1 no longer available. .
Follow Pyruvate Carboxylase Deficiency Disease: new trials by email Literature Review of Pyruvate Carboxylase Deficiency Disease trials Research Report on Pyruvate Carboxylase 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 |
|---|---|---|---|---|---|---|
| Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism triheptanoin - Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2) | No longer available | Jerry Vockley, MD, PhD | 2021-12-10 |
Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of (1) · Barth Syndrome (1) · Trifunctional Protein Deficiency With Myopathy And Neuropathy (1) · VLCAD deficiency (1)
Abnormalities, Multiple (1) · Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Carbohydrate Metabolism, Inborn Errors (1) · Cardiovascular Abnormalities (1) · Cardiovascular Diseases (1) · Central Nervous System Diseases (1) · Congenital Abnormalities (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Genetic Diseases, Inborn (1) · Genetic Diseases, X-Linked (1) · Heart Defects, Congenital (1) · Heart Diseases (1) · Lipid Metabolism Disorders (1)
triheptanoin (1)
United States (1)