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NCT06887777
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
- Locations
-
- Hôpital Universitaire Necker - Enfants Malades Paris, France
Recruiting
Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
NCT06340685
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
Recruiting
Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies
NCT03056794
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
- Locations
-
- University of Pittsburgh Pittsburgh, Pennsylvania, United States
Recruiting
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
NCT01793168
- Conditions
- 1p36 Deletion Syndrome, 3-Methylglutaconic Aciduria, Type V, 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome, +698 more
- Locations
-
- Sanford Health Sioux Falls, South Dakota, United States
- Online Patient Enrollment System Sydney, Australia
Completed
Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.
NCT03734263
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- Federico II University Naples, Italy
Active, Not Recruiting
Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
NCT02616484
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- Children's Hospital of Orange County Orange, California, United States
- Stanford University Stanford, California, United States
- Children's National Medical Center Washington D.C., District of Columbia, United States
- University of Florida Gainesville, Florida, United States