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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)

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: NCT02836418
Recruitment Status : Completed
First Posted : July 19, 2016
Last Update Posted : April 24, 2017
Sponsor:
Information provided by (Responsible Party):
aTyr Pharma, Inc.

Tracking Information
First Submitted Date  ICMJE June 30, 2016
First Posted Date  ICMJE July 19, 2016
Last Update Posted Date April 24, 2017
Actual Study Start Date  ICMJE July 12, 2016
Actual Primary Completion Date April 18, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 14, 2016)
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 14, 2016)
Change from Baseline in CK levels [ Time Frame: 12-weeks ]
Change from Baseline in CK levels
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 Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy
Official Title  ICMJE 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
Brief Summary 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).
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Facioscapulohumeral Muscular Dystrophy
  • Limb Girdle Muscular Dystrophy
Intervention  ICMJE Drug: ATYR1940
ATYR1940 at a dose no greater than 3.0 mg/kg administered via IV infusion over 90 minutes once weekly.
Study Arms  ICMJE 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.
Intervention: Drug: ATYR1940
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: February 28, 2017)
8
Original Estimated Enrollment  ICMJE
 (submitted: July 14, 2016)
24
Actual Study Completion Date  ICMJE April 18, 2017
Actual Primary Completion Date April 18, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 16 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 Denmark,   Italy,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02836418
Other Study ID Numbers  ICMJE ATYR1940-C-006
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party aTyr Pharma, Inc.
Study Sponsor  ICMJE aTyr Pharma, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Gennyne Walker aTyr Pharma, Inc.
PRS Account aTyr Pharma, Inc.
Verification Date April 2017

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