An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)
- Conditions
- Facioscapulohumeral muscular dystrophygenetic myopathy10028396
- Registration Number
- NL-OMON44009
- Lead Sponsor
- aTyr Pharma, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 8
1. Enrolled in >=Cohort 2 and completed the double-blind treatment period in the parent study.;2. Demonstrated, in the Investigator*s opinion, acceptable tolerability of study drug.;3. In the Investigator*s opinion, patient has shown acceptable compliance with study drug 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 study drug treatment.;5. Provided written informed consent after the nature of the study has been explained and prior to the performance of any research-related procedures.
1. At any time during participation in the parent study, met a study drug discontinuation criterion, including, but not limited to:;a. Jo-1 Ab levels >=1.5 U/mL.;b. Clinical evidence of a generalized infusion-related reaction (IRR).;c. Clinical evidence of a National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) (version 4.03) >=1.5 U/mL;d. Pregnancy.;e. Progression of disease that, in the opinion of the Investigator, precluded further participation in the study.;f. Withdrawal of consent.;g. Other findings that, at the discretion of the Investigator and/or Sponsor, indicated that study drug administration should be discontinued.;2. 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).;3. Planned to receive any vaccination during study participation.;4. 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.;5. 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.;6. 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 1-month Follow-up visit. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device. ;7. 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Safety and tolerability will be evaluated by the following in all patients:<br /><br>1. Change from baseline in physical examination, including neurological<br /><br>examination.<br /><br>2. Incidence of AEs, including serious and severe AEs.<br /><br>3. Change from baseline in safety laboratory test results.<br /><br>4. Change from baseline in electrocardiogram (ECG) findings.<br /><br>5. Change from baseline in vital sign measurements and pulmonary evaluations<br /><br>(pulmonary function tests and pulse oximetry).<br /><br>6. Anti-drug antibody (ADA) titers and Jo-1 antibody levels.<br /><br>7. Exploratory characterization of immune response to ATYR1940.</p><br>
- Secondary Outcome Measures
Name Time Method