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Trial record 16 of 29 for:    lgmd

The Safety and Biological Activity of ATYR1940 in Patients With Limb Girdle or Facioscapulohumeral Muscular Dystrophies (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: NCT02579239
Recruitment Status : Completed
First Posted : October 19, 2015
Last Update Posted : May 17, 2017
Sponsor:
Information provided by (Responsible Party):
aTyr Pharma, Inc.

Tracking Information
First Submitted Date  ICMJE September 28, 2015
First Posted Date  ICMJE October 19, 2015
Last Update Posted Date May 17, 2017
Actual Study Start Date  ICMJE November 5, 2015
Actual Primary Completion Date October 5, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 15, 2015)
  • Tolerability Primary Outcome Measure - incidence of treatment-emergent adverse events (AEs) and serious adverse events overal (SAEs) overall and by intensity [ Time Frame: 12 weeks ]
    Incidences of Adverse Events (AEs), including serious and severe AEs
  • Safety Primary Outcome Measure - change from Baseline in safety laboratory test results [ Time Frame: 12 weeks ]
    Change from Baseline in safety laboratory test results (clinical chemistry, hematology, and urinalysis)
  • Safety Primary Outcome Measure - change from Baseline in ECG [ Time Frame: 12 weeks ]
    Change from Baseline in 12-lead ECG findings
  • Safety Primary Outcome Measure - change from Baseline in vital signs measurements and pulmonary evaluations [ Time Frame: 12 week ]
    Change from Baseline in vital signs measurements (blood pressure, pulse, respiratory rate, and body temperature) and pulmonary evaluations (PFTs and pulse oximetry)
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02579239 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 15, 2015)
  • Pharmacodynamic Additional Outcome Measure - changes in muscular dystrophy-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 in serum- and/or plasma-based muscle biomarkers. [ Time Frame: 12 weeks ]
    Serum-based muscle biomarkers
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE The Safety and Biological Activity of ATYR1940 in Patients With Limb Girdle or Facioscapulohumeral Muscular Dystrophies
Official Title  ICMJE An Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophies
Brief Summary The purpose of this study is to assess the safety and biological activity of ATYR1940 in patients with Limb Girdle (LGMD2B) or Facioscapulohumeral Muscular Dystrophy (FSHD).
Detailed Description ATYR1940-C-004 is a Phase1b/2 open-label intrapatient study dose escalation study aiming to evaluate the safety, tolerability, immunogenicity, biological activity of intravenous ATYR1940, administered once weekly for 8 weeks, then twice a weekly for 4 weeks in adult patients with LGMD2B and FSHD. Approximately 8 LGMD2B and 8 FHSD patients will be enrolled.
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
  • Limb-Girdle Muscular Dystrophies
  • Facioscapulohumeral Muscular Dystrophy
Intervention  ICMJE
  • Biological: ATYR1940
    Intrapatient dose escalation of intravenous ATYR1940 administered twice weekly at doses of 0.3, 1.0, or 3.0 mg/kg for up to 12 weeks.
  • Biological: Placebo
    Patients will receive an initial infusion of placebo at Week 1, supplied as normal saline and administered via IV infusion over a 30-minute period.
Study Arms  ICMJE
  • Experimental: ATYR1940
    Intrapatient dose escalation of intravenous ATYR1940 administered twice weekly at doses of 0.3, 1.0, or 3.0 mg/kg for up to 12 weeks.
    Intervention: Biological: ATYR1940
  • Placebo Comparator: Placebo
    Patients will receive an initial infusion of placebo at Week 1, supplied as normal saline and administered via IV infusion over a 30-minute period.
    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)
18
Original Estimated Enrollment  ICMJE
 (submitted: October 15, 2015)
16
Actual Study Completion Date  ICMJE October 5, 2016
Actual Primary Completion Date October 5, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Provided informed consent
  • Investigator's opinion, patient is willing and able to complete all study procedures and comply with the study visit schedule.

Patients with LGMD2B:

  • Established, genetically confirmed diagnosis of LGMD2B.
  • Either the presence of a STIR-positive muscle on lower extremity skeletal muscle MRI, or, if no STIR-positive muscles, meets muscle biomarker criteria.

Patients with FSHD:

  • Established, genetically confirmed diagnosis of FSHD.
  • The presence of a STIR-positive muscle on lower extremity skeletal muscle MRI.

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 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 LGMD2B or 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 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Denmark,   France,   United States
Removed Location Countries Italy
 
Administrative Information
NCT Number  ICMJE NCT02579239
Other Study ID Numbers  ICMJE ATYR1940-C-004
2015-001910-88 ( EudraCT Number )
Has Data Monitoring Committee No
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