316. Carnitine cycle disorder
4 clinical trials, 10 drugs (DrugBank: 4 drugs), 2 drug target genes, 9 drug target pathways
Searched query = "Carnitine cycle disorder", "Carnitine palmitoyltransferase I deficiency", "CPT1 deficiency", "Carnitine palmitoyltransferase II deficiency", "CPT2 deficiency", "Carnitine-acylcarnitine translocase deficiency", "CACT deficiency", "Carnitine transporter deficiency", "OCTN-2 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02517307 (ClinicalTrials.gov) | February 2016 | 2/7/2015 | Fatty Acid Oxidation Defects and Insulin Sensitivity | Role of Fatty Acid Oxidation Defects in Insulin Sensitivity | Very Long-chain Acyl-CoA Dehydrogenase Deficiency;Trifunctional Protein Deficiency;Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency;Medium-chain Acyl-CoA Dehydrogenase Deficiency;Normal Volunteers;Carnitine Palmitoyltransferase II Deficiency, Myopathic | Drug: Intralipid/Heparin;Drug: Glycerol/Saline;Drug: Hyperinsulinemic euglycemic clamp | Oregon Health and Science University | NULL | Recruiting | 18 Years | N/A | All | 50 | N/A | United States |
| 2 | NCT02226419 (ClinicalTrials.gov) | August 2013 | 5/8/2013 | Fat and Sugar Metabolism During Exercise, With and Without L-carnitine in Patients With Carnitine Transporter Deficiency | Fat and Carbohydrate Metabolism During Exercise, With and Without L-carnitine Supplementation in Patients With Carnitine Transporter Deficiency | Carnitine Transporter, Plasma-membrane, Deficiency of | Dietary Supplement: Break in L-carnitine treatment | Karen Lindhardt Madsen | Rigshospitalet, Denmark | Completed | 18 Years | N/A | Both | 14 | N/A | NULL |
| 3 | NCT00983788 (ClinicalTrials.gov) | October 2009 | 23/9/2009 | Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects | Evaluation of the Effect of Bezafibrate on Muscle Metabolism During Exercise in Patients With CPTII and VLCAD Deficiency | Carnitine Palmitoyltransferase II Deficiency;Very Long Chain Acyl Coa Dehydrogenase Deficiency | Drug: Bezafibrate;Other: Placebo | Rigshospitalet, Denmark | Institut de Myologie, Pitié-Salpêtrière Hospital, France | Completed | 18 Years | 70 Years | Both | 12 | Phase 2 | Denmark |
| 4 | NCT00336167 (ClinicalTrials.gov) | June 2006 | 9/6/2006 | Bezafibrate Trial in CPT2 Deficiency | Clinical Trial on the Effect of Bezafibrate in the Muscular Form of Carnitine Palmitoyltransferase 2 Deficiency | Carnitine Palmitoyl Transferase 2 Deficiency | Drug: bezafibrate (drug) | Assistance Publique - Hôpitaux de Paris | Association Française contre les Myopathies (AFM), Paris;Institut National de la Santé Et de la Recherche Médicale, France | Recruiting | 18 Years | N/A | Both | 12 | Phase 3 | France |