DDrare: Database of Drug Development for Rare Diseases

Bezafibrate (400mg) in addition to standard 15-20 mg/kg/jour udca therapy (DrugBank: Bezafibrate, UDCA)

6 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
20	副腎白質ジストロフィー	0
93	原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］	0
94	原発性硬化性胆管炎	1
160	先天性魚鱗癬	0
316	カルニチン回路異常症	0
324	メチルグルタコン酸尿症	0

20. 副腎白質ジストロフィー　［臨床試験数：49，薬物数：86（DrugBank：29），標的遺伝子数：18，標的パスウェイ数：112］

Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy", "AMN"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 49 trial found

93. 原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］　［臨床試験数：230，薬物数：215（DrugBank：51），標的遺伝子数：34，標的パスウェイ数：107］

Searched query = "Primary biliary cholangitis", "Primary biliary cirrhosis", "PBC"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 230 trial found

94. 原発性硬化性胆管炎　［臨床試験数：134，薬物数：105（DrugBank：37），標的遺伝子数：18，標的パスウェイ数：131］

Searched query = "Primary sclerosing cholangitis", "PSC"
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.

1 / 134 trial found

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	NCT04309773 (ClinicalTrials.gov)	March 2020	18/10/2019	Efficacy of 24 Month of Bezafibrate in Primary Sclerosing Cholangitis With Persistent Cholestasis Despite Ursodeoxycholic Acid Therapy	Double Blind, Multicentric, Randomized, Placebo-controlled Trial, Evaluating the Efficacy of 24 Month of Bezafibrate in Primary Sclerosing Cholangitis With Persistent Cholestasis Despite Ursodeoxycholic Acid Therapy	Primary Sclerosing Cholangitis;Cholestasis	Drug: Bezafibrate (400mg) in addition to standard 15-20 mg/kg/jour UDCA therapy;Drug: Placebo of Bezafibrate in addition to standard UDCA therapy	Assistance Publique - Hôpitaux de Paris	NULL	Not yet recruiting	18 Years	75 Years	All	104	Phase 3	France

160. 先天性魚鱗癬　［臨床試験数：32，薬物数：62（DrugBank：18），標的遺伝子数：15，標的パスウェイ数：107］

Searched query = "Congenital ichthyosis", "Keratinopathic ichthyosis", "Epidermolytic ichthyosis", "Harlequin ichthyosis", "Congenital Ichthyosiform Erythroderma", "Foliate ichthyosis", "Ichthyosis syndrome", "Netherton syndrome", "Sjogren-Larsson syndrome", "Sjögren-Larsson syndrome", "Keratitis-ichtyosis-deafness syndrome", "Dorfman-Chanarin syndrome", "Neutral lipid storage disease", "NLSD", "Multiple sulfatase deficiency", "Austin disease", "Recessive X-linked ichthyosis", "RXLI", "X-linked recessive ichthyosis", "Ichthyosis, brittle hair, impaired intelligence, decreased fertility and short stature", "IBID", "Trichothiodystrophy", "Follicular ichthyosis", "Congenital hemidysplasia, ichthyosiform erythroderma or nevus, and limb defects syndrome", "CHILD syndrome", "Conradi-Hunermann-Happle syndrome", "Conradi-Hünermann-Happle syndrome", "CHHS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 32 trial found

316. カルニチン回路異常症　［臨床試験数：4，薬物数：10（DrugBank：4），標的遺伝子数：2，標的パスウェイ数：9］

Searched query = "Carnitine cycle disorder", "Carnitine palmitoyltransferase I deficiency", "CPT1 deficiency", "Carnitine palmitoyltransferase II deficiency", "CPT2 deficiency", "Carnitine-acylcarnitine translocase deficiency", "CACT deficiency", "Carnitine transporter deficiency", "OCTN-2 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 4 trial found

324. メチルグルタコン酸尿症　［臨床試験数：3，薬物数：4（DrugBank：3），標的遺伝子数：1，標的パスウェイ数：8］

Searched query = "Methylglutaconic aciduria", "3-methylglutaconyl-CoA hydratase deficiency", "3-MGA", "Barth syndrome", "Costeff syndrome", "Mitochondrial respiratory chain disorder", "Dilated cardiomyopathy with ataxia syndrome", "DCMA syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 3 trial found