DDrare: Database of Drug Development for Rare Diseases

36. 表皮水疱症
［臨床試験数：147，薬物数：170（DrugBank：40），標的遺伝子数：32，標的パスウェイ数：113］

Searched query = "Epidermolysis bullosa", "EBS", "JEB", "DDEB", "RDEB", "Kindler syndrome"
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 = 3400930060797; ABH001; AC-203; AGLE 102; ALLANTOIN; ALLO-ASC-DFU; ASC; AVCN583601 (3% Cannabidiol cream); Allantoin; Allo-APZ2-EB; Allogeneic mesenchymal stromal cells; Allogeneic skin-derived ABCB5-positive mesenchymal stem cells; Allogenic mesenchymal stromal cells; Alwextin cream; Anti-thymocyte globulin; BIOOPA dressing; BIRCH BARK EXTRACT; Birch bark extract; Blood test; Botulinic toxin; Broccoli; Busilvex; Busulfan; CALCIPOTRIOL; CCP-020; COL7A1-SIN retroviral vector engineered autologous tissue-engineered skin; CX501; Calcipotriol; Cannabidiol; Cell transplantation; Cultured allogeneic fibroblasts; Cyclophosphamide; Cyclosporine; Cyclosporine A; Células madre mesenquimales haploidénticas derivadas de médula ósea; DIACEREIN; Device: Occlusive non adherent dressing; Diacerein; Diacerein 1% Ointment; Diacerein 1% ointment; Diacerin cream; Donor mesenchymal stem cell infusions; Dysport; Dysport® (Botulinumtoxin A (Btx A)); EB-101; Episalvan gel; Erythrocine; Erythrocine oral; Erythromycin; Erythromycine; Ex-vivo expanded autologous human keratinocytes suspension containing epidermal stem cells genetically modified with a gamma-retroviral (rv) vector expressing the full-length LAMB3 cDNA.; FCX-007 (debcoemagene autoficel; FLudarabine; Fibroblasts; Fludarabine; Fludarabine phosphate; Gene-modified autologous fibroblasts; Genetically corrected cultured epidermal autograft (ATMP); Gentamicin; Gentamicin Sulfate; Gentamicin Sulfate, Injectable; Gentamicin sulfate 0.1% ointment; Granulocyte Colony Stimulating Factor; Granulocyte Colony Stimulating Factor (GCSF); HOLOGENE17; HOLOGENE7; HP802-247; Hologene 5; Human Allogeneic WJ-MSCs; Human Umbilical Cord Blood-derived Mesenchymal Stem Cells; Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh; Human fibroblast-derived dermal substitute, Dermagraft; ICX-RHY-013; Infectogenta; Isotretinoin; Ixekizumab; Jojoba oil; Jojoba oil with broccoli sprout extract; LOSARTAN POTASSIUM; LZRSE-Col7A1 Engineered Autologous Epidermal Sheets; Losartan; Losartan HEXAL; Losartan potassium; MESENCHYMAL STEM CELLS (MSCS); Mesenchymal Stem Cells extracted from bone marrow; Mesenchymal Stromal Cells; Mesenchymal stem cells derived from bone marrow (BM-MSCs); Mesenchymal stromal cells; Methoxsalen; Morphine; Morphine sulphate in intrasite gel; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Na; Normal saline; ON 01910.NA; ON 01910.Na; Oleogel-S10; Oral erythromycin; Other: Control wound treatment; Other: Quality-of-Life Assessment; POLYPHENON E; PTR-01; Phosphate; Polyphenon E; Potassium; Pregabalin; Procedure: Bone marrow infusion; Procedure: Bone marrow or umbilical cord blood (UCG) stem cell transplantation; Procedure: Mesenchymal stem cell transplantation; Procedure: hematopoietic bone marrow transplantation; Psorcutan-Ointment; QR-313; QR-313 Gel for Topical (Cutaneous) Administration; REGE pro dressing; RGN-137; Radiation: Total Body Irradiation; Radiation: Total body irradiation; Reduced Intensity Transplant conditioning; Rigosertib; Rigosertib Oral Capsules / Rigosertib Intravenous; Rigosertib Sodium; Rituximab; Ropivacaine; SD-101; SD-101 Dermal Cream (6%); SD-101 dermal cream (3%); SD-101 dermal cream (6%); SD-101-6.0 cream; SIN LV Mediated ex vivo transduced autologous fibroblasts expressing codon-optimised COL7A1; SIN RV-mediated COL7A1 corrected autologous skin equivalent; See below for FCX-007 description); Sericare; Sericare®; Serlopitant; Serlopitant Tablet; SimpleCare; Sirolimus; Sirolimus 2%; Sirolimus, 2%; Sulfate; TC-MSC; THYMOGLOBULINE; Tacrolimus; Taltz; Taltz 80mg; Tetracyclin; Thymoglobulin; Thymoglobuline; Thymosin Beta 4; Thymosin beta 4; Topical BPM31510 3.0% Cream; Topical Beremagene Geperpavec; Topical beremagene geperpavec; Topical opiod; Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL17A1-encoding retroviral vector; Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL7A1-encoding retroviral vector; Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL7A1-encoding retroviral vector, obtained from secondary culture of ex vivo expanded autologous human keratinocytes.; Trimethoprim; Triterpene dry extract from birch cork; Unavailable; VPD-737; Vehicle; Vehicle (SD-101 0%); WHARTON’S JELLY DERIVED MESENCHYMAL STEM CELLS; WJ-MSCs; Zorblisa; [Hologene 5 DS]; [allo-APZ2-EB]

