- Trials with a EudraCT protocol (1)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
1 result(s) found for: eudract_number:2019-002979-34.
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EudraCT Number: 2019-002979-34 | Sponsor Protocol Number: LTFU-ABO-102 | Start Date*: 2020-05-18 | |||||||||||
Sponsor Name:Abeona Therapeutics Inc | |||||||||||||
Full Title: A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH) | |||||||||||||
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 (Ongoing) | |||||||||||||
Trial results: (No results available) |
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