DDrare: Database of Drug Development for Rare Diseases

124. 皮質下梗塞と白質脳症を伴う常染色体優性脳動脈症
［臨床試験数：11，薬物数：13（DrugBank：4），標的遺伝子数：3，標的パスウェイ数：11］

Searched query = "Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy", "CADASIL", "Autosomal dominant cerebral artery disease with subcortical infarct and leukoencephalopathy", "Autosomal dominant cerebral artery disease"
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.

Drug = 6R-BH4; ARICEPT 5*28 CPR 5 MG; Antiplatelets; Aricept; BH4; Dabigatran; Donepezil; Donepezil hydrochloride; E2020; Fremanezumab; HOV-12020 (Palm tocotrienols complex); Phenoptin; Sapropterin

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	EUCTR2007-004370-55-IT (EUCTR)	14/12/2007	11/12/2007	Effects of tetrahydrobiopterin (6R-BH4) on flow-mediated dilation in CADASIL patients: a randomised controlled trial - CADASIL BH4	Effects of tetrahydrobiopterin (6R-BH4) on flow-mediated dilation in CADASIL patients: a randomised controlled trial - CADASIL BH4	CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy), is a rare autosomal dominant disorder characterized by recurrent strokes starting in mid-adulthood and leading in some to severe motor disability with pseudobulbar palsy and dementia of the subcortical type MedDRA version: 9.1;Level: HLGT;Classification code 10008804;Term: Chromosomal abnormalities and abnormal gene carriers	Product Name: Phenoptin Product Code: 6R-BH4 INN or Proposed INN: sapropterin	AZIENDA OSPEDALIERA OSPEDALE NIGUARDA CA' GRANDA (A.O. DI RILIEVO NAZIONALE)	NULL	Not Recruiting			Female: yes Male: yes			Italy

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EUCTR2007-004370-55-IT
(EUCTR)

14/12/2007

11/12/2007

Effects of tetrahydrobiopterin (6R-BH4) on flow-mediated dilation in CADASIL patients: a randomised controlled trial - CADASIL BH4

CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy), is a rare autosomal dominant disorder characterized by recurrent strokes starting in mid-adulthood and leading in some to severe motor disability with pseudobulbar palsy and dementia of the subcortical type
MedDRA version: 9.1;Level: HLGT;Classification code 10008804;Term: Chromosomal abnormalities and abnormal gene carriers

Product Name: Phenoptin
Product Code: 6R-BH4
INN or Proposed INN: sapropterin

AZIENDA OSPEDALIERA OSPEDALE NIGUARDA CA' GRANDA (A.O. DI RILIEVO NAZIONALE)

NULL

Not Recruiting

Female: yes
Male: yes

Italy