Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
- Conditions
- First Posted Date
- 2020-08-28
- Last Posted Date
- 2022-04-29
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 16
- Registration Number
- NCT04531189
- Locations
- 🇺🇸
Northwestern University Feinberg School of Medicine, Chicago, Illinois, United States
🇺🇸Seattle Children's Hospital, Seattle, Washington, United States
🇺🇸Yale University School of Medicine, New Haven, Connecticut, United States
A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102
- Conditions
- Interventions
- First Posted Date
- 2020-04-24
- Last Posted Date
- 2024-08-02
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 33
- Registration Number
- NCT04360265
- Locations
- 🇦🇺
Women's and Children's Hospital, North Adelaide, South Australia, Australia
🇺🇸Nationwide Children's Hospital, Columbus, Ohio, United States
🇪🇸Hospital ClÃnico Universitario de Santiago, Santiago De Compostela, Spain
A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects
- First Posted Date
- 2020-02-13
- Last Posted Date
- 2020-03-30
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 120
- Registration Number
- NCT04269122
- Locations
- 🇺🇸
PPD Phase 1 Unit, Austin, Texas, United States
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
- First Posted Date
- 2020-02-06
- Last Posted Date
- 2024-11-28
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 74
- Registration Number
- NCT04259281
- Locations
- 🇺🇸
Rare Disease Research, Atlanta, Georgia, United States
🇺🇸Rush University Medical Center, Chicago, Illinois, United States
🇺🇸Boston Children's Hospital, Boston, Massachusetts, United States
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
- Conditions
- Interventions
- First Posted Date
- 2019-09-13
- Last Posted Date
- 2023-07-25
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 5
- Registration Number
- NCT04088734
- Locations
- 🇺🇸
Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
🇦🇺Adelaide Women's and Children's Hospital, North Adelaide, South Australia, Australia
🇪🇸Hospital ClÃnico Universitario de Santiago, Santiago De Compostela, Spain
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
- Conditions
- Interventions
- First Posted Date
- 2019-05-31
- Last Posted Date
- 2024-11-22
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 12
- Registration Number
- NCT03970278
- Locations
- 🇺🇸
University of Texas Health Science Center at Houston, Houston, Texas, United States
🇳🇱University Medical Center Groningen, Groningen, Netherlands
🇪🇸Complejo Hospitalario Universitario de Santiago, Santiago De Compostela, A Coruna, Spain
Expanded Access to Burosumab
- First Posted Date
- 2018-12-13
- Last Posted Date
- 2024-09-05
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Registration Number
- NCT03775187
Expanded Access to Mepsevii
- First Posted Date
- 2018-12-13
- Last Posted Date
- 2024-12-16
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Registration Number
- NCT03775174
Expanded Access to Triheptanoin
- First Posted Date
- 2018-12-12
- Last Posted Date
- 2024-09-05
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Registration Number
- NCT03773770
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
- Conditions
- Interventions
- First Posted Date
- 2018-12-07
- Last Posted Date
- 2020-08-28
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 20
- Registration Number
- NCT03768817
- Locations
- 🇺🇸
Kaiser Permanente, Los Angeles, California, United States
🇺🇸Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States