A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Active, not recruiting · NCT04884815 · Registry updated 2026-09-25Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Wilson disease, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, inborn metal metabolism disorder, inherited neurodegenerative disorder, liver disorder, metabolic disease, movement disorder, nervous system disorder, neurodegenerative disease | Active, not recruiting | Phase 1 / Phase 2 | 2026-09-25 | NCT04884815 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Ultragenyx Pharmaceutical Inc | 24 |
16 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Centro Hospitalar Universitário de São João | Porto | Portugal | 3 |
| Centro Hospitalar Universitário Lisboa Norte | Lisbon | Portugal | 1 |
| Duke University Medical Center | Durham | United States | 505 |
| Hospital Universitario Vall d'Hebron - PPDS | Barcelona | Spain | 16 |
| Indiana University | Indianapolis | United States | 160 |
| Kings College NHS Foundation | London | United Kingdom | 1 |
| Massachusetts General Hospital | Boston | United States | 1015 |
| Northwestern University | Chicago | United States | 727 |
| Seattle Children's Hospital | Seattle | United States | 281 |
| Stanford University | Redwood City | United States | 25 |
| University Hospitals Cleveland Medical Center | Cleveland | United States | 234 |
| University of California Davis | Sacramento | United States | 1 |
| University of California Los Angeles | Los Angeles | United States | 87 |
| University of Michigan | Ann Arbor | United States | 538 |
| University of Utah | Salt Lake City | United States | 106 |
| Vanderbilt University Medical Center | Nashville | United States | 1 |