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Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular Dystrophy (FSHD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02603562
Recruitment Status : Completed
First Posted : November 13, 2015
Last Update Posted : May 17, 2017
Sponsor:
Information provided by (Responsible Party):
aTyr Pharma, Inc.

Tracking Information
First Submitted Date  ICMJE November 5, 2015
First Posted Date  ICMJE November 13, 2015
Last Update Posted Date May 17, 2017
Actual Study Start Date  ICMJE March 30, 2016
Actual Primary Completion Date December 13, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 10, 2015)
  • 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: Changes from Baseline after 12 weeks ]
    Changes from Baseline in safety laboratory test results
  • Changes from Baseline in pulmonary evaluation [ Time Frame: Changes from Baseline after 12 week ]
    Change from Baseline in pulmonary evaluation
  • Changes from Baseline in visual assessment [ Time Frame: Changes from Baseline after 12 weeks ]
    Changes in Baseline in visual acuity
  • Changes from Baseline in hearing [ Time Frame: Changes from Baseline after 12 weeks ]
    Safety Primary Outcome Measure - Changes from Baseline in hearing based on audiometry
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 10, 2015)
  • Immunogenicity Outcome Measure - Incidence and level of ADA [ Time Frame: 12 weeks ]
    Incidence and level of ADA titers
  • Immunogenicity Outcome Measure - Incidence and level of Jo-1 Ab [ Time Frame: 12 weeks ]
    Incidence and level of Jo-1 Ab titers
  • Immunogenicity Outcome Measure - Incidence of infusion reactions [ Time Frame: 12 weeks ]
    Incidence of infusion reactions
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: November 10, 2015)
  • Pharmacodynamic Additional Outcome Measure - Changes in FSHD-related inflammatory immune state [ Time Frame: 12 weeks ]
    Effects assessed by changes in muscular dystrophy-related inflammatory immune state in peripheral blood
  • Pharmacodynamic Additional Outcome Measure - Changes from baseline clinical parameters [ Time Frame: 12 weeks ]
    Changes from baseline in muscle strength based on manual muscle strength
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular Dystrophy
Official Title  ICMJE An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy
Brief Summary The purpose of this study is to assess the safety and biological activity of ATYR1940 in patients with early onset facioscapulohumeral muscular dystrophy (FSHD).
Detailed Description A Phase 1b/2 open-label, intrapatient dose escalation study aiming to evaluate the safety, tolerability, immunogenicity, biological and pharmacodynamic activity of intravenous ATYR1940, administered once weekly for 12 weeks, in early onset FSHD patients with signs or symptoms prior to 10 years of age. In Stage 1, up to 8 patients between the ages of 16 and 25 years will be enrolled. Stage 2 of enrollment will include patients with early onset FSHD between the ages of 12 and 15 years.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Facioscapulohumeral Muscular Dystrophy (FSHD)
Intervention  ICMJE
  • Biological: ATYR1940
    ATYR1940 will be administered as an IV infusion at doses of 0.3, 1.0, and 3.0 mg/kg using intrapatient dose escalation. The dose level in this study will not exceed 3.0 mg/kg
  • Biological: Placebo
    An initial IV infusion of placebo will be supplied as normal saline, and administered over a 30-minute period at Week 1.
Study Arms  ICMJE
  • Experimental: ATYR1940
    Intrapatient dose escalation ATYR1940: ATYR1940 will be administered as an IV infusion at doses of 0.3, 1.0, and 3.0 mg/kg for up to 12 Weeks. The dose level in this study will not exceed 3.0 mg/kg.
    Intervention: Biological: ATYR1940
  • Placebo Comparator: Placebo
    An initial IV infusion of placebo will be supplied as normal saline, and administered over a 30-minute period at Week 1.
    Intervention: Biological: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 19, 2016)
8
Original Estimated Enrollment  ICMJE
 (submitted: November 10, 2015)
16
Actual Study Completion Date  ICMJE February 14, 2017
Actual Primary Completion Date December 13, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Established, genetically confirmed diagnosis of FSHD.
  • Onset of FSHD signs or symptoms prior to 10 years of age, as documented in the patient's medical record or based on patient or family report.
  • Provide written informed consent or assent
  • In the Investigator's opinion, patient is willing and able to complete all study procedures and comply with the weekly study visit schedule.

Exclusion Criteria:

  • Currently receiving treatment with an immunomodulatory agent including targeted biological therapies within the 3 months before baseline; corticosteroids within 3 months before baseline; or high-dose non-steroidal anti-inflammatory agents within 2 weeks before baseline.
  • Currently receiving curcumin or albuterol; use of a product that putatively enhances muscle growth or activity on a chronic basis within 4 weeks before baseline; statin treatment initiation or significant adjustment to statin regimen within 3 months before baseline (stable, chronic statin use is permissible).
  • Use of an investigational product or device within 30 days before baseline.
  • Evidence of an alternative diagnosis other than FSHD or a coexisting myopathy or dystrophy, based on prior muscle biopsy or other available investigations.
  • History of severe restrictive or obstructive lung disease, or evidence for interstitial lung disease on screening chest radiograph.
  • History of anti-synthetase syndrome, prior Jo-1 Ab-positivity, or a positive or equivocally positive Jo-1 Ab test result during screening.
  • Chronic infection, such as hepatitis B, hepatitis C, or human immunodeficiency virus or a history of tuberculosis.
  • Vaccination within 8 weeks before baseline or vaccination is planned during study participation.
  • Symptomatic cardiomyopathy or severe cardiac arrhythmia, that may, in the Investigator's opinion, limit the patient's ability to complete the study protocol.
  • Muscle biopsy within 30 days before baseline.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years to 25 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Italy,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02603562
Other Study ID Numbers  ICMJE ATYR1940-C-003
2014-003346-27 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party aTyr Pharma, Inc.
Study Sponsor  ICMJE aTyr Pharma, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Kelly Blackburn aTyr Pharma
PRS Account aTyr Pharma, Inc.
Verification Date May 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP