CompletedPhase 2NCT01517880

A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Studying GNE myopathy

Last synced from ClinicalTrials.gov May 8, 2026

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Key facts

Sponsor: Ultragenyx Pharmaceutical Inc
Principal Investigator: Alan Pestronk, MD, MD
Washington University School of Medicine
Intervention: Sialic Acid Extended Release (SA-ER)(drug)
Enrollment: 46 enrolled
Eligibility: 18-65 years · All sexes
Timeline: 2012 – 2013

Study locations (4)

UCLA Medical Center, Los Angeles, California, United States
Washington University School of Medicine, St Louis, Missouri, United States
New York University School of Medicine, New York, New York, United States
Hadassah University Hospital, Jerusalem, Israel

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT01517880 on ClinicalTrials.gov

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