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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)

Primary Purpose

Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
ATYR1940
Sponsored by
aTyr Pharma, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Facioscapulohumeral Muscular Dystrophy focused on measuring FSHD, LGMD

Eligibility Criteria

16 Years - 25 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • University of California, Irvine, ALS and Neuromuscular Center
  • Stanford University
  • University of Utah
  • Rigshospitalet, University of Copenhagen
  • Foundation IRCCS Neurological Institute Carlo

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ATYR1940

Arm Description

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.

Outcomes

Primary Outcome Measures

Incidences of Treatment-Emergent adverse events and serious adverse events
Incidences of adverse events including serious and severe adverse events overall and by intensity
Changes from Baseline in safety laboratory test results
Changes from Baseline in safety laboratory test results
Changes from Baseline in pulmonary evaluation of PFTs
Change from Baseline in pulmonary evaluation of PFTs
Change in Baseline in ECG findings
Change in Baseline in 12-lead electrocardiogram findings
Change from Baseline in vital signs
Change from Baseline in vital signs
Immunogenicity Outcome Measure - Incidence of ADA
Incidence of ADA titers
Immunogenicity Outcome Measure - Incidence of Jo-1 Ab
Incidence of Jo-1 Ab titers
Change from Baseline in Muscle strength using MMT
Change from Baseline in muscle strength using MMT
Changes from Baseline in pulmonary evaluation of pulse oximetry
Change from Baseline in pulmonary evaluation of pulse oximetry

Secondary Outcome Measures

Change from Baseline in CK levels
Change from Baseline in CK levels

Full Information

First Posted
June 30, 2016
Last Updated
April 20, 2017
Sponsor
aTyr Pharma, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02836418
Brief Title
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy
Acronym
FSHD
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
Study Type
Interventional

2. Study Status

Record Verification Date
April 2017
Overall Recruitment Status
Completed
Study Start Date
July 12, 2016 (Actual)
Primary Completion Date
April 18, 2017 (Actual)
Study Completion Date
April 18, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
aTyr Pharma, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Keywords
FSHD, LGMD

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ATYR1940
Arm Type
Experimental
Arm Description
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 Type
Drug
Intervention Name(s)
ATYR1940
Intervention Description
ATYR1940 at a dose no greater than 3.0 mg/kg administered via IV infusion over 90 minutes once weekly.
Primary Outcome Measure Information:
Title
Incidences of Treatment-Emergent adverse events and serious adverse events
Description
Incidences of adverse events including serious and severe adverse events overall and by intensity
Time Frame
12-weeks
Title
Changes from Baseline in safety laboratory test results
Description
Changes from Baseline in safety laboratory test results
Time Frame
12-weeks
Title
Changes from Baseline in pulmonary evaluation of PFTs
Description
Change from Baseline in pulmonary evaluation of PFTs
Time Frame
12-weeks
Title
Change in Baseline in ECG findings
Description
Change in Baseline in 12-lead electrocardiogram findings
Time Frame
12-weeks
Title
Change from Baseline in vital signs
Description
Change from Baseline in vital signs
Time Frame
12-weeks
Title
Immunogenicity Outcome Measure - Incidence of ADA
Description
Incidence of ADA titers
Time Frame
12-weeks
Title
Immunogenicity Outcome Measure - Incidence of Jo-1 Ab
Description
Incidence of Jo-1 Ab titers
Time Frame
12-weeks
Title
Change from Baseline in Muscle strength using MMT
Description
Change from Baseline in muscle strength using MMT
Time Frame
12-weeks
Title
Changes from Baseline in pulmonary evaluation of pulse oximetry
Description
Change from Baseline in pulmonary evaluation of pulse oximetry
Time Frame
12-weeks
Secondary Outcome Measure Information:
Title
Change from Baseline in CK levels
Description
Change from Baseline in CK levels
Time Frame
12-weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Enrolled in and completed the treatment period in the parent study. Demonstrated, in the Sponsor's and Investigator's opinions, acceptable tolerability of ATYR1940. 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. Is, in the opinion of the Investigator and Sponsor, a suitable candidate for continued ATYR1940 treatment. 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: 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). Planned to receive any vaccination during study participation. 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. 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. 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. 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gennyne Walker
Organizational Affiliation
aTyr Pharma, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of California, Irvine, ALS and Neuromuscular Center
City
Irvine
State/Province
California
ZIP/Postal Code
92697
Country
United States
Facility Name
Stanford University
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Rigshospitalet, University of Copenhagen
City
Copenhagen
Country
Denmark
Facility Name
Foundation IRCCS Neurological Institute Carlo
City
Milan
ZIP/Postal Code
20133
Country
Italy

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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