NCT01517880View on ClinicalTrials.gov

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

PHASE2CompletedINTERVENTIONAL
Enrollment

46

Participants

Timeline

Start Date

May 31, 2012

Primary Completion Date

November 30, 2013

Study Completion Date

November 30, 2013

Conditions
GNE MyopathyHereditary Inclusion Body Myopathy
Interventions
DRUG

Sialic Acid Extended Release (SA-ER)

SA-ER will be administered in doses of 3000mg per day or 6000mg per day

DRUG

Placebo

Subjects will be randomized to the placebo arm for the first 24 weeks of the study. Then, subjects in this arm will be re-randomized into either the 3,000 mg per day or 6,000 mg per day arm for the remaining 24 weeks of the study (total study duration 48 weeks).

Trial Locations (4)

10016

New York University School of Medicine, New York

63110

Washington University School of Medicine, St Louis

90095

UCLA Medical Center, Los Angeles

91120

Hadassah University Hospital, Jerusalem

All Listed Sponsors
lead

Ultragenyx Pharmaceutical Inc

INDUSTRY

NCT01517880 - 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 | Biotech Hunter | Biotech Hunter