Trials / Terminated
TerminatedNCT02731690
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
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
- Status
- Terminated
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 42 (actual)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
The primary objective of this Phase 2 study is to evaluate the safety of open-label 6 g/day Ace-ER in GNEM participants with severe ambulatory impairment.
Conditions
- Hereditary Inclusion Body Myopathy
- Distal Myopathy With Rimmed Vacuoles
- Distal Myopathy, Nonaka Type
- GNE Myopathy
- Quadriceps Sparing Myopathy
- Inclusion Body Myopathy 2
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Aceneuramic Acid Extended-Release | oral tablets |
Timeline
- Start date
- 2016-04-29
- Primary completion
- 2018-01-10
- Completion
- 2018-01-10
- First posted
- 2016-04-07
- Last updated
- 2019-02-19
- Results posted
- 2019-02-19
Locations
5 sites across 3 countries: United States, Bulgaria, Canada
Source: ClinicalTrials.gov record NCT02731690. Inclusion in this directory is not an endorsement.