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Pyruvate Metabolism, Inborn Errors - 12 Studies Found

Status Study
Active, not recruiting Study Name: A Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency
Condition: Pyruvate Kinase Deficiency
Date: 2015-06-10
Interventions: Drug: AG-348 A range of doses of AG-348 will be tested based on the assessment of safety, tolerability a
Active, not recruiting Study Name: Pyruvate Kinase Deficiency Natural History Study
Condition:
  • Pyruvate Kinase Deficiency
  • Congenital Non-Spherocytic Hemolytic Anemia

Date: 2014-01-28
Recruiting Study Name: The Leigh Syndrome Registry
Condition:
  • Leigh Syndrome
  • Leigh Disease
  • Leigh's Necrotizing Encephalo
    Date: 2017-04-28
Recruiting Study Name: Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies
Condition: Pyruvate Dehydrogenase Complex Deficiency Disease
Date: 2017-01-27
Interventions: Other: No intervention This is an observational study. The investigators will collect data about exposur
Not yet recruiting Study Name: Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
Condition: Pyruvate Dehydrogenase Complex Deficiency
Date: 2015-11-17
Interventions:
  • Drug: Dichloroacetate (DCA) Pa
Completed Study Name: A Dose-escalating Clinical Trial With KH176
Condition:
  • MELAS
  • LHON
  • Leigh Syndrome
  • Drug: KH176
  • Drug: placebo
Enrolling by invitation Study Name: Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
Condition: Leigh Syndrome
Date: 2014-08-21
Interventions: Drug: EPI-743 EPI-743 is a quinone oxidation product of alpha tocotrienol
Completed Study Name: Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
Condition: Inherited Mitochondrial Disease, Including Leigh Syndrome
Date: 2013-12-17
Interventions: Drug: Cysteamine Bitartrate Cysteamine Bitartrate Delayed-release Capsules
Enrolling by invitation Study Name: Tissue Sample Study for Mitochondrial Disorders
Condition:
  • Mitochondrial Disorders
  • Mitochondrial Disease
  • Melas
    • <
    Date: 2012-08-31
Recruiting Study Name: Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Condition:
  • Rare Disorders
  • Undiagnosed Disorders
  • Disorders of Unknown
    Date: 2013-02-13
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