Zavesca (Miglustat) ( DrugBank: Miglustat )

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1 disease

告示番号	疾患名（ページ内リンク）	臨床試験数
19	ライソゾーム病	2

19. ライソゾーム病

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臨床試験数 : 899 /  薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

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	NCT00672022 (ClinicalTrials.gov)	July 2004	2/5/2008	Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses	Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset GM2 Gangliosidosis: Single and Steady State Oral Doses	GM2 Gangliosidoses;Tay-Sachs;Sandhoff Disease	Drug: Zavesca (Miglustat)	Children's Research Institute	Actelion	Completed	6 Months	5 Years	Both	10	Phase 3	United States
2	EUCTR2022-002514-16-Outside-EU/EEA (EUCTR)		29/07/2022	A Clinical Study to evaluate the Safety and Efficacy of miglustat (Zavesca®) for the treatment of Niemann-Pick Disease Type C (NPC) in Chinese subjects	A single-arm uncontrolled 12-month Clinical Study to evaluate the Safety and Efficacy of miglustat (Zavesca®) for the Treatment of Niemann-Pick Disease Type C (NPC) in Chinese subjects	Niemann-Pick Disease Type C (NPC) MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Zavesca (Miglustat) Product Name: Miglustat INN or Proposed INN: Miglustat Other descriptive name: Zavesca	Actelion Pharmaceuticals Trading (Shanghai) Co., Ltd	NULL	NA			Female: yes Male: yes		Phase 4	China