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Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
aTyr Pharma, Inc.
ClinicalTrials.gov Identifier:
NCT02836418
First received: June 30, 2016
Last updated: April 20, 2017
Last verified: April 2017
  Purpose
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).

Condition Intervention Phase
Facioscapulohumeral Muscular Dystrophy
Limb Girdle Muscular Dystrophy
Drug: ATYR1940
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
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 Dystrophy

Resource links provided by NLM:


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

Primary Outcome Measures:
  • 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 Frame: 12-weeks ]
    Change from Baseline in pulmonary evaluation of pulse oximetry


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


Enrollment: 8
Actual Study Start Date: July 12, 2016
Study Completion Date: April 18, 2017
Primary Completion Date: April 18, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ATYR1940
All patients will receive ATYR1940 at the highest tolerated dose received in the parent study for 12-weeks. After 12 weeks, if the patient is demonstrating good tolerability, theATYR1940 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.
Drug: ATYR1940
ATYR1940 at a dose no greater than 3.0 mg/kg administered via IV infusion over 90 minutes once weekly.

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   16 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Enrolled in and completed the treatment period in the parent study.
  2. Demonstrated, in the Sponsor's and Investigator's opinions, acceptable tolerability of ATYR1940.
  3. In the Investigator's opinion, patient has shown acceptable compliance with ATYR1940 and the study procedures in the parent study and is willing and able to comply with all procedures in the current study.
  4. Is, in the opinion of the Investigator and Sponsor, a suitable candidate for continued ATYR1940 treatment.
  5. Provide written informed consent or assent after the nature of the study has been explained and prior to the performance of any research-related procedures.

Exclusion Criteria:

  1. Is expected to require treatment with curcumin or systemic albuterol (intermittent inhaled albuterol is permissible) during study participation; or use of a product that putatively enhances muscle growth (e.g., insulin-like growth factor, growth hormone) or activity (e.g., Coenzyme Q, Coenzyme A, creatine, L-carnitine) on a chronic basis; or statin treatment initiation or significant adjustment to statin regimen (stable, chronic statin use is permissible).
  2. Planned to receive any vaccination during study participation.
  3. Abnormal baseline findings, medical condition(s), or laboratory findings that, in the Investigator's opinion, might jeopardize the patient's safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.
  4. 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.
  5. If female and of childbearing potential (premenopausal and not surgically sterile), has a positive pregnancy test at entry or is unwilling to use contraception from the time of entry through the 3-month Follow-up visit. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device.
  6. If male, is unwilling to use a condom plus spermicide during sexual intercourse from the time of entry 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: NCT02836418

Locations
United States, California
University of California, Irvine, ALS and Neuromuscular Center
Irvine, California, United States, 92697
Stanford University
Stanford, California, United States, 94305
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84132
Denmark
Rigshospitalet, University of Copenhagen
Copenhagen, Denmark
Italy
Foundation IRCCS Neurological Institute Carlo
Milan, Italy, 20133
Sponsors and Collaborators
aTyr Pharma, Inc.
Investigators
Study Director: Gennyne Walker aTyr Pharma, Inc.
  More Information

Responsible Party: aTyr Pharma, Inc.
ClinicalTrials.gov Identifier: NCT02836418     History of Changes
Other Study ID Numbers: ATYR1940-C-006
Study First Received: June 30, 2016
Last Updated: April 20, 2017
Individual Participant Data  
Plan to Share IPD: No

Keywords provided by aTyr Pharma, Inc.:
FSHD
LGMD

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

ClinicalTrials.gov processed this record on April 28, 2017