Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Recruiting · NCT02716246 · Registry updated 2026-09-22Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 3A | Recruiting | Phase 2 / Phase 3 | 2026-09-22 | NCT02716246 |
2 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Abeona Therapeutics, Inc | 5 |
| Ultragenyx Pharmaceutical Inc | 24 |
5 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Children's Hospital of Pittsburgh | Pittsburgh | United States | 16 |
| Hospital Clínico Universitario de Santiago | Santiago de Compostela | Spain | 8 |
| Nationwide Children's Hospital | Columbus | United States | 309 |
| Vall d'Hebron Barcelona Hospital Campus | Barcelona | Spain | 1 |
| Women's and Children's Hospital | North Adelaide | Australia | 13 |