NCT02603562

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: aTyr Pharma, Inc. · Industry
• Sex: All
• Age: 16 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with early onset FSHD.

Detailed description

A Phase 1b/2 open-label, intraparticipant dose-escalation study aiming to evaluate the safety, tolerability, immunogenicity, biological and pharmacodynamic activity of intravenous ATYR1940, administered once weekly for 12 weeks, in early onset FSHD participants with signs or symptoms prior to 10 years of age.

Conditions

• Facioscapulohumeral Muscular Dystrophy (FSHD)

Interventions

Type	Name	Description
BIOLOGICAL	ATYR1940	Concentrate for solution for infusion

Timeline

• Start date: 2016-03-30
• Primary completion: 2016-12-12
• Completion: 2016-12-12
• First posted: 2015-11-13
• Last updated: 2023-10-19
• Results posted: 2023-10-19

Locations

7 sites across 3 countries: United States, France, Italy