Clinical Trial: Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients With Muscular Dystrophy

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

Official Title: A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Biological Activity of ATYR1940 in Adult Patients With Molecularly Defi

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:

Study ATYR1940-C-002 is a multi-national, multi-center, double-blind, randomized, placebo-controlled, ascending dose study designed to evaluate the safety, tolerability, PK, immunogenicity, and pharmacodynamic effects of ATYR1940 in patients with FSHD. Up to 44 patients are planned to be enrolled at multiple study centers in the U.S. and Europe; the actual number of patients enrolled will depend on the number of cohorts initiated.

Patients will be screened for study eligibility during the Screening period within 3 weeks before Baseline (i.e., Day 1, the first day of Study Drug administration). Eligible patients, based on Screening assessments, will be randomly assigned to treatment with ATYR1940 or placebo. Patients who are randomized are considered to be enrolled in the study.

After enrollment, patients will enter the 1-week single-blind placebo period during which all patients will receive a single 30-minute IV infusion of placebo. Thereafter, patients will enter the double-blind treatment period and will receive 30-minute IV infusions of Study Drug (ATYR1940 or placebo) according to their random treatment assignment.


Sponsor: aTyr Pharma, Inc.

Current Primary Outcome:

  • Safety and tolerability of ATYR1940 in adult patients with molecularly defined genetic muscular dystrophies [ Time Frame: Up to 1 month ]
    Change from Baseline of physical examination
  • Safety and tolerability of ATYR1940 in adult patients with molecularly defined genetic muscular dystrophies [ Time Frame: Up to 1 month ]
    Change from Baseline in vital sign measurements and pulmonary evaluation
  • Safety and tolerability of ATYR1940 in adult patients with molecularly defined genetic muscular dystrophies [ Time Frame: Up to 1 month ]
    Incidences of Adverse Events (AEs), including serious and severe AEs
  • Safety and tolerability of ATYR1940 in adult patients with molecularly defined genetic muscular dystrophies [ Time Frame: Up to 1 month ]
    Change from Baseline in safety laboratory test results


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Pharmacokinetics of ATYR1940 in adult patients with molecularly defined genetic muscular dystrophies [ Time Frame: Up to 1 month ]
    Cmax: Observed maximum serum concentration
  • Pharmacokinetics of ATYR1940 in adult patients with molecularly defined genetic muscular dystrophies [ Time Frame: Up to 1 month ]
    Tmax: Time to attain maximum serum concentration
  • Pharmacokinetics of ATYR1940 in adult patients with molecularly defined genetic muscular dystrophies [ Time Frame: Up to 1 month ]
    T 1/2: Elimination half-life
  • Pharmacokinetics of ATYR1940 in adult patients with molecularly defined genetic muscular dystrophies [ Time Frame: Up to 1 month ]
    AUC 0-t: Area under the serum concentration-time curve up to time t


Original Secondary Outcome: Same as current

Information By: aTyr Pharma, Inc.

Dates:
Date Received: September 7, 2014
Date Started: August 2014
Date Completion:
Last Updated: February 27, 2017
Last Verified: February 2017