Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
- Conditions
- Interventions
- First Posted Date
- 2013-11-25
- Last Posted Date
- 2020-06-19
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 36
- Registration Number
- NCT01993186
- Locations
- 🇦🇺
Melbourne Brain Centre, Heidelberg, Victoria, Australia
🇺🇸Children's Hospital Colorado - University of Colorado, Denver, School of Medicine, Aurora, Colorado, United States
🇺🇸Neurology & Epilepsy Research Center, Orlando, Florida, United States
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
- Conditions
- Interventions
- First Posted Date
- 2013-06-25
- Last Posted Date
- 2021-02-11
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 29
- Registration Number
- NCT01886378
- Locations
- 🇺🇸
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸University of Southern Florida, Tampa, Florida, United States
🇺🇸Boston Children's Hospital, Boston, Massachusetts, United States
An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
- First Posted Date
- 2013-05-17
- Last Posted Date
- 2019-01-04
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 3
- Registration Number
- NCT01856218
- Locations
- 🇬🇧
Manchester Academic Health Science Centre, Manchester, United Kingdom
An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy
- Conditions
- Interventions
- First Posted Date
- 2013-04-12
- Last Posted Date
- 2018-04-11
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 59
- Registration Number
- NCT01830972
- Locations
- 🇺🇸
UCLA Medical Center, Los Angeles, California, United States
🇺🇸NYU Medical Center, New York, New York, United States
🇮🇱Hadassah University Hospital, Jerusalem, Israel
Study of Safety and Tolerability of BPS804 in Patients With Late-stage Chronic Kidney Disease
- Conditions
- Interventions
- First Posted Date
- 2013-03-07
- Last Posted Date
- 2022-09-14
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Registration Number
- NCT01806610
GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
- First Posted Date
- 2013-02-06
- Last Posted Date
- 2018-04-27
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 319
- Registration Number
- NCT01784679
- Locations
- 🇧🇬
Bulgarian Neuromuscular Disease Association, Sofia, Bulgaria
🇫🇷Association Institut de Myologie, Paris, France
🇨🇦McMaster University, Hamilton, Ontario, Canada
A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
- Conditions
- Interventions
- First Posted Date
- 2012-01-25
- Last Posted Date
- 2016-06-16
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 46
- Registration Number
- NCT01517880
- Locations
- 🇺🇸
UCLA Medical Center, Los Angeles, California, United States
🇺🇸Washington University School of Medicine, St. Louis, Missouri, United States
🇺🇸New York University School of Medicine, New York, New York, United States
Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta
- First Posted Date
- 2011-08-16
- Last Posted Date
- 2021-05-11
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 10
- Registration Number
- NCT01417091
- Locations
- 🇩🇪
Novartis Investigative Site, Wuerzburg, Germany
Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)
- First Posted Date
- 2011-08-01
- Last Posted Date
- 2022-09-16
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 8
- Registration Number
- NCT01406977
- Locations
- 🇩🇪
Mereo BioPharma 3 Ltd Investigative Site, Wuerzburg, Germany
Safety and Efficacy of Multiple Dosing Regimens of BPS804 in Post Menopausal Women With Low Bone Mineral Density
- Conditions
- Interventions
- First Posted Date
- 2011-08-01
- Last Posted Date
- 2022-09-15
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 44
- Registration Number
- NCT01406548
- Locations
- 🇺🇸
Novartis Investigative Site, Duncansville, Pennsylvania, United States