- Trials with a EudraCT protocol (1)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
1 result(s) found for: eudract_number:2018-000504-42.
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EudraCT Number: 2018-000504-42 | Sponsor Protocol Number: ABT-003 | Start Date*: 2019-05-21 | |||||||||||
Sponsor Name:Abeona Therapeutics Inc | |||||||||||||
Full Title: A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients with Middle and Advanced Phases of MPS IIIA Disease | |||||||||||||
Medical condition: MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progress... | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Prematurely Ended) | |||||||||||||
Trial results: View results |
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