A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Biological Activity of ATYR1940 in Adult Patients With Molecularly Defined Genetic Muscular Dystrophies
Abstract
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies
Conditions: Facioscapulohumeral Muscular Dystrophy (FSHD).
Primary outcome: Number of Participants With Treatment Emergent Adverse Events (TEAEs); Number of Participants With Positive Anti-Drug Antibodies (ADA); Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result; Number of Participants With a Clinically Significant Laboratory Abnormality; Number of Participants With a Physical Examination Abnormality; Number of Participants With a Vital Sign-Related Event Resulting in a TEAE; Number of Participants With a Pulmonary Function Event Resulting in a TEAE.
Enrollment: 20 participants.
How strong is the evidence?
Evidence Hub reads this as a randomized trial, done in people. Its evidence level is 3 of 5 (Moderate · trial / cohort / secondary analysis). Trials without a large randomized design, cohorts and secondary analyses give moderate evidence. Reported size: 20 participants or records.
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