4 completed, 3 unknown, 1 active not recruiting. 2 Phase 2, 1 Early Phase 1, 1 Not applicable, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Completed 2 |
| Early Phase 1 | 1 | Completed 1 |
| Not applicable | 1 | Unknown 1 |
| Phase 1 | 1 | Completed 1 |
| Phase 3 | 1 | Unknown 1 |
Follow Carnitine palmitoyl transferase 2 deficiency: new trials by email Literature Review of Carnitine palmitoyl transferase 2 deficiency trials Research Report on Carnitine palmitoyl transferase 2 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 |
|---|---|---|---|---|---|---|
| Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan Obtaining Dry Blood Spots From Healthy Newborns (Aged 1-7 Days), Obtaining Dry Blood Spots From Healthy Children Aged 8 Days - 7 Years, Obtaining Dry Blood Spots From Healthy Children Aged 8-18 Years - Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease | Unknown | West Kazakhstan Medical University | 2250 | 2022-10-03 | 2023-06-18 | |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders Nutritional Ketone Supplement, Isocaloric Placebo Supplement - Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Carnitine Palmitoyltransferase Deficiency 2 | Early Phase 1 | Completed | Oregon Health and Science University | 5 | 2022-08-01 | 2024-04-02 |
| 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 | |
| Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy Sugar - Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors | Not applicable | Unknown | Copenhagen University Hospital, Rigshospitalet | 60 | 2016-01-01 | 2019-10-16 |
| Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects Bezafibrate, Placebo - Carnitine Palmitoyltransferase II Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency | Phase 2 | Completed | Copenhagen University Hospital, Rigshospitalet | 12 | 2009-10-01 | 2012-05-30 |
| Bezafibrate Trial in CPT2 Deficiency bezafibrate (drug) - Carnitine Palmitoyl Transferase 2 Deficiency | Phase 3 | Unknown | Assistance Publique - Hôpitaux de Paris | 12 | 2006-06-01 | 2007-04-09 |
| High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders Diet counseling - Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency | Phase 1 / Phase 2 | Completed | Oregon Health and Science University | 13 | 2006-01-01 | 2020-04-29 |
Trifunctional Protein Deficiency With Myopathy And Neuropathy (6) · VLCAD deficiency (6) · Medium chain acyl CoA dehydrogenase deficiency (4) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (3) · Acidemia, isovaleric (3) · Argininosuccinic Aciduria (3) · Beta ketothiolase deficiency (3) · Biotinidase Deficiency (3) · Carbamoyl-Phosphate Synthase I Deficiency Disease (3) · Carnitine palmitoyl transferase 1A deficiency (3) · Carnitine-Acylcarnitine Translocase Deficiency (3) · Citrullinemia (3) · Glutaric Acidemia I (3) · Glycogen Storage Disease Type II (3)
Amino Acid Metabolism, Inborn Errors (4) · Brain Diseases (4) · Brain Diseases, Metabolic (4) · Brain Diseases, Metabolic, Inborn (4) · Central Nervous System Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Metabolic Diseases (4) · Mitochondrial Diseases (4) · Nervous System Diseases (4) · Nutritional and Metabolic Diseases (4) · Carbohydrate Metabolism, Inborn Errors (3) · Connective Tissue Diseases (3) · Genetic Diseases, X-Linked (3) · Hyperhomocysteinemia (3)
Bezafibrate (2) · Aging (1) · Diet, Fat-Restricted (1) · Diet, High-Protein (1) · Diet, High-Protein Low-Carbohydrate (1) · Pharmaceutical Preparations (1) · Sugars (1)
Copenhagen University Hospital, Rigshospitalet (2) · Oregon Health and Science University (2) · Assistance Publique - Hôpitaux de Paris (1) · Centre Hospitalier Universitaire de Liege (1) · RTI International (1) · West Kazakhstan Medical University (1)
Denmark (2) · United States (2) · Belgium (1) · France (1) · Kazakhstan (1)
Curt Scharfe, MD, PhD (1) · Gulmira M. Zharmakhanova, MD, PhD (1) · Karen L Madsen, MD (1) · Laurent Servais (1) · Lyazzat M. Syrlybayeva, MD (1) · Victoria I. Kononets, MD, MS (1)
PMID 30990523, PMID 31315600, PMID 31492716, PMID 35142618, PMID 38283151