Setrusumab in Pediatric Japanese Subjects with Osteogenesis Imperfecta
- First Posted Date
- 2024-10-10
- Last Posted Date
- 2024-11-18
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 5
- Registration Number
- NCT06636071
- Locations
- 🇯🇵
Osaka Metropolitan University Hospital, Osaka, Japan
🇯🇵Osaka University Hospital, Osaka, Japan
🇯🇵Keio University Hospital, Tokyo, Japan
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
- First Posted Date
- 2024-10-10
- Last Posted Date
- 2024-11-25
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 140
- Registration Number
- NCT06636383
- Locations
- 🇺🇸
Children's Hospital of Orange County, Orange, California, United States
🇺🇸Children's Hospital Colorado, Denver, Colorado, United States
🇺🇸University of Connecticut Health Center, Hartford, Connecticut, United States
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS
- First Posted Date
- 2024-09-27
- Last Posted Date
- 2024-12-20
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 120
- Registration Number
- NCT06617429
- Locations
- 🇺🇸
Rare Disease Research, Atlanta, Georgia, United States
🇺🇸Rush University, Chicago, Illinois, United States
🇪🇸Clinical Trial Site, Seville, Spain
Long-term Extension of GTX-102 in Angelman Syndrome
- First Posted Date
- 2024-05-16
- Last Posted Date
- 2024-11-15
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 75
- Registration Number
- NCT06415344
- Locations
- 🇺🇸
Rare Disease Research, LLC, Atlanta, Georgia, United States
🇦🇺Queensland Children's Hospital, South Brisbane, Queensland, Australia
🇨🇦Childrens Hospital London Health Sciences Centre, London, Ontario, Canada
A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD
- Conditions
- Interventions
- First Posted Date
- 2023-07-06
- Last Posted Date
- 2024-09-30
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 60
- Registration Number
- NCT05933200
- Locations
- 🇯🇵
Juntendo University Hospital, Bunkyo City, Tokyo, Japan
🇯🇵The Jikei University Hospital, Minato City, Tokyo, Japan
🇨🇿General University Hospital in Prague-GUH (Všeobecná fakultnà nemocnice v Praze- VFN), Prague, Czechia
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
- Conditions
- Interventions
- First Posted Date
- 2023-03-14
- Last Posted Date
- 2024-08-02
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 69
- Registration Number
- NCT05768854
- Locations
- 🇺🇸
Cook Children's Medical Center, Fort Worth, Texas, United States
🇺🇸Washington University School of Medicine, Saint Louis, Missouri, United States
🇺🇸Children's Hospital Colorado, Aurora, Colorado, United States
Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency
- Conditions
- Interventions
- First Posted Date
- 2022-04-25
- Last Posted Date
- 2024-11-20
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 50
- Registration Number
- NCT05345171
- Locations
- 🇵🇹
Centro Hospitalar Universitario de Sao Joao, Porto, Portugal
🇺🇸University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States
🇺🇸University of California, Los Angeles, California, United States
Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta
- First Posted Date
- 2022-04-05
- Last Posted Date
- 2023-06-29
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 2
- Registration Number
- NCT05312697
- Locations
- 🇺🇸
New Mexico Clinical Research & Osteoporosis Center, Albuquerque, New Mexico, United States
Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III
- Conditions
- First Posted Date
- 2022-01-19
- Last Posted Date
- 2023-09-28
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 14
- Registration Number
- NCT05196165
- Locations
- 🇺🇸
Baylor College of Medicine, Houston, Texas, United States
🇺🇸University of Texas, Houston, Texas, United States
🇺🇸Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
- Conditions
- Interventions
- First Posted Date
- 2021-12-01
- Last Posted Date
- 2024-12-05
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 52
- Registration Number
- NCT05139316
- Locations
- 🇺🇸
Children's Hospital of Orange County, Orange, California, United States
🇺🇸Children's Hospital Colorado, Aurora, Colorado, United States
🇺🇸University of Connecticut Health Center, Farmington, Connecticut, United States