Pyruvate Dehydrogenase Complex Deficiency Disease: Clinical Trials

1 completed, 1 recruiting. 2 Phase 2.

By phase and status

Phase	Trials	Status
Phase 2	2	Completed 1, 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

Trials (filtered) newest first · largest first

All 2 trials

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
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

Narrower conditions

Fatigue (1)

Broader conditions

Brain Diseases (2)  ·  Brain Diseases, Metabolic (2)  ·  Brain Diseases, Metabolic, Inborn (2)  ·  Carbohydrate Metabolism, Inborn Errors (2)  ·  Central Nervous System Diseases (2)  ·  Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2)  ·  Genetic Diseases, Inborn (2)  ·  Genetic Diseases, X-Linked (2)  ·  Heredodegenerative Disorders, Nervous System (2)  ·  Intellectual Disability (2)  ·  Metabolic Diseases (2)  ·  Metabolism, Inborn Errors (2)  ·  Mitochondrial Diseases (2)  ·  Nervous System Diseases (2)  ·  Neurobehavioral Manifestations (2)

Interventions

4-phenylbutyric acid (1)  ·  glycerol phenylbutyrate (1)

Sponsors

Assistance Publique - Hôpitaux de Paris (1)  ·  Fondazione Telethon (1)

Countries

France (1)  ·  Italy (1)