| Dojolvi (triheptanoin) / Ultragenyx |
2022-001539-10: A Randomized Study to Determine the Effect of Triheptanoin in Pediatric Patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Estudio aleatorizado para determinar el efecto de la triheptanoína en pacientes pediátricos con trastornos de la oxidación de ácidos grasos de cadena larga (LC-FAOD) |
|
|
| Ongoing | 3 | 60 | Europe, RoW | Dojolvi®, Miglyol® 812 N, UX007, NA, Oral liquid, Dojolvi®, Miglyol® 812 N | Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc. | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Trastornos de la oxidación de ácidos grasos de cadena larga (LC-FAOD), Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Trastornos de la oxidación de ácidos grasos de cadena larga (LC-FAOD), Diseases [C] - Nutritional and Metabolic Diseases [C18] | | | | |
NCT05933200 / 2022-001539-10: A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD |
|
|
| Recruiting | 3 | 60 | Europe, RoW | Triheptanoin, UX007, Dojolvi, MCT Oil, Medium-chain Triglyceride | Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmaceutical Inc. | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | 09/26 | 09/26 | | |
A-TC7, NCT04513002: Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis |
|
|
| Completed | 2a/2b | 30 | RoW | Triheptanoin | The University of Queensland, National Health and Medical Research Council, Australia | Ataxia Telangiectasia | 03/23 | 07/23 | | |
NCT03301532: Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D |
|
|
| Completed | 2 | 10 | US | Triheptanoin, C7 oil | University of Texas Southwestern Medical Center, National Institute of Neurological Disorders and Stroke (NINDS) | GLUT1DS1 | 06/21 | 07/22 | | |
NCT03181399: Diet Treatment Glucose Transporter Type 1 Deficiency (G1D) |
|
|
| Active, not recruiting | 2 | 45 | US | Triheptanoin, C7 | University of Texas Southwestern Medical Center, National Institute of Neurological Disorders and Stroke (NINDS) | GLUT1DS1, Epilepsy, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders, Glucose Transport Defect, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome | 09/24 | 09/24 | | |
NCT06067802: Study of Triheptanoin for the Prevention of Hypoglycemia in Patients with Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) |
|
|
| Recruiting | 2 | 8 | US | Triheptanoin, Dojolvi | Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc | Medium-chain Acyl-CoA Dehydrogenase Deficiency | 03/26 | 07/26 | | |
ACTRN12614000082606: Pilot study of the treatment of patients with sporadic Inclusion Body Myositis with the Anaplerotic medication Triheptanoin |
|
|
| Not yet recruiting | 1 | 3 | | | Ultragenyx | Sporadic Inclusion body Myositis | | | | |
NCT06340685: Triheptanoin for Children with Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency |
|
|
| Recruiting | 1 | 6 | US | Triheptanoin, Dojolvi | Jirair Krikor Bedoyan, Ultragenyx Pharmaceutical Inc | Pyruvate Dehydrogenase Complex Deficiency | 12/26 | 06/27 | | |
NCT02500082: Triheptanoin (UX007) to Treat Citrate Transporter Deficiency |
|
|
| No Longer Available | N/A | | US | triheptanoin, UX007 | Irina A Anselm | Citrate Transporter Deficiency, SLC13A5 Gene Mutation | | | | |
NCT02968953: Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome |
|
|
| No Longer Available | N/A | | US | Triheptanoin, UX007 | Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc | Glucose Transporter 1 Deficiency Syndrome | | | | |
| No Longer Available | N/A | | US | Triheptanoin, C7 oil, Heptanoate, heptanoic acid | Juan Pascual | Glut1 Deficiency Syndrome, Glucose Transporter Type 1 Deficiency Syndrome | | | | |
NCT01461304: Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism |
|
|
| No Longer Available | N/A | | US | triheptanoin, C7 | Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome | | | | |
| Available | N/A | | NA | Triheptanoin, UX007 | Ultragenyx Pharmaceutical Inc | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | | | | |
