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Pyruvate Metabolism, Inborn Errors - 12 Studies Found
Estado | Estudiar |
Active, not recruiting |
Nombre del estudio: A Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency Condición: Pyruvate Kinase Deficiency Fecha: 2015-06-10 Intervenciones: Drug: AG-348 A range of doses of AG-348 will be tested based on the assessment of safety, tolerability a |
Active, not recruiting |
Nombre del estudio: Pyruvate Kinase Deficiency Natural History Study Condición:
Fecha: 2014-01-28 |
Recruiting |
Nombre del estudio: The Leigh Syndrome Registry Condición:
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Recruiting |
Nombre del estudio: Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies Condición: Pyruvate Dehydrogenase Complex Deficiency Disease Fecha: 2017-01-27 Intervenciones: Other: No intervention This is an observational study. The investigators will collect data about exposur |
Not yet recruiting |
Nombre del estudio: Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency: Condición: Pyruvate Dehydrogenase Complex Deficiency Fecha: 2015-11-17 Intervenciones:
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Completed |
Nombre del estudio: A Dose-escalating Clinical Trial With KH176 Condición:
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Enrolling by invitation |
Nombre del estudio: Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome Condición: Leigh Syndrome Fecha: 2014-08-21 Intervenciones: Drug: EPI-743 EPI-743 is a quinone oxidation product of alpha tocotrienol |
Completed |
Nombre del estudio: Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease Condición: Inherited Mitochondrial Disease, Including Leigh Syndrome Fecha: 2013-12-17 Intervenciones: Drug: Cysteamine Bitartrate Cysteamine Bitartrate Delayed-release Capsules |
Enrolling by invitation |
Nombre del estudio: Tissue Sample Study for Mitochondrial Disorders Condición:
Fecha: 2012-08-31 |
Recruiting |
Nombre del estudio: Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Condición:
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