Rebisufligene etisparvovec
- Generic Name
- Rebisufligene etisparvovec
- Drug Type
- Biotech
- CAS Number
- 1966978-67-5
- Unique Ingredient Identifier
- V28L3Q0LVZ
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Phase 1
Terminated
- Conditions
- MPS IIIASanfilippo AMucopolysaccharidosis IIISanfilippo Syndrome
- 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
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Phase 2
Recruiting
- Conditions
- Sanfilippo SyndromeSanfilippo AMPS IIIAMucopolysaccharidosis III
- Interventions
- Drug: Prophylactic Immunomodulatory (IM) TherapyDrug: Optimized Prophylactic IM TherapyDrug: Adjuvant IM Therapy
- First Posted Date
- 2016-03-23
- Last Posted Date
- 2025-04-15
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Target Recruit Count
- 36
- Registration Number
- NCT02716246
- Locations
- 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇺🇸Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
🇦🇺Women's and Children's Hospital, North Adelaide, South Australia, Australia
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