Trials / Terminated
TerminatedNCT02736188
Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
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)
- Status
- Terminated
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 143 (actual)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
The objective of this study is to evaluate the long-term safety and efficacy of Ace-ER treatment in subjects with GNEM.
Detailed description
This Phase 3b extension study will assess the long-term safety of Ace-ER in patients who participated in and completed study UX001-CL301 (NCT02377921), study UX001-CL202 (NCT01830972), and study UX001-CL203 (NCT02731690).
Conditions
- Hereditary Inclusion Body Myopathy
- Distal Myopathy With Rimmed Vacuoles
- Distal Myopathy, Nonaka Type
- GNE Myopathy
- Quadriceps Sparing Myopathy
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Aceneuramic Acid Extended-Release Tablets |
Timeline
- Start date
- 2016-05-02
- Primary completion
- 2018-01-10
- Completion
- 2018-01-10
- First posted
- 2016-04-13
- Last updated
- 2023-03-24
- Results posted
- 2019-02-19
Locations
14 sites across 7 countries: United States, Bulgaria, Canada, France, Israel, Italy, United Kingdom
Source: ClinicalTrials.gov record NCT02736188. Inclusion in this directory is not an endorsement.