NCT01784679View on ClinicalTrials.gov

GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

CompletedOBSERVATIONAL
Enrollment

319

Participants

Timeline

Start Date

April 5, 2013

Primary Completion Date

November 30, 2017

Study Completion Date

November 30, 2017

Conditions
Hereditary Inclusion Body MyopathyGNE MyopathyNonaka DiseaseQuadriceps Sparing Myopathy (QSM)Distal Myopathy With Rimmed Vacuoles (DMRV)
Trial Locations (5)

75013

Association Institut de Myologie, Paris

92697

University of California, Irvine, Irvine

Unknown

Bulgarian Neuromuscular Disease Association, Sofia

L8N3Z5

McMaster University, Hamilton

NE1 4LP

The Newcastle upon Tyne Hospitals, Newcastle upon Tyne

All Listed Sponsors
collaborator

Newcastle University

OTHER

lead

Ultragenyx Pharmaceutical Inc

INDUSTRY

NCT01784679 - GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM) | Biotech Hunter | Biotech Hunter