- Conditions
- Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy, Quadriceps Sparing Myopathy
- Interventions
- Aceneuramic Acid Extended-Release Tablets
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 143 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Orange, California • St Louis, Missouri + 1 more
Search Results
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Showing 1–7
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- Conditions
- GNE Myopathy, Hereditary Inclusion Body Myopathy
- Interventions
- Sialic Acid Extended Release (SA-ER), Placebo
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 3
- States / cities
- Los Angeles, California • St Louis, Missouri • New York, New York
- Conditions
- GNE Myopathy
- Interventions
- UX016, Placebo
- Drug · Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 2
- States / cities
- Orange, California • Iselin, New Jersey
- Conditions
- Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy, Quadriceps Sparing Myopathy, Inclusion Body Myopathy 2
- Interventions
- Aceneuramic Acid Extended-Release
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 3
- States / cities
- Orange, California • St Louis, Missouri • New York, New York
- Conditions
- Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy
- Interventions
- aceneuramic acid extended-release (Ace-ER), Placebo
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 89 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 4
- States / cities
- Irvine, California • St Louis, Missouri • New York, New York
- Conditions
- GNE Myopathy
- Interventions
- ManNAc, Placebo
- Drug · Other
- Lead sponsor
- Leadiant Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Iowa City, Iowa • Kansas City, Kansas + 7 more
- Conditions
- Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease, Quadriceps Sparing Myopathy (QSM), Distal Myopathy With Rimmed Vacuoles (DMRV)
- Interventions
- Not listed
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 319 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Irvine, California