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Clinical trial

Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

NCT02736188 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT02736188
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Registry record

As filed in ClinicalTrials.gov.

Title
Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 3
Overall status
Terminated
Start date
May 2, 2016
Completion date
January 10, 2018
Enrollment
143 (actual)
Sites
14

Conditions

As the registry lists them; a condition with its own page is linked.

distal myopathy with rimmed vacuoles, distal myopathy, nonaka type, gne myopathy, hereditary inclusion body myopathy, quadriceps sparing myopathy