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	EUCTR2018-003890-91-PL (EUCTR)	29/09/2020	13/11/2018	Treatment of wound in the course of Epidermolysis Bullosa, chronic venous leg ulceration and thermal injury by biological dressing made of mesynchemal stem cells seeded on acellular human skin.	The development of innovative advanced therapy medicinal product (biological dressing of the human race) in the treatment of Epidermolysis Bullosa (EB) and other chronic wounds.	BIOOPA dressing will be used in the treatment of wounds in the course of Epidermolysis Bullosa, chronic venous leg ulceration and thermal injury (second degree burn). MedDRA version: 20.0;Level: PT;Classification code 10014989;Term: Epidermolysis bullosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]	Product Name: BIOOPA dressing INN or Proposed INN: Human Allogeneic WJ-MSCs Other descriptive name: WHARTON’S JELLY DERIVED MESENCHYMAL STEM CELLS	Medical University of Warsaw	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	100	Phase 1;Phase 2	Poland

No.

TrialID

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enrollment

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registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2018-003890-91-PL
(EUCTR)

29/09/2020

13/11/2018

Treatment of wound in the course of Epidermolysis Bullosa, chronic venous leg ulceration and thermal injury by biological dressing made of mesynchemal stem cells seeded on acellular human skin.

The development of innovative advanced therapy medicinal product (biological dressing of the human race) in the treatment of Epidermolysis Bullosa (EB) and other chronic wounds.

BIOOPA dressing will be used in the treatment of wounds in the course of Epidermolysis Bullosa, chronic venous leg ulceration and thermal injury (second degree burn).
MedDRA version: 20.0;Level: PT;Classification code 10014989;Term: Epidermolysis bullosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Product Name: BIOOPA dressing
INN or Proposed INN: Human Allogeneic WJ-MSCs
Other descriptive name: WHARTON’S JELLY DERIVED MESENCHYMAL STEM CELLS

Medical University of Warsaw

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

100

Phase 1;Phase 2

Poland