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Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
Recruiting
Trial ID: NCT02603562,25513,26554
Purpose
The purpose of this study is to assess the safety and biological activity of ATYR1940 in
participants with early onset FSHD.
Official Title
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
Eligibility
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Intervention(s):
biological: ATYR1940
Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Jennie Perez
650-497-3080