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 w ... show all
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