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	NCT04644627 (ClinicalTrials.gov)	December 1, 2020	20/11/2020	Topical Gentamicin Nonsense Suppression Therapy of EB	TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS	Epidermolysis Bullosa	Drug: Gentamicin Sulfate	Oslo University Hospital	NULL	Not yet recruiting	N/A	N/A	All	6	Phase 1;Phase 2	Norway
2	EUCTR2020-002337-15-NO (EUCTR)	16/09/2020	29/06/2020	TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA	TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS (THE GENTELBULL STUDY) - GENTELBULL	Epidermolysis bullosa caused by nonsense mutations or splice site mutations;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]	Trade Name: Infectogenta Product Name: Gentamicin sulfate 0.1% ointment	Oslo University Hospital	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	6	Phase 2	Norway
3	NCT04140786 (ClinicalTrials.gov)	October 31, 2019	23/10/2019	Optimizing IV Gentamicin in JEB	Optimization of Intravenous Gentamicin Treatment to Restore Functional Laminin 332 in JEB Patients With Nonsense Mutations	Junctional Epidermolysis Bullosa	Drug: Gentamicin Sulfate, Injectable	University of Southern California	NULL	Recruiting	N/A	N/A	All	6	Phase 1;Phase 2	United States
4	NCT03392909 (ClinicalTrials.gov)	July 5, 2018	2/1/2018	Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)	Restoration of Full-Length Type VII Collagen in RDEB Patients With Nonsense Mutations After Intravenous Gentamicin Treatment	Recessive Dystrophic Epidermolysis Bullosa	Drug: Gentamicin	University of Southern California	NULL	Recruiting	7 Years	N/A	All	9	Phase 1;Phase 2	United States
5	NCT03526159 (ClinicalTrials.gov)	June 1, 2018	17/4/2018	Gentamicin for Junctional Epidermolysis Bullosa	A Pilot Study of the Restoration of Functional Laminin 332 in JEB Patients With Nonsense Mutations After Topical and Intravenous Gentamicin Treatment	Junctional Epidermolysis Bullosa	Drug: Gentamicin Sulfate	University of Southern California	NULL	Recruiting	N/A	N/A	All	6	Phase 1;Phase 2	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
6	NCT03012191 (ClinicalTrials.gov)	February 2, 2017	4/1/2017	Gentamicin for RDEB	Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa Patients With Nonsense Mutations	Recessive Dystrophic Epidermolysis Bullosa	Drug: Gentamicin Sulfate	University of Southern California	NULL	Completed	N/A	N/A	All	6	Phase 1;Phase 2	United States
7	NCT02698735 (ClinicalTrials.gov)	February 25, 2016	19/2/2016	Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients		Recessive Dystrophic Epidermolysis Bullosa	Drug: Gentamicin;Drug: Placebo	University of Southern California	NULL	Completed	N/A	N/A	All	5	Phase 1;Phase 2	NULL

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

NCT04644627
(ClinicalTrials.gov)

December 1, 2020

20/11/2020

Topical Gentamicin Nonsense Suppression Therapy of EB

TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS

Epidermolysis Bullosa

Drug: Gentamicin Sulfate

Oslo University Hospital

NULL

Not yet recruiting

N/A

All

Phase 1;Phase 2

Norway

EUCTR2020-002337-15-NO
(EUCTR)

16/09/2020

29/06/2020

TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA

TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS (THE GENTELBULL STUDY) - GENTELBULL

Epidermolysis bullosa caused by nonsense mutations or splice site mutations;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trade Name: Infectogenta
Product Name: Gentamicin sulfate 0.1% ointment

Oslo University Hospital

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Norway

NCT04140786
(ClinicalTrials.gov)

October 31, 2019

23/10/2019

Optimizing IV Gentamicin in JEB

Optimization of Intravenous Gentamicin Treatment to Restore Functional Laminin 332 in JEB Patients With Nonsense Mutations

Junctional Epidermolysis Bullosa

Drug: Gentamicin Sulfate, Injectable

University of Southern California

NULL

Recruiting

N/A

All

Phase 1;Phase 2

United States

NCT03392909
(ClinicalTrials.gov)

July 5, 2018

2/1/2018

Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Restoration of Full-Length Type VII Collagen in RDEB Patients With Nonsense Mutations After Intravenous Gentamicin Treatment

Recessive Dystrophic Epidermolysis Bullosa

Drug: Gentamicin

University of Southern California

NULL

Recruiting

7 Years

N/A

All

Phase 1;Phase 2

United States

NCT03526159
(ClinicalTrials.gov)

June 1, 2018

17/4/2018

Gentamicin for Junctional Epidermolysis Bullosa

A Pilot Study of the Restoration of Functional Laminin 332 in JEB Patients With Nonsense Mutations After Topical and Intravenous Gentamicin Treatment

Junctional Epidermolysis Bullosa

Drug: Gentamicin Sulfate

University of Southern California

NULL

Recruiting

N/A

All

Phase 1;Phase 2

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

NCT03012191
(ClinicalTrials.gov)

February 2, 2017

4/1/2017

Gentamicin for RDEB

Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa Patients With Nonsense Mutations

Recessive Dystrophic Epidermolysis Bullosa

Drug: Gentamicin Sulfate

University of Southern California

NULL

Completed

N/A

All

Phase 1;Phase 2

United States

NCT02698735
(ClinicalTrials.gov)

February 25, 2016

19/2/2016

Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients

Recessive Dystrophic Epidermolysis Bullosa

Drug: Gentamicin;Drug: Placebo

University of Southern California

NULL

Completed

N/A

All

Phase 1;Phase 2

NULL