4 recruiting, 2 unknown, 1 active not recruiting, 1 available. 2 Phase 2, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Completed 1, Recruiting 1 |
| Phase 1 | 1 | Recruiting 1 |
| Phase 3 | 1 | Active not recruiting 1 |
Follow Pyruvate Dehydrogenase Complex Deficiency Disease: new trials by email Literature Review of Pyruvate Dehydrogenase Complex Deficiency Disease trials Research Report on Pyruvate Dehydrogenase Complex Deficiency Disease
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI) Glycerol Phenylbutyrate 1100 MG/ML [Ravicti] - Pyruvate Dehydrogenase Complex Deficiency Disease | Phase 2 | Recruiting | Assistance Publique - Hôpitaux de Paris | 15 | 2025-10-01 | 2026-03-31 |
| Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency Triheptanoin - Pyruvate Dehydrogenase Complex Deficiency | Phase 1 | Recruiting | Jirair Krikor Bedoyan | 6 | 2024-07-11 | 2026-02-17 |
| Natural History Study of Pyruvate Dehydrogenase Deficiency Pyruvate Dehydrogenase Complex Deficiency, Pyruvate Dehydrogenase E1 Alpha Deficiency | Unknown | Great Ormond Street Hospital for Children NHS Foundation Trust | 50 | 2020-11-01 | 2023-12-05 | |
| Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency: Dichloroacetate (DCA), Placebo, Genotype - Pyruvate Dehydrogenase Complex Deficiency | Phase 3 | Active not recruiting | Saol Therapeutics Inc | 34 | 2020-07-14 | 2025-05-04 |
| Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. sodium phenylbutyrate - Pyruvate Dehydrogenase Complex Deficiency | Phase 2 | Completed | Fondazione Telethon | 1 | 2018-10-01 | 2021-10-13 |
| Long-term Treatment With the Ketogenic Diet in Epilepsy Color duplex ultrasound of the carotid arteries - Epilepsy, GLUT1DS1 | Unknown | Oslo University Hospital | 30 | 2018-08-06 | 2022-10-21 | |
| Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies No intervention - Pyruvate Dehydrogenase Complex Deficiency Disease | Recruiting | University of Pittsburgh | 150 | 2015-09-01 | 2026-10-05 | |
| Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Rare Disorders, Undiagnosed Disorders | Recruiting | Sanford Health | 20000 | 2010-07-01 | 2026-10-28 | |
| Expanded Access Treatment Protocol With DCA for Patients With PDCD Dichloroacetate (DCA) - Pyruvate Dehydrogenase Complex Deficiency | Available | Saol Therapeutics Inc | 2025-05-08 |
3-Methylglutaconic Aciduria, Type V (1) · Achalasia Addisonianism Alacrimia syndrome (1) · Acrodysostosis (1) · Activated PI3K-delta Syndrome (1) · Addison Disease (1) · Alagille Syndrome (1) · Alstrom Syndrome (1) · Amnesia, Transient Global (1) · Aniridia (1) · Aniridia and Absent Patella (1) · Aniridia, Partial, with Unilateral Renal Agenesis and Psychomotor Retardation (1) · Ataxia (1) · Ataxia Neuropathy Spectrum (1) · Ataxia Telangiectasia (1)
Brain Diseases (9) · Brain Diseases, Metabolic (9) · Brain Diseases, Metabolic, Inborn (9) · Carbohydrate Metabolism, Inborn Errors (9) · Central Nervous System Diseases (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Genetic Diseases, X-Linked (9) · Heredodegenerative Disorders, Nervous System (9) · Intellectual Disability (9) · Metabolic Diseases (9) · Metabolism, Inborn Errors (9) · Nervous System Diseases (9) · Neurobehavioral Manifestations (9) · Neurologic Manifestations (9)
Dichloroacetic Acid (2) · 4-phenylbutyric acid (1) · Genotype (1) · glycerol phenylbutyrate (1) · triheptanoin (1)
Saol Therapeutics Inc (2) · Assistance Publique - Hôpitaux de Paris (1) · Fondazione Telethon (1) · Great Ormond Street Hospital for Children NHS Foundation Trust (1) · Jirair Krikor Bedoyan (1) · Oslo University Hospital (1) · Sanford Health (1) · University of Pittsburgh (1)
United States (4) · France (1) · Italy (1) · Norway (1) · United Kingdom (1)
Anette Ramm-Pettersen, MD PhD (1) · Jirair Bedoyan, MD, PhD (1) · Jirair K. Bedoyan, MD, PhD (1) · Kiki Diorgu, M.D. (1) · Richard Neibeger, MD (1) · Shamima Rahman, PhD (1)
U54NS078059 (1)