DDrare: Database of Drug Development for Rare Diseases

227. オスラー病
［臨床試験数：49，薬物数：69（DrugBank：21），標的遺伝子数：23，標的パスウェイ数：132］

Searched query = "Osler disease", "Hereditary hemorrhagic telangiectasia", "Osler-Weber-Rendu 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 = AVASTIN; AVASTIN 25 mg/ml - Konzentrat zur Herstellung einer Infusionsloesung; Acetate; Avastin; BEVACIZUMAB; Bevacizumab; Bevacuzimab spray; Blood samples; Chloride; Dicynone; Doxycycline; Doxycycline Hyclate; EMEA/H/C/000712; ETAMSYLATE; Estriol; Etamsylate; Ferrous sulphate 200mg oral tablet; Floseal; Gold; Itraconazole; Mupirocin; NINTEDANIB; NaCl; Nintedanib; Nintédanib; OCTREOTIDE; OCTREOTIDE ACETATE; OFEV; Octreotide; Octreotide LAR; Other: Epistaxis charts; Other: Packing; Other: Standard Treatment; Other: Standard care; PHYSIOLOGISCHE Kochsalzloesung Fresenius - Infusionsloesung; Pazopanib; Pegylated Interferon Alpha2b; Pomalidomide; Pomalidomide Oral Product; Propranolol; Propranolol treatment; RVG 18236; Ranibizumab; SODIUM CHLORIDE; SUB16402MIG; Sandostatin LAR; Sandostatin LAR 20 mg; Sclerotherapy; Sodium chloride; Soy protein isolate; Sporanox; Sterile saline; Submucosal Bevacizumab; THALIDOMIDE; THALIDOMIDE CELGENE*28CPS 50MG; TIMOLOL MALEATE; Tacrolimus; Tacrolimus (Advagraf); Tacrolimus capsule (low-dose); Tamoxifen; Thalidomide; Timolol; Timolol Alcon 0.5% collyre; Timolol Gel; Timolol nasal spray; Topical timolol maleate; Tranexamic Acid; Tranexamic acid; Vit D

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	NCT04646356 (ClinicalTrials.gov)	October 20, 2020	20/10/2020	Tacrolimus Crossover Trial for Hereditary Hemorrhagic Telangiectasia (HHT)	Tacrolimus Crossover Trial for Hereditary Hemorrhagic Telangiectasia (HHT)	Hereditary Hemorrhagic Telangiectasia;Epistaxis Nosebleed	Drug: Tacrolimus capsule (low-dose);Drug: Placebo	St. Michael's Hospital, Toronto	United States Department of Defense	Recruiting	18 Years	N/A	All	30	Phase 2	Canada
2	EUCTR2019-003585-40-NL (EUCTR)	17/12/2019	30/09/2019	Tacrolimus for bleeding in hereditary hemorrhagic telangiectasia patients	An uncontrolled, open label pilot-study assessing the efficacy in reducing bleeding severity, and the safety of oral tacrolimus in patients with hereditary hemorrhagic telangiectasia - Tacrolimus for bleeding in HHT patients	Gastrointestinal bleeding and epistaxis caused by hereditary hemorrhagic telangiectasia. Hereditary Hemorrhagic Telangiectasia (HHT) is an autosomal dominant inherited disease characterized by mucocutaneous telangiectasis. Telangiectasis predominantly observed in the nasal mucosa and gut, and are abnormal, thin walled blood vessel that can easily rupture leading to hemorrhage. MedDRA version: 20.0;Level: LLT;Classification code 10038554;Term: Rendu-Osler-Weber syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		St. Antonius Hospital	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Phase 2	Netherlands

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

NCT04646356
(ClinicalTrials.gov)

October 20, 2020

20/10/2020

Tacrolimus Crossover Trial for Hereditary Hemorrhagic Telangiectasia (HHT)

Hereditary Hemorrhagic Telangiectasia;Epistaxis Nosebleed

Drug: Tacrolimus capsule (low-dose);Drug: Placebo

St. Michael's Hospital, Toronto

United States Department of Defense

Recruiting

18 Years

N/A

All

Phase 2

Canada

EUCTR2019-003585-40-NL
(EUCTR)

17/12/2019

30/09/2019

Tacrolimus for bleeding in hereditary hemorrhagic telangiectasia patients

An uncontrolled, open label pilot-study assessing the efficacy in reducing bleeding severity, and the safety of oral tacrolimus in patients with hereditary hemorrhagic telangiectasia - Tacrolimus for bleeding in HHT patients

Gastrointestinal bleeding and epistaxis caused by hereditary hemorrhagic telangiectasia. Hereditary Hemorrhagic Telangiectasia (HHT) is an autosomal dominant inherited disease characterized by mucocutaneous telangiectasis. Telangiectasis predominantly observed in the nasal mucosa and gut, and are abnormal, thin walled blood vessel that can easily rupture leading to hemorrhage.
MedDRA version: 20.0;Level: LLT;Classification code 10038554;Term: Rendu-Osler-Weber syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

St. Antonius Hospital

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Netherlands