Rebisufligene etisparvovec
- Generic Name
- Rebisufligene etisparvovec
- Brand Names
- -
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1966978-67-5
- Unique Ingredient Identifier
- V28L3Q0LVZ
- Associated Conditions
- -
- Associated Therapies
- -
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Phase 2
Active, not recruiting
- Conditions
- Mucopolysaccharidosis IIIMPS IIIASanfilippo SyndromeSanfilippo A
- Interventions
- Biological: UX111Drug: Prophylactic Immunomodulatory (IM) TherapyDrug: Optimized Prophylactic IM TherapyDrug: Adjuvant IM Therapy
- First Posted Date
- 2016-03-23
- Last Posted Date
- 2025-01-06
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 36
- Registration Number
- NCT02716246
- Locations
- 🇪🇸
Vall d'Hebron Barcelona Hospital Campus, Barcelona, Spain
🇦🇺Women's and Children's Hospital, North Adelaide, South Australia, Australia
🇺🇸Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
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