1 active not recruiting, 1 no longer available, 1 terminated. 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 1 | Terminated 1 |
Follow Mitochondrial Trifunctional Protein Deficiency: new trials by email Literature Review of Mitochondrial Trifunctional Protein Deficiency trials Research Report on Mitochondrial Trifunctional Protein Deficiency
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 |
|---|---|---|---|---|---|---|
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Vitamin E Treatment for Long-Chain 3-Hydroxyacyl Coenzyme A (CoA) Dehydrogenase (LCHAD) Associated Neuropathy Vitamin E supplement - Peripheral Neuropathy, Mitochondrial Trifunctional Protein Deficiency | Not applicable | Terminated | Oregon Health and Science University | 1 | 2010-07-01 | 2015-10-21 |
| 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 |
Trifunctional Protein Deficiency With Myopathy And Neuropathy (3) · Barth Syndrome (2) · VLCAD deficiency (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Acidemia, isovaleric (1) · Acrodermatitis enteropathica (1) · Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of (1) · Adenine phosphoribosyltransferase deficiency (1) · Adrenal hyperplasia, congenital, type 5 (1) · Adrenocorticotropic hormone deficiency (1) · Amaurosis congenita of Leber, type 2 (1) · Anemia, Sickle Cell (1)
Nervous System Diseases (3) · Abnormalities, Multiple (2) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Carbohydrate Metabolism, Inborn Errors (2) · Cardiovascular Abnormalities (2) · Cardiovascular Diseases (2) · Central Nervous System Diseases (2) · Congenital Abnormalities (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Genetic Diseases, X-Linked (2) · Heart Defects, Congenital (2) · Heart Diseases (2)
alpha-Tocopherol (1) · triheptanoin (1)
Jerry Vockley, MD, PhD (1) · Oregon Health and Science University (1) · RTI International (1)
United States (3)
Curt Scharfe, MD, PhD (1) · Jerry Vockley, MD, PhD (1) · Melanie Gillingham, Ph.D (1)
F32DK065400 (1)