Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
Trial ID or NCT#
The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with early onset FSHD.
An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy
- - Established, genetically confirmed diagnosis of FSHD. - Onset of FSHD signs or symptoms prior to 10 years of age, as documented in the participant's medical record or based on participant or family report. - Provide written informed consent or assent - In the Investigator's opinion, participant is willing and able to complete all study procedures and comply with the weekly study visit schedule.
- - 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 or activity 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 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 participant's ability to complete the study protocol. - Muscle biopsy within 30 days before baseline.
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