Clinical Trials
60
Trial Phases
3 Phases
Drug Approvals
4
Clinical Trials
Distribution across different clinical trial phases (39 trials with phase data)• Click on a phase to view related trials
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
- First Posted Date
- 2024-10-10
- Last Posted Date
- 2025-05-13
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 6
- 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
- Conditions
- Glycogen Storage Disease Type Ia
- First Posted Date
- 2024-10-10
- Last Posted Date
- 2025-07-08
- 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 Angelman Syndrome (AS)
- Conditions
- Angelman Syndrome
- Interventions
- Procedure: Sham-LP
- First Posted Date
- 2024-09-27
- Last Posted Date
- 2025-08-06
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 129
- Registration Number
- NCT06617429
- Locations
- 🇺🇸
Cedars Sinai, Los Angeles, California, United States
🇺🇸UCSD, Rady Children's Hospital, San Diego, California, United States
🇺🇸UCSF, San Francisco, California, United States
Long-term Extension of GTX-102 in Angelman Syndrome
- First Posted Date
- 2024-05-16
- Last Posted Date
- 2025-07-15
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 75
- Registration Number
- NCT06415344
- Locations
- 🇺🇸
University of California, Los Angeles (UCLA), Los Angeles, California, United States
🇺🇸University of California, San Diego - Rady Children's Hospital, San Diego, California, United States
🇺🇸Rare Disease Research, LLC, Atlanta, Georgia, United States
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
- Conditions
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
- Interventions
- Dietary Supplement: MCT Oil
- First Posted Date
- 2023-07-06
- Last Posted Date
- 2025-06-12
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 60
- Registration Number
- NCT05933200
- Locations
- 🇨🇿
General University Hospital in Prague-GUH (Všeobecná fakultní nemocnice v Praze- VFN), Prague, Czechia
🇩🇪Universitätsklinikum Freiburg, Freiburg, Germany
🇯🇵Juntendo University Hospital, Bunkyo City, Tokyo, Japan
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News
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