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 36. 表皮水疱症 [臨床試験数:80,薬物数:118(DrugBank:31),標的遺伝子数:27,標的パスウェイ数:94] 

Searched query = "Epidermolysis bullosa", "Kindler syndrome"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04140786October 31, 201911 November 2019Optimizing IV Gentamicin in JEBOptimization of Intravenous Gentamicin Treatment to Restore Functional Laminin 332 in JEB Patients With Nonsense MutationsJunctional Epidermolysis BullosaDrug: Gentamicin Sulfate, InjectableUniversity of Southern CaliforniaRecruitingN/AN/AAll6Phase 1/Phase 2United States
2NCT03786237September 201930 September 2019Rigosertib for RDEB-SCCA Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell CarcinomaEpidermolysis Bullosa Dystrophica;Squamous Cell CarcinomaDrug: Rigosertib Oral Capsules / Rigosertib Oral Liquid Solution / Rigosertib IntravenousProf. Johann BauerRecruiting18 Years79 YearsAll12Phase 1/Phase 2Austria
3NCT03928093August 7, 20192 September 2019Pregabalin Treatment for RDEB Pain and ItchA Double-blind, Randomized, Cross-over, Feasibility Trial of Pregabalin for the Treatment of Recessive Dystrophic Epidermolysis Bullosa-associated Neuropathic Pain and ItchPain, Neuropathic;Itch;Epidermolysis BullosaDrug: PregabalinThe Hospital for Sick ChildrenEpidemolysis Bullosa Research PartnershipRecruiting8 Years40 YearsAll15Phase 3Canada
4NCT03578029May 22, 20199 September 2019Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis BullosaA Randomized,Single-Blind, Placebo-controlled, Self-matched Pairing, Independent Evaluated Study to Evaluate the Efficacy and Safety of RGN-137 Topical Gel in Subjects With Junctional and Dystrophic Epidermolysis Bullosa (CELEB)Junctional Epidermolysis Bullosa;Dystrophic Epidermolysis BullosaDrug: RGN-137;Drug: PlaceboLenus Therapeutics, LLCRecruiting4 YearsN/AAll15Phase 2United States
5NCT03836001April 18, 201927 May 2019A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis BullosaA Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis BullosaEpidermolysis BullosaDrug: Serlopitant Tablet;Drug: Placebo Oral TabletStanford UniversityEpidermolysis Bullosa Research Partnership;Menlo TherapeuticsRecruiting13 YearsN/AAll40Phase 2United States
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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agemax
Inclusion_
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Target_
size
PhaseCountries
6NCT03529877January 16, 201930 September 2019Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis BullosaAn Interventional, Multicenter, Single Arm, Phase I/IIa Clinical Trial to Investigate the Efficacy and Safety of Allo-APZ2-EB on Epidermolysis Bullosa (EB)Recessive Dystrophic Epidermolysis BullosaBiological: allo-APZ2-EBRHEACELL GmbH & Co. KGFGK Clinical Research GmbH;Granzer Regulatory Consulting & Services;Ticeba GmbHRecruiting12 Months55 YearsAll18Phase 1/Phase 2United States;Austria;France;Germany;Italy;United Kingdom
7NCT03752905January 9, 201925 February 2019A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)A Phase 1/2 Randomized, Saline-Controlled, Single-Blind, Multiple Ascending Dose, Dose-Escalation, Multi-Center Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)Recessive Dystrophic Epidermolysis BullosaDrug: PTR-01;Drug: Normal salinePhoenix Tissue Repair, Inc.Recruiting18 YearsN/AAll14Phase 1/Phase 2United States
8EUCTR2018-001009-98-AT05/11/201828 February 2019Study to investigate the efficacy and safety of allo-APZ2-EB on wound healing of epidermolysis bullosa (EB)An interventional, multicenter, single arm, phase I/IIa clinical trial to investigate the efficacy and safety of allo-APZ2-EB on epidermolysis bullosa (EB)Recessive dystrophic epidermolysis bullosa (RDEB)
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: allo-APZ2-EB
Product Code: allo-APZ2-EB
Pharmaceutical Form: Suspension for injection
Current Sponsor code: T202-3
Other descriptive name: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells
Concentration unit: million organisms/ml million organisms/millilitre
Concentration type: equal
Concentration number: 10-
RHEACELL GmbH & Co. KGAuthorised Female: yes
Male: yes
18Phase 1;Phase 2France;United States;Austria;Germany;Italy;United Kingdom
9NCT03468322October 20, 201829 April 2019A Double-blind, Intra-individual Comparison, POC Trial of AC-203 in EB PatientsA Double-blind, Intra-individual Comparison, Proof-of-concept Trial of Topical AC-203 in Patients With Inherited Epidermolysis BullosaInherited Epidermolysis BullosaDrug: AC-203;Drug: VehicleTWi Biotechnology, Inc.Not recruiting2 YearsN/AAll9Phase 2Taiwan
10NCT03632265October 1, 201820 August 2018Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis BullosaVITAL: A Pivotal Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) (GENE TRANSFER)Recessive Dystrophic Epidermolysis BullosaBiological: EB-101Jean Yuh TangAbeona Therapeutics, IncNot recruiting6 YearsN/AAll15Phase 3
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
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Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03605069July 2, 201826 August 2019Topical QR-313 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Due to Mutation(s) in Exon 73 of the COL7A1geneA First in Human, Double-blind, Randomized, Intra-subject Placebo-controlled, Multiple Dose Study of QR-313 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With RDEB Due to Mutation(s) in Exon 73 of the COL7A1 GeneEpidermolysis Bullosa Dystrophica, RecessiveDrug: QR-313;Drug: PlaceboWings Therapeutics Inc.Recruiting6 YearsN/AAll8Phase 1/Phase 2United States;France;Spain
12NCT03526159July 1, 201825 June 2018Gentamicin for Junctional Epidermolysis BullosaA Pilot Study of the Restoration of Functional Laminin 332 in JEB Patients With Nonsense Mutations After Topical and Intravenous Gentamicin TreatmentJunctional Epidermolysis BullosaDrug: Gentamicin SulfateUniversity of Southern CaliforniaNot recruitingN/AN/AAll6Phase 1/Phase 2United States
13NCT03392909July 201825 June 2018Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)Restoration of Full-Length Type VII Collagen in RDEB Patients With Nonsense Mutations After Intravenous Gentamicin TreatmentRecessive Dystrophic Epidermolysis BullosaDrug: GentamicinUniversity of Southern CaliforniaNot recruiting7 YearsN/AAll9Phase 1/Phase 2United States
14NCT04153630May 17, 201811 November 2019Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis BullosaSafety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis BullosaEpidermolysis Bullosa Dystrophica, RecessiveBiological: mesenchymal stem cells derived from bone marrow (BM-MSCs)Instituto de Investigación Hospital Universitario La PazUniversidad Carlos III Madrid (TERMeG);St John’s Institute of Dermatology Kings College London;Instituto de Salud Carlos III;DEBRA;CIBER Enfermedades rarasNot recruiting12 Months18 YearsAll9Phase 1/Phase 2Spain
15NCT03472287May 10, 201825 February 2019Study of Diacerein After Maximum Use in Patients With EBA Multi-center Study to Evaluate the Pharmacokinetics of Diacerein and Rhein and the Safety of Diacerein After Maximum Use, Topical Administration of CCP-020 (Diacerein 1% Ointment) to Patients With Epidermolysis Bullosa (EB)Epidermolysis BullosaDrug: CCP-020 (Diacerein 1% Ointment)Castle Creek Pharmaceuticals, LLCRecruiting6 MonthsN/AAll20Phase 1United States;Netherlands;United Kingdom;France
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Status
Inclusion_
agemin
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agemax
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PhaseCountries
16NCT03536143May 7, 201811 February 2019Topical Bercolagene Telserpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis BullosaA Phase II Study of Bercolagene Telserpavec (KB103), a Non-Integrating, Replication-Incompetent Herpes Simplex Virus 1 (HSV-1) Vector Expressing the Human Collagen VII (COL7) Protein, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)Dystrophic Epidermolysis BullosaBiological: Topical bercolagene telserpavecKrystal Biotech, Inc.Recruiting5 YearsN/AAll4Phase 2United States
17NCT03453632March 1, 201812 March 2018Injections of Botulinic Toxin in Plantar Lesions of Localized Epidermolysis Bullosa SimplexEvaluation of the Efficacy of Injections of Botulinic Toxin in Plantar Lesions of Patients Suffering From Localized Epidermolysis Bullosa Simplex : Double Blind Randomized Controlled Study.Epidermolysis Bullosa SimplexDrug: Botulinic toxin;Drug: PlaceboUniversity Hospital, ToulouseNot recruiting18 YearsN/AAll25Phase 2/Phase 3France
18EUCTR2017-003757-41-AT04/01/201830 April 2018An International Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)An International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)Epidermolysis Bullosa Simplex (EBS)
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: Diacