- Trials with a EudraCT protocol (3)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
3 result(s) found for: Primary carnitine deficiency.
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EudraCT Number: 2022-001539-10 | Sponsor Protocol Number: UX007-CL302 | Start Date*: 2022-12-27 | |||||||||||
Sponsor Name:Ultragenyx Pharmaceutical Inc. | |||||||||||||
Full Title: A Randomized, Double-blind, Multicenter Study to Determine the Effect of Triheptanoin Compared with Even-chain, Medium-chain Triglycerides (MCT) on Major Clinical Events (MCEs) in Pediatric Patient... | |||||||||||||
Medical condition: Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | |||||||||||||
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Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Trial now transitioned) PL (Trial now transitioned) CZ (Trial now transitioned) DE (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2014-001637-88 | Sponsor Protocol Number: 2014-650 | Start Date*: 2014-08-12 | |||||||||||
Sponsor Name:John Vissing | |||||||||||||
Full Title: A phase 2a study to explore treatment with Sodium Valproate in adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV) | |||||||||||||
Medical condition: McArdle disease (Glycogen storage disorder type V) | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: DK (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2012-002933-12 | Sponsor Protocol Number: 11/0090 | Start Date*: 2014-04-07 | |||||||||||
Sponsor Name:University College London, | |||||||||||||
Full Title: A Phase II pilot study to explore treatment with Sodium Valproate in Adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV) | |||||||||||||
Medical condition: McArdle disease (Glycogen storage disease type V, GSDV). The condition is an inherited disorder of skeletal muscle that causes exercise intolerance. The condition can give way to potential rhabdom... | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) | |||||||||||||
Trial results: View results |
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