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)
- First Posted Date
- 2016-04-13
- Last Posted Date
- 2023-03-24
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 143
- Registration Number
- NCT02736188
- Locations
- ๐ฎ๐น
University of Messina, Messina, Italy
๐บ๐ธWashington University School of Medicine, St. Louis, Saint Louis, Missouri, United States
๐ฎ๐นUniversity of Milan, Milan, Italy
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
- First Posted Date
- 2016-04-07
- Last Posted Date
- 2019-02-19
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 42
- Registration Number
- NCT02731690
- Locations
- ๐บ๐ธ
NYU Langone Medical Center, New York, New York, United States
๐จ๐ฆMcMaster University, Hamilton, Ontario, Canada
๐บ๐ธUniversity of California, Irvine, Orange, California, United States
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
- Conditions
- Interventions
- First Posted Date
- 2016-03-23
- Last Posted Date
- 2024-07-16
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 28
- Registration Number
- NCT02716246
- Locations
- ๐ช๐ธ
Vall d'Hebron Barcelona Hospital Campus, Barcelona, Spain
๐ฆ๐บWomen's and Children's Hospital, North Adelaide, South Australia, Australia
๐บ๐ธChildren's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
- First Posted Date
- 2015-12-02
- Last Posted Date
- 2018-11-14
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 6
- Registration Number
- NCT02618915
- Locations
- ๐บ๐ธ
Orthopaedic Institute for Children, Los Angeles, California, United States
๐บ๐ธBoston Children's Hospital, Boston, Massachusetts, United States
๐บ๐ธVanderbilt Hemostasis-Thrombosis Clinic, Nashville, Tennessee, United States
Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
- First Posted Date
- 2015-11-09
- Last Posted Date
- 2020-06-11
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 15
- Registration Number
- NCT02599961
- Locations
- ๐บ๐ธ
Miami Children's Hospital, Miami, Florida, United States
๐บ๐ธSeattle Children's Hospital, Seattle, Washington, United States
๐ฉ๐ฐCopenhagen University Hospital, Copenhagen, Denmark
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
- Conditions
- Interventions
- First Posted Date
- 2015-05-01
- Last Posted Date
- 2020-07-30
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 12
- Registration Number
- NCT02432144
- Locations
- ๐บ๐ธ
Baylor College of Medicine, Houston, Texas, United States
๐ง๐ทHospital Infantil Candido Fontoura Sao Paulo, Sao Paulo, Brazil
๐บ๐ธChildren's Hospital of Orange County, Orange, California, United States
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
- Conditions
- Interventions
- First Posted Date
- 2015-04-16
- Last Posted Date
- 2019-10-30
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 8
- Registration Number
- NCT02418455
- Locations
- ๐บ๐ธ
New York University Langone Medical Center, New York, New York, United States
๐บ๐ธChildren's National Health System, Washington, District of Columbia, United States
๐ต๐นCentro Hospitalar do Porto, Porto, Portugal
Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
- First Posted Date
- 2015-03-04
- Last Posted Date
- 2019-06-27
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 89
- Registration Number
- NCT02377921
- Locations
- ๐ง๐ฌ
UMHAT "Alexandrovska", Sofia, Bulgaria
๐ฎ๐นUniversitร Cattolica, Rome, Italy
๐ฌ๐งThe Newcastle upon Tyne Hospitals, Newcastle Upon Tyne, Tyne And Wear, United Kingdom
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
- Conditions
- Interventions
- First Posted Date
- 2014-09-03
- Last Posted Date
- 2020-07-30
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 12
- Registration Number
- NCT02230566
- Locations
- ๐บ๐ธ
Children's Hospital Oakland, Oakland, California, United States
๐บ๐ธMiami Children's Hospital, Miami, Florida, United States
๐บ๐ธChildren's Hospital of Orange County, Orange, California, United States
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
- Conditions
- Interventions
- First Posted Date
- 2014-08-12
- Last Posted Date
- 2023-08-01
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 94
- Registration Number
- NCT02214160
- Locations
- ๐บ๐ธ
Children's National Health System, Washington, District of Columbia, United States
๐บ๐ธUniversity of South Florida, Tampa, Florida, United States
๐บ๐ธVanderbilt Medical Center, Nashville, Tennessee, United States