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	NCT02582775 (ClinicalTrials.gov)	March 2016	16/10/2015	MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs	MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions	Epidermolysis Bullosa	Drug: Thymoglobulin;Drug: Cyclophosphamide;Drug: Fludarabine;Radiation: Total Body Irradiation;Procedure: Bone marrow infusion;Drug: Tacrolimus;Drug: Mycophenolate Mofetil;Biological: Donor mesenchymal stem cell infusions;Drug: Busulfan	Masonic Cancer Center, University of Minnesota	NULL	Recruiting	N/A	25 Years	All	84	Phase 2	United States
2	EUCTR2012-000605-72-NL (EUCTR)	06/02/2014	12/08/2013	Stem cell transplantation with cord blood and mesenchymal stem cells after reduced intensity conditioning for severe forms of the blistering disease epidermolysis bullosa	Unrelated cord blood transplantation after reduced toxicity conditioning with mesenchymal stromal cell co-infusion in patients with severe epidermolysis bullosa - CB+MSCforEB	The source population consists of patients referred to or within the UMC Groningen because they have diagnosed clinically and genetically severe generalized RDEB.;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]	Product Name: TC-MSC INN or Proposed INN: Mesenchymal stromal cells Trade Name: Busilvex Trade Name: Fludarabine Product Name: FLudarabine Trade Name: THYMOGLOBULINE Product Name: Thymoglobuline	Universitair Medisch Centrum Utrecht	NULL	Not Recruiting			Female: yes Male: yes	11	Phase 2	Netherlands
3	NCT01033552 (ClinicalTrials.gov)	January 2010	14/12/2009	Biochemical Correction of Severe EB by Allo HSCT and Off-the-shelf MSCs	MT2009-09: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation and Off-the-shelf Mesenchymal Stem Cells	Epidermolysis Bullosa	Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Anti-thymocyte globulin;Drug: Cyclosporine A;Drug: Mycophenolate mofetil;Procedure: Mesenchymal stem cell transplantation;Radiation: Total body irradiation;Procedure: Bone marrow or umbilical cord blood (UCG) stem cell transplantation	Masonic Cancer Center, University of Minnesota	NULL	Recruiting	N/A	25 Years	All	75	Phase 2	United States
4	NCT00478244 (ClinicalTrials.gov)	April 2007	23/5/2007	Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa	Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa	Epidermolysis Bullosa	Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Procedure: hematopoietic bone marrow transplantation	Masonic Cancer Center, University of Minnesota	NULL	Terminated	N/A	25 Years	All	7	N/A	United States

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

NCT02582775
(ClinicalTrials.gov)

March 2016

16/10/2015

MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs

MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions

Epidermolysis Bullosa

Drug: Thymoglobulin;Drug: Cyclophosphamide;Drug: Fludarabine;Radiation: Total Body Irradiation;Procedure: Bone marrow infusion;Drug: Tacrolimus;Drug: Mycophenolate Mofetil;Biological: Donor mesenchymal stem cell infusions;Drug: Busulfan

Masonic Cancer Center, University of Minnesota

NULL

Recruiting

N/A

25 Years

All

Phase 2

United States

EUCTR2012-000605-72-NL
(EUCTR)

06/02/2014

12/08/2013

Stem cell transplantation with cord blood and mesenchymal stem cells after reduced intensity conditioning for severe forms of the blistering disease epidermolysis bullosa

Unrelated cord blood transplantation after reduced toxicity conditioning with mesenchymal stromal cell co-infusion in patients with severe epidermolysis bullosa - CB+MSCforEB

The source population consists of patients referred to or within the UMC Groningen because they have diagnosed clinically and genetically severe generalized RDEB.;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Product Name: TC-MSC
INN or Proposed INN: Mesenchymal stromal cells
Trade Name: Busilvex
Trade Name: Fludarabine
Product Name: FLudarabine
Trade Name: THYMOGLOBULINE
Product Name: Thymoglobuline

Universitair Medisch Centrum Utrecht

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

Netherlands

NCT01033552
(ClinicalTrials.gov)

January 2010

14/12/2009

Biochemical Correction of Severe EB by Allo HSCT and Off-the-shelf MSCs

MT2009-09: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation and Off-the-shelf Mesenchymal Stem Cells

Epidermolysis Bullosa

Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Anti-thymocyte globulin;Drug: Cyclosporine A;Drug: Mycophenolate mofetil;Procedure: Mesenchymal stem cell transplantation;Radiation: Total body irradiation;Procedure: Bone marrow or umbilical cord blood (UCG) stem cell transplantation

Masonic Cancer Center, University of Minnesota

NULL

Recruiting

N/A

25 Years

All

Phase 2

United States

NCT00478244
(ClinicalTrials.gov)

April 2007

23/5/2007

Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa

Epidermolysis Bullosa

Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Procedure: hematopoietic bone marrow transplantation

Masonic Cancer Center, University of Minnesota

NULL

Terminated

N/A

25 Years

All

N/A

United States