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	NCT04177498 (ClinicalTrials.gov)	January 1, 2021	7/11/2019	Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC	A Pilot, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma	Recessive Dystrophic Epidermolysis Bullosa	Drug: Rigosertib Sodium;Other: Quality-of-Life Assessment	Thomas Jefferson University	Onconova Therapeutics, Inc.	Not yet recruiting	18 Years	79 Years	All	6	Early Phase 1	United States
2	NCT03786237 (ClinicalTrials.gov)	January 2021	14/11/2018	Rigosertib for RDEB-SCC	A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma	Epidermolysis Bullosa Dystrophica;Squamous Cell Carcinoma	Drug: Rigosertib Oral Capsules / Rigosertib Intravenous	Prof. Johann Bauer	NULL	Recruiting	18 Years	79 Years	All	12	Phase 1;Phase 2	Austria
3	EUCTR2016-003832-19-AT (EUCTR)	11/12/2017	09/08/2017	Clinical Trial to evaluate the efficacy of the drug Rigosertib against non- melanoma skin cancer in Butterfly Children.	A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients with Recessive Dystrophic Epidermolysis bullosa associated Locally Advanced/Metastatic Squamous Cell Carcinoma - Rigosertib for RDEB-SCC	Recessive dystrophic epidermolysis bullosa (RDEB) is a severe genodermatose caused by mutations in COL7A1, characterized by generalized skin blistering and involvement of mucous membranes. Aggressive metastasizing squamous cell carcinomas (SCCs) are a common complication, which reduce patients’ average life expectancy to less than 40 years. The aim of this study is to evaluate anti-tumor activity of oral Rigosertib, a PLK1 inhibitor, in RDEB patients diagnosed with SCCs.;Therapeutic area: Diseases [C] - Cancer [C04]	Product Name: Rigosertib Product Code: ON 01910.Na INN or Proposed INN: rigosertib Other descriptive name: ON 01910.NA Product Name: Rigosertib Product Code: ON 01910.Na INN or Proposed INN: rigosertib Other descriptive name: ON 01910.NA	Gemeinn. Salzburger Landeskliniken BetriebsGesmbH, University Hospital for Dermatology, EB-House Austria	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	12	Phase 2	Austria

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

NCT04177498
(ClinicalTrials.gov)

January 1, 2021

7/11/2019

Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC

A Pilot, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma

Recessive Dystrophic Epidermolysis Bullosa

Drug: Rigosertib Sodium;Other: Quality-of-Life Assessment

Thomas Jefferson University

Onconova Therapeutics, Inc.

Not yet recruiting

18 Years

79 Years

All

Early Phase 1

United States

NCT03786237
(ClinicalTrials.gov)

January 2021

14/11/2018

Rigosertib for RDEB-SCC

A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma

Epidermolysis Bullosa Dystrophica;Squamous Cell Carcinoma

Drug: Rigosertib Oral Capsules / Rigosertib Intravenous

Prof. Johann Bauer

NULL

Recruiting

18 Years

79 Years

All

Phase 1;Phase 2

Austria

EUCTR2016-003832-19-AT
(EUCTR)

11/12/2017

09/08/2017

Clinical Trial to evaluate the efficacy of the drug Rigosertib against non- melanoma skin cancer in Butterfly Children.

A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients with Recessive Dystrophic Epidermolysis bullosa associated Locally Advanced/Metastatic Squamous Cell Carcinoma - Rigosertib for RDEB-SCC

Recessive dystrophic epidermolysis bullosa (RDEB) is a severe genodermatose caused by mutations in COL7A1, characterized by generalized skin blistering and involvement of mucous membranes. Aggressive metastasizing squamous cell carcinomas (SCCs) are a common complication, which reduce patients’ average life expectancy to less than 40 years. The aim of this study is to evaluate anti-tumor activity of oral Rigosertib, a PLK1 inhibitor, in RDEB patients diagnosed with SCCs.;Therapeutic area: Diseases [C] - Cancer [C04]

Product Name: Rigosertib
Product Code: ON 01910.Na
INN or Proposed INN: rigosertib
Other descriptive name: ON 01910.NA
Product Name: Rigosertib
Product Code: ON 01910.Na
INN or Proposed INN: rigosertib
Other descriptive name: ON 01910.NA

Gemeinn. Salzburger Landeskliniken BetriebsGesmbH, University Hospital for Dermatology, EB-House Austria

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Austria