Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
4 studies found for:    "pyruvate carboxylase deficiency" OR "Pyruvate Metabolism, Inborn Errors"
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Rank Status Study
1 Available Compassionate Use of Triheptanoin (C7) for Fatty Acid Oxidation Disorders and Glycogen Storage Disease
Conditions: Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency;   Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2);   Mitochondrial Trifunctional Protein Deficiency;   Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency;   Glycogen Storage Disorders;   Pyruvate Carboxylase Deficiency, Type B;   Acyl-coA Dehydrogenase, Type 9
Intervention: Drug: triheptanoin
2 Recruiting Pyruvate Kinase Deficiency Natural History Study
Conditions: Pyruvate Kinase Deficiency;   Congenital Non-Spherocytic Hemolytic Anemia
Intervention:
3 Active, not recruiting Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
Condition: Leigh Syndrome
Interventions: Drug: Placebo;   Drug: EPI-743 15 mg/kg;   Drug: EPI-743 5 mg/kg
4 Enrolling by invitation Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
Condition: Leigh Syndrome
Intervention: Drug: EPI-743

Indicates status has not been verified in more than two years