Cabaletta

( DrugBank: Cabaletta / KEGG DRUG: - )

2 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
18	脊髄小脳変性症（多系統萎縮症を除く。）	1

告示番号	疾患名（ページ内リンク）	臨床試験数
113	筋ジストロフィー	2

18. 脊髄小脳変性症（多系統萎縮症を除く。）

臨床試験数 : 76 / 薬物数 : 98 - (DrugBank : 31) / 標的遺伝子数 : 44 - 標的パスウェイ数 : 65

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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	NCT02147886 (ClinicalTrials.gov)	July 2014	21/5/2014	Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabalettain Patients With Machado-J ...	A Single-Center, Randomized, Double-Blind, Parallel-Group, Dose-Controlled Study, to Assess Safety, ...	Machado-Joseph Disease / Spinocerebellar Ataxia 3	Drug: Cabaletta for IV infusion once weekly during 24 weeks	Bioblast Pharma Ltd.	NULL	Completed	18 Years	75 Years	Both	15	Phase 2	Israel

臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170

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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	NCT02328482 (ClinicalTrials.gov)	January 2015	25/12/2014	Continuation Protocol to Protocol BBCO-001	A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to ...	Muscular Dystrophy, Oculopharyngeal (OPMD)	Drug: Tehalose 30gr	Bioblast Pharma Ltd.	NULL	Completed	18 Years	80 Years	All	9	Phase 3	Canada
2	NCT02015481 (ClinicalTrials.gov)	February 2014	8/12/2013	Safety Tolerability and Efficacy Study of Cabalettato Treat Oculopharyngeal Muscular Dystrophy (OPMD ...	Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Caba ...	Oculopharyngeal Muscular Dystrophy	Drug: Cabaletta	Bioblast Pharma Ltd.	NULL	Completed	18 Years	80 Years	All	25	Phase 2	United States;Canada;Israel