Skip to content
Pharma Observatory a Scientist.com platform

Clinical trial

A Phase 2 Open-label Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment

NCT02731690 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT02731690
Email me this page as a PDF

Search trials, sites, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.

Get the NCT02731690 report as a PDF

The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 2 Open-label Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 2
Overall status
Terminated
Start date
April 29, 2016
Completion date
January 10, 2018
Enrollment
42 (actual)
Sites
5

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, inclusion body myopathy 2, quadriceps sparing myopathy