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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
aTyr Pharma, Inc.
ClinicalTrials.gov Identifier:
NCT02531217
First received: June 25, 2015
Last updated: May 15, 2017
Last verified: May 2017
  Purpose
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.

Condition Intervention Phase
Facioscapulohumeral Muscular Dystrophy Biological: ATYR1940 Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Muscular Dystrophy (FSHD)

Resource links provided by NLM:


Further study details as provided by aTyr Pharma, Inc.:

Primary Outcome Measures:
  • 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)


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


Estimated Enrollment: 32
Study Start Date: September 2015
Estimated Study Completion Date: August 2018
Estimated Primary Completion Date: May 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ATYR1940
ATYR1940 will be given intravenously at a dose of 3.0 mg/kg, weekly
Biological: ATYR1940
Biological: ATYR1940

Detailed Description:
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.
  Eligibility

Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient has an established, genetically-confirmed, diagnosis of facioscapulohumeral dystrophy with clinical findings meeting existing criteria
  • Patient is a male or female aged 18 to 65 years, inclusive.
  • Patients who previously participated in Study ATYR1940-C-002 and who meet the entry criteria above for the current study will be eligible for enrollment.

Exclusion Criteria:

  • Patient is currently receiving treatment with an immunomodulatory agent or has a history of such treatment, including targeted biological therapies (e.g., etanercept, omalizumab) within the 3 months before Baseline; corticosteroids within 4 weeks before Baseline; or non-steroidal anti-inflammatory agents (NSAIDs) within 2 weeks before Baseline.
  • Patient has a severe retinopathy.
  • Patient has a history of obstructive or restrictive lung disease (including interstitial lung disease, pulmonary fibrosis, or asthma), or evidence for interstitial lung disease on Screening chest radiograph.
  • Patient has evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, dermatological, or gastrointestinal disease, or has a condition that requires immediate surgical intervention or other treatment or may not allow safe participation.
  • Patient has used any investigational product or device (other than a mobility assistance device) within 30 days before Baseline.
  • If female and of childbearing potential (premenopausal and not surgically sterile), patient has a positive pregnancy test at Screening or is unwilling to use contraception from the time of Screening through the 1-month Follow-up visit. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device.
  • If male, patient is unwilling to use a condom plus spermicide during sexual intercourse from the time of Screening through the 1 month Follow-up visit.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02531217

Locations
United States, Ohio
aTyr Pharma Investigative Site
Columbus, Ohio, United States, 43210
Italy
aTyr Pharma Investigative Site
Rome, Italy, 00168
Netherlands
aTyr Pharma Investigative Site
Nijmegen, Netherlands
Sponsors and Collaborators
aTyr Pharma, Inc.
Investigators
Study Director: aTyr Pharma Inc. aTyr Pharma Inc. Sponsor GmbH
  More Information

Responsible Party: aTyr Pharma, Inc.
ClinicalTrials.gov Identifier: NCT02531217     History of Changes
Other Study ID Numbers: ATYR1940-C-005
2015-001912-36 ( EudraCT Number )
Study First Received: June 25, 2015
Last Updated: May 15, 2017

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Facioscapulohumeral
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 23, 2017