A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies
- Conditions
- Facioscapulohumeral muscular dystrophygenetic myopathy10028396
- Registration Number
- NL-OMON42284
- Lead Sponsor
- aTyr Pharma, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 16
1. Patient is a male or female aged 18 to 65 years, inclusive.;2. Patient has an established, genetically-confirmed, diagnosis of FSHD with clinical findings meeting existing criteria.;3. Patient has provided written informed consent after the nature of the study has been explained and prior to the performance of any research-related procedures.;4. Cohorts >=2 only: Patient has imaging findings meeting defined criteria for muscle inflammation in at least 1 skeletal muscle (as per MRI Procedural Manual).
1. Patient is currently receiving treatment with an immunomodulatory agent or has a history of such treatment, including targeted biological therapies (e.g., etanercept, omalizumab) within the 3 months before Baseline; corticosteroids within 4 weeks before Baseline; or high-dose non-steroidal anti-inflammatory agents (NSAIDs) (either chronic or intermittent) within 2 weeks before Baseline.;2. Patient has evidence of an alternative diagnosis other than FSHD, based on prior muscle biopsy or genetic test findings.;3. Patient has a presumptive diagnosis of FSHD, based on clinical assessment, but does not yet have genetic confirmation of the diagnosis.;4. Patient has a history of obstructive or restrictive lung disease (including interstitial lung disease, pulmonary fibrosis, or asthma), or evidence for interstitial lung disease on Screening chest radiograph.;5. Patient has a history of anti-synthetase syndrome, prior Jo-1 antibody (Ab)-positivity, or has a positive or equivocally positive Jo 1 Ab test result during Screening.;6. Patient has symptomatic cardiomyopathy or severe cardiac arrhythmia that may, in the Investigator*s opinion, limit the patient*s ability to complete the study protocol.;7. Patient has 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.;8. Patient has used any investigational product or device (other than a mobility assistance device) within 30 days before Baseline.;9. Patient underwent muscle biopsy within 30 days before Baseline.;10. If female and of childbearing potential (premenopausal and not surgically sterile), patient has a positive pregnancy test at Screening or is unwilling to use contraception.
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:<br /><br>• Change from Baseline of physical examination.<br /><br>• Incidence of AEs.<br /><br>• Change from Baseline in safety laboratory test results.<br /><br>• Change from Baseline in ECG findings.<br /><br>• Change from Baseline in vital sign measurements and pulmonary evaluations.<br /><br>• Antibody test results.<br /><br>• Incidence of infusion reactions and infusion site examination findings.<br /><br><br /><br>The following PK parameters will be determined or calculated from the drug<br /><br>concentration time data as follows using WinNonlin:<br /><br>• Standard PK parameters (Cmax, tmax, t1/2, etc).</p><br>
- Secondary Outcome Measures
Name Time Method <p>The PD effects of ATYR1940 will be evaluated by the following:<br /><br>• Changes in FSHD-related inflammatory immune state in peripheral blood and<br /><br>muscle.<br /><br>• Changes in the following clinical parameters.</p><br>