NCT02377921View on ClinicalTrials.gov

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

PHASE3CompletedINTERVENTIONAL
Enrollment

89

Participants

Timeline

Start Date

May 20, 2015

Primary Completion Date

June 9, 2017

Study Completion Date

June 9, 2017

Conditions
Hereditary Inclusion Body MyopathyDistal Myopathy With Rimmed VacuolesDistal Myopathy, Nonaka TypeGNE Myopathy
Interventions
DRUG

aceneuramic acid extended-release (Ace-ER)

tablets for oral use

DRUG

Placebo

tablets for oral use

Trial Locations (13)

10016

New York University School of Medicine, New York

10029

Icahn School of Medicine at Mount Sinai, New York

63110

Washington University School of Medicine, St Louis

92697

University of California, Irvine, Irvine

Unknown

"UMHAT Alexandrovska", Sofia

CHU La Réunion - site GHSR, Saint-Pierre

Institut de Myologie GH Pitié-Salpêtrière, Paris

Hadassah-Hebrew University Medical Center, Jerusalem

University of Messina, Messina

University of Milan, Milan

Università Cattolica, Rome

L8N3Z5

McMaster University, Hamilton

NE1 4LP

The Newcastle upon Tyne Hospitals, Newcastle upon Tyne

All Listed Sponsors
lead

Ultragenyx Pharmaceutical Inc

INDUSTRY

NCT02377921 - Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) | Biotech Hunter | Biotech Hunter