DDrare: Database of Drug Development for Rare Diseases

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.

Drug = 11C-CSar; 3108006977; ACETYLCYSTEINE; ACIDO OBETICOLICO; ATRA; AZT; Acetylcysteine; All-trans retinoic acid; Azathioprine; BEFIZAL L.P. 400 mg, comprimé enrobé à libération prolongée; BEZAFIBRATE; BTT1023; BTT1023 IV Infusion 20 mg/mL, 5 mL Drug Product; Befizal; Bezafibrate; Bezafibrate (400mg) in addition to standard 15-20 mg/kg/jour UDCA therapy; Bezalip; Budesonide; C10AB02; CILO; CM-101; Calcium; Capecitabine; Cenicriviroc; Cenicriviroc 150 mg; Cilofexor; Cis-4-Hydroxy-L-Proline; Cladribine; Curcumin; DHA; DUR-928; Docosahexaenoic Acid (DHA); Docosahexaenoic acid; Engineered recombinant human FGF19; Entyvio; Erlotinib; Erlotinib (Tarceva); FLUIMUCIL*20CPR EFF 600MG; Fecal Microbiota Transplantation; Fenofibrate; Fluimucil; GS-6624; GS-9674; Gluten-free diet; HTD1801; Humanised IgG1 monoclonal antibody against human eotaxin-2; Hymecromone; ICG; INT-747, OCA; LUM001; MBX-8025; MLN0002; Metronidazole; Minocycline; Mitomycin; Mitomycin C; Mizoribine; NGM282; NorUDCA; NorUrsodeoxycholic Acid; NorUrsodeoxycholic acid; Normal saline; Norursodeoxycholic acid; OCA; OCA 1,5 mg; OCA 5 mg; Oral Vancomycin; Oral all-trans retinoic acid (ATRA); Orbcel-C; PLN-74809; Phenylbutyrate; Probiotics; Procedure: Liver transplantation; Procedure: Pre liver transplantation laparoscopic hand assisted staging; Proline; Radiation: 45 Gy external radiations; Radiation: Endoluminal bile duct Brachytherapy; Rec-h-FGF19; Retinoic acid; Rituximab; SELADELPAR; Seladelpar; Simtuzimab; Simtuzumab; Simvastatin; Simvastatin 40mg; Sulfasalazine; TBC; Thalidomide; UCMSC; UDCA; Ursodeoxycholic Acid; Ursodeoxycholic acid; Ursodeoxycholic acid (UDCA); Ursofalk; Ursolic acid; VEDOLIZUMAB; Vancomycin; Vedolizumab; Vedolizumab IV; Vidofludimus; Vidofludimus calcium; Volixibat; Xifaxan; [18F]FP-R01-MG-F2

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	EUCTR2014-005558-21-GB (EUCTR)	25/03/2015	06/01/2015	A phase 2a trial to evaluate the safety and tolerability of LUM001 in subjects with Primary Sclerosing Cholangitis (PSC) during 14 weeks of treatment.	A Pilot, Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Primary Sclerosing Cholangitis (PSC) - CAMEO STUDY	Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease of unknown aetiology.PSC is characterized by inflammation and fibrosis of the intra- and extrahepatic biliary tree resulting in diffuse multifocal stricture formation leading to biliary cirrhosis, portal hypertension and liver failure. PSC is a life-threatening and debilitating disease. The median survival from diagnosis in symptomatic patients with PSC has been estimated to be 12 years. MedDRA version: 17.1;Level: LLT;Classification code 10036732;Term: Primary sclerosing cholangitis;System Organ Class: 100000004871;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]	Product Name: LUM001 INN or Proposed INN: LUM001	Lumena Pharmaceuticals LLC	NULL	Not Recruiting			Female: yes Male: yes	20	Phase 2	United States;United Kingdom
2	NCT02061540 (ClinicalTrials.gov)	March 2014	11/2/2014	Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis	A Pilot, Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Primary Sclerosing Cholangitis	Primary Sclerosing Cholangitis (PSC)	Drug: LUM001	Mirum Pharmaceuticals, Inc.	NULL	Completed	18 Years	80 Years	All	27	Phase 2	United States;Canada;United Kingdom

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

EUCTR2014-005558-21-GB
(EUCTR)

25/03/2015

06/01/2015

A phase 2a trial to evaluate the safety and tolerability of LUM001 in subjects with Primary Sclerosing Cholangitis (PSC) during 14 weeks of treatment.

A Pilot, Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Primary Sclerosing Cholangitis (PSC) - CAMEO STUDY

Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease of unknown aetiology.PSC is characterized by inflammation and fibrosis of the intra- and extrahepatic biliary tree resulting in diffuse multifocal stricture formation leading to biliary cirrhosis, portal hypertension and liver failure. PSC is a life-threatening and debilitating disease. The median survival from diagnosis in symptomatic patients with PSC has been estimated to be 12 years.
MedDRA version: 17.1;Level: LLT;Classification code 10036732;Term: Primary sclerosing cholangitis;System Organ Class: 100000004871;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Product Name: LUM001
INN or Proposed INN: LUM001

Lumena Pharmaceuticals LLC

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United States;United Kingdom

NCT02061540
(ClinicalTrials.gov)

March 2014

11/2/2014

Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis

A Pilot, Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Primary Sclerosing Cholangitis

Primary Sclerosing Cholangitis (PSC)

Drug: LUM001

Mirum Pharmaceuticals, Inc.

NULL

Completed

18 Years

80 Years

All

Phase 2

United States;Canada;United Kingdom