DDrare: Database of Drug Development for Rare Diseases

316. カルニチン回路異常症　［臨床試験数：3，薬物数：10（DrugBank：4），標的遺伝子数：2，標的パスウェイ数：8］

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 of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Drug = Bezafibrate; Bezafibrate (drug); Break in l-carnitine treatment; Carnitine; Glycerol; Glycerol/saline; Heparin; Hyperinsulinemic euglycemic clamp; Intralipid/heparin; L-carnitine

No.	TrialID	Date_ enrollement	Last_Refreshed_ on	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT02517307	February 2016	29 July 2019	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		Recruiting	18 Years	N/A	All	50	N/A	United States
2	NCT02226419	August 2013	25 May 2015	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	Not recruiting	18 Years	N/A	Both	14	N/A
3	NCT00336167	June 2006	19 February 2015	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