- Trials with a EudraCT protocol (4)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
4 result(s) found for: Neuronal Ceroid Lipofuscinosis.
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EudraCT Number: 2021-000706-21 | Sponsor Protocol Number: AT-GTX-501-02 | Start Date*: 2021-12-10 | |||||||||||
Sponsor Name:Amicus Therapeutics, Inc. | |||||||||||||
Full Title: LONG-TERM FOLLOW-UP OF AT-GTX-501 SCAAV9 GENE TRANSFER IN SUBJECTS WITH CLN6 BATTEN DISEASE | |||||||||||||
Medical condition: Patients with variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease), a subset of patients with CLN6 Batten disease. | |||||||||||||
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Population Age: Children, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2012-005430-11 | Sponsor Protocol Number: 190-201 | Start Date*: 2013-08-15 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc. | |||||||||||||
Full Title: A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Late-Infantile Neuronal Ceroid Lipo... | |||||||||||||
Medical condition: Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2). | |||||||||||||
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Population Age: Children, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) DE (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2015-000891-85 | Sponsor Protocol Number: 190-203 | Start Date*: 2017-09-05 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc | |||||||||||||
Full Title: A Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 years of age with CLN2 Disease | |||||||||||||
Medical condition: Neuronal Ceroid Lipofuscinosis type 2 (CLN2) disease | |||||||||||||
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Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2014-003480-37 | Sponsor Protocol Number: 190-202 | Start Date*: 2015-03-10 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc. | |||||||||||||
Full Title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease | |||||||||||||
Medical condition: Late-Infantile Neuronal Ceroid Lipofuscinosis type 2 (CLN2). | |||||||||||||
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Population Age: Children, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
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