- Trials with a EudraCT protocol (3)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
		
	   		
	   		    
                    
                   	
                   	    3 result(s) found for: Cisterna.
                    
                
			
   			
		
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| EudraCT Number: 2020-001109-22 | Sponsor Protocol Number: PBGM01-001 | Start Date*: Information not available in EudraCT | 
| Sponsor Name:Passage Bio, Inc. | ||
| Full Title: A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered into the Cisterna Magna of Pediatric Su... | ||
| Medical condition: GM1 gangliosidosis (GM1) is an autosomal recessive disorder that results from mutations in the human galactosidase beta 1 gene (GLB1), which encodes beta-galactosidase (β-gal). | ||
| Disease: | ||
| Population Age: Infants and toddlers, Under 18 | Gender: Male, Female | |
| Trial protocol: GB (GB - no longer in EU/EEA) | ||
| Trial results: (No results available) | ||
| EudraCT Number: 2020-005229-95 | Sponsor Protocol Number: PBKR03-001 | Start Date*: 2021-08-23 | |||||||||||
| Sponsor Name:Passage Bio, Inc. | |||||||||||||
| Full Title: A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Dis... | |||||||||||||
| Medical condition: Krabbe disease (globoid cell leukodystrophy) is an autosomal recessive lysosomal storage disease (LSD) caused by mutations in the gene encoding the hydrolytic enzyme galactosylceramidase (galactoce... | |||||||||||||
| 
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| Population Age: Infants and toddlers, Under 18 | Gender: Male, Female | ||||||||||||
| Trial protocol: NL (Prematurely Ended) | |||||||||||||
| Trial results: (No results available) | |||||||||||||
| EudraCT Number: 2019-003159-12 | Sponsor Protocol Number: J4B-MC-OKAA | Start Date*: 2021-12-24 | |||||||||||
| Sponsor Name:Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company | |||||||||||||
| Full Title: A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of LY3884963 in Patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM) | |||||||||||||
| Medical condition: Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) | |||||||||||||
| 
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| Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
| Trial protocol: BE (Trial now transitioned) ES (Temporarily Halted) FR (Trial now transitioned) | |||||||||||||
| Trial results: (No results available) | |||||||||||||
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