weeks in this extension study, patients will receive ATYR1940 at the highest tolerated dose received in the parent study; no dose adjustments are allowed during this 12-week period. After 12 weeks, if the patient is demonstrating good tolerability, the ATYR1940 dose may be increased on a patient-specific basis at the Investigator's discretion, in consultation with the Sponsor and Medical Monitor. ATYR1940 dose increases to >3.0 mg/kg are not permissible.

All patients will receive ATYR1940 on a weekly basis in this study, regardless of the frequency of dosing in the parent study. ATYR1940 will be administered via IV infusion over 90 minutes. If medically indicated, the infusion duration and volume may be adjusted at the Investigator's discretion in consultation with the Medical Monitor and Sponsor.

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: 12-weeks ]
  Changes from Baseline in safety laboratory test results
• Changes from Baseline in pulmonary evaluation of PFTs [ Time Frame: 12-weeks ]
  Change from Baseline in pulmonary evaluation of PFTs
• Change in Baseline in ECG findings [ Time Frame: 12-weeks ]
  Change in Baseline in 12-lead electrocardiogram findings
• Change from Baseline in vital signs [ Time Frame: 12-weeks ]
  Change from Baseline in vital signs
• Immunogenicity Outcome Measure - Incidence of ADA [ Time Frame: 12-weeks ]
  Incidence of ADA titers
• Immunogenicity Outcome Measure - Incidence of Jo-1 Ab [ Time Frame: 12-weeks ]
  Incidence of Jo-1 Ab titers
• Change from Baseline in Muscle strength using MMT [ Time Frame: 12-weeks ]
  Change from Baseline in muscle strength using MMT
• Changes from Baseline in pulmonary evaluation of pulse oximetry [ Time F
  
  

  Original Primary Outcome: Same as current
  
  

  Current Secondary Outcome: Change from Baseline in CK levels [ Time Frame: 12-weeks ]

  Change from Baseline in CK levels
  
  
  

  Original Secondary Outcome: Same as current
  
  Information By: aTyr Pharma, Inc.
  

  Dates:
  Date Received: June 30, 2016
  Date Started: July 12, 2016
  Date Completion:
  Last Updated: April 20, 2017
  Last Verified: April 2017