A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

NCT02731690 · clinicaltrials.gov ↗

PHASE2

Phase

TERMINATED

Status

42

Enrollment

INDUSTRY

Sponsor class

Conditions

Hereditary Inclusion Body Myopathy
Distal Myopathy With Rimmed Vacuoles
Distal Myopathy, Nonaka Type
GNE Myopathy
Quadriceps Sparing Myopathy
Inclusion Body Myopathy 2

Interventions

DRUG: Aceneuramic Acid Extended-Release

Sponsor

Ultragenyx Pharmaceutical Inc