- Trials with a EudraCT protocol (5)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
5 result(s) found for: Gangliosides.
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EudraCT Number: 2019-002827-14 | Sponsor Protocol Number: TreatSPG11 | Start Date*: 2021-01-19 | |||||||||||
Sponsor Name:IRCCS Fondazione Stella Maris | |||||||||||||
Full Title: Phase 2 pharmacological experimental study to test the safety of miglustat in subjects with hereditary spastic paraplegia type 11 | |||||||||||||
Medical condition: Spastic paraparesis type 11 (SPG11), caused by mutations in spatacsin protein, is characterized by the association of peripheral neuropathy, parkinsonism, ataxia, cognitive impairment, hypotrophy o... | |||||||||||||
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Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Completed) | |||||||||||||
Trial results: (No results available) |
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 |
EudraCT Number: 2006-004661-34 | Sponsor Protocol Number: 2006.426/19 | Start Date*: 2006-12-08 | |||||||||||
Sponsor Name:HOSPICES CIVILS DE LYON | |||||||||||||
Full Title: Evaluation d’un traitement par miglustat (Zavesca®) chez les patients atteints de mucopolysaccharidose de type III (maladie de Sanfilippo). Essai thérapeutique de phase IIb randomisé en aveugle con... | |||||||||||||
Medical condition: Mucopolysaccharidose de type III | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2014-001411-39 | Sponsor Protocol Number: ABT-002 | Start Date*: 2018-09-04 |
Sponsor Name:Abeona Therapeutics Inc | ||
Full Title: Phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB | ||
Medical condition: MPS IIIB is a devastating lysosomal storage disease, caused by a N-α-acetylglucosaminidase (NAGLU) gene defect. Infants with MPS IIIB appear normal at birth, but the disease is relentlessly progre... | ||
Disease: | ||
Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults | Gender: Male, Female | |
Trial protocol: ES (Prematurely Ended) DE (Prematurely Ended) FR (Prematurely Ended) GB (GB - no longer in EU/EEA) | ||
Trial results: (No results available) |
EudraCT Number: 2019-004909-27 | Sponsor Protocol Number: DNLI-E-0002 | Start Date*: 2020-07-22 | |||||||||||
Sponsor Name:Denali Therapeutics Inc. | |||||||||||||
Full Title: A PHASE 1/2, MULTICENTER, OPEN-LABEL STUDY TO DETERMINE THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROME | |||||||||||||
Medical condition: Hunter Syndrome (Mucopolysaccharidosis Type II [MPS II]) | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) NL (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
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