Carglumic acid ( DrugBank: Carglumic acid )

3 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
245	プロピオン酸血症	4
246	メチルマロン酸血症	4
251	尿素サイクル異常症	1

245. プロピオン酸血症

臨床試験数 : 13 / 薬物数 : 16 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 6

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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	NCT05040178 (ClinicalTrials.gov)	June 30, 2022	16/6/2021	An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics	A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hypera ...	Hyperammonemia;Methylmalonic Acidemia;Propionic Acidemia	Drug: Carglumic Acid	Recordati Rare Diseases	Target PharmaSolutions, Inc.	Recruiting	N/A	N/A	All	20		United States
2	NCT04284917 (ClinicalTrials.gov)	November 28, 2019	24/2/2020	Long-term Efficacy of Carglumic Acid in Organic Acidemia.	Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid(Carbaglu®) in Patients Wi ...	Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)	Drug: Carglumic Acid (Carbaglu®)	National Taiwan University Hospital	NULL	Enrolling by invitation	18 Years	N/A	All	5	N/A	Taiwan
3	NCT04176523 (ClinicalTrials.gov)	January 15, 2019	20/11/2019	Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ...	Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ...	Methylmalonic Acidemia;Propionic Acidemia	Drug: Carglumic Acid	Recordati Rare Diseases	NULL	Recruiting	6 Months	99 Years	All	75		France;Germany;Italy;Norway;Spain;Sweden;United Kingdom
4	NCT02426775 (ClinicalTrials.gov)	November 2015	22/4/2015	Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia	Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carba ...	Propionic Acidemia;Methylmalonic Acidemia	Drug: Carglumic Acid	King Abdullah International Medical Research Center	NULL	Completed	N/A	15 Years	All	33	Phase 3	Saudi Arabia

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246. メチルマロン酸血症

臨床試験数 : 23 / 薬物数 : 31 - (DrugBank : 8) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 23

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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	NCT05040178 (ClinicalTrials.gov)	June 30, 2022	16/6/2021	An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics	A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hypera ...	Hyperammonemia;Methylmalonic Acidemia;Propionic Acidemia	Drug: Carglumic Acid	Recordati Rare Diseases	Target PharmaSolutions, Inc.	Recruiting	N/A	N/A	All	20		United States
2	NCT04284917 (ClinicalTrials.gov)	November 28, 2019	24/2/2020	Long-term Efficacy of Carglumic Acid in Organic Acidemia.	Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid(Carbaglu®) in Patients Wi ...	Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)	Drug: Carglumic Acid (Carbaglu®)	National Taiwan University Hospital	NULL	Enrolling by invitation	18 Years	N/A	All	5	N/A	Taiwan
3	NCT04176523 (ClinicalTrials.gov)	January 15, 2019	20/11/2019	Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ...	Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ...	Methylmalonic Acidemia;Propionic Acidemia	Drug: Carglumic Acid	Recordati Rare Diseases	NULL	Recruiting	6 Months	99 Years	All	75		France;Germany;Italy;Norway;Spain;Sweden;United Kingdom
4	NCT02426775 (ClinicalTrials.gov)	November 2015	22/4/2015	Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia	Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carba ...	Propionic Acidemia;Methylmalonic Acidemia	Drug: Carglumic Acid	King Abdullah International Medical Research Center	NULL	Completed	N/A	15 Years	All	33	Phase 3	Saudi Arabia

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251. 尿素サイクル異常症

臨床試験数 : 54 / 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28

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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	JPRN-jRCTs041190055	24/07/2019	24/07/2019	Carglumic Acid Administration for OTC Deficienscy	A Clinical Study of Carglumic Acidin Patients with Late-Onset Ornithine Transcarbamylase Deficiency ...	ornitine transcarbamylase deficiency patients	administraton of carglumic acid	Ito Tetsuya	NULL	Complete	Not applicable	< 75age old	Both	2	Phase 4	Japan

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