X-linked Hypophosphatemia Disease Monitoring Program
- Conditions
- Interventions
- First Posted Date
- 2018-08-29
- Last Posted Date
- 2024-06-13
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 780
- Registration Number
- NCT03651505
- Locations
- ๐บ๐ธ
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
๐บ๐ธUniversity of California, Los Angeles, Los Angeles, California, United States
๐บ๐ธWashington University School of Medicine, Saint Louis, Missouri, United States
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
- Conditions
- First Posted Date
- 2018-08-17
- Last Posted Date
- 2024-10-26
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 11
- Registration Number
- NCT03636438
- Locations
- ๐บ๐ธ
Boston Children's Hospital, Boston, Massachusetts, United States
๐บ๐ธUniversity Hospital Cleveland Medical Center/Case Western Reserve University, Cleveland, Ohio, United States
๐ฌ๐งQueen Elizabeth Hospital, Department of Endocrinology, Birmingham, United Kingdom
Mucopolysaccharidosis VII Disease Monitoring Program
- Conditions
- Interventions
- First Posted Date
- 2018-07-30
- Last Posted Date
- 2024-11-20
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 50
- Registration Number
- NCT03604835
- Locations
- ๐บ๐ธ
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
๐ง๐ทHospital de Clรญnicas de Porto Alegre, Porto Alegre, Rio Grande Do Sul, Brazil
๐บ๐ธChildren's Hospital of Orange County, Orange, California, United States
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
- First Posted Date
- 2018-05-07
- Last Posted Date
- 2022-11-18
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 12
- Registration Number
- NCT03517085
- Locations
- ๐บ๐ธ
Michigan Medicine University of Michigan, Ann Arbor, Michigan, United States
๐บ๐ธUCONN Health, Farmington, Connecticut, United States
๐บ๐ธUT Health - McGovern Medical School, Houston, Texas, United States
An Exploratory Study of BPS804 Treatment in Adult Patients With Type I, III or IV Osteogenesis Imperfecta
- First Posted Date
- 2017-07-13
- Last Posted Date
- 2023-07-03
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Registration Number
- NCT03216486
- Locations
- ๐บ๐ธ
Mereo Investigator Site, Houston, Texas, United States
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
- First Posted Date
- 2017-04-18
- Last Posted Date
- 2023-07-05
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 112
- Registration Number
- NCT03118570
- Locations
- ๐ฌ๐ง
Mereo Investigator Site, London, United Kingdom
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
- Conditions
- Interventions
- First Posted Date
- 2016-12-13
- Last Posted Date
- 2023-01-26
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 16
- Registration Number
- NCT02991144
- Locations
- ๐จ๐ฆ
Alberta's Children's Hospital, Calgary, Alberta, Canada
๐บ๐ธIcahn School of Medicine at Mount Sinai, New York, New York, United States
๐บ๐ธThe Children's Hospital Colorado, Aurora, Colorado, United States
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
- Conditions
- First Posted Date
- 2016-11-23
- Last Posted Date
- 2022-01-06
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 6
- Registration Number
- NCT02971969
- Locations
- ๐ฌ๐ง
Haemophilia, Haemostasis & Thrombosis Centre, Basingstoke, Hampshire, United Kingdom
๐บ๐ธArkansas Children's Hospital, Little Rock, Arkansas, United States
๐ฌ๐งManchester Haemophilia Comprehensive Care Center, Manchester, United Kingdom
Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
- Conditions
- Interventions
- First Posted Date
- 2016-11-09
- Last Posted Date
- 2020-06-16
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 44
- Registration Number
- NCT02960217
- Locations
- ๐บ๐ธ
Colorado Children's Hospital, Aurora, Colorado, United States
๐บ๐ธNicklaus Children's Hospital, Miami, Florida, United States
๐บ๐ธCenter for Rare Neurological Diseases, Norcross, Georgia, United States
Observational Study of Males With Creatine Transporter Deficiency
- Conditions
- First Posted Date
- 2016-10-13
- Last Posted Date
- 2022-11-14
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 50
- Registration Number
- NCT02931682
- Locations
- ๐บ๐ธ
University of California San Diego, La Jolla, California, United States
๐บ๐ธRush University Medical Center, Chicago, Illinois, United States
๐บ๐ธNational Institutes of Health Clinical Center, Bethesda, Maryland, United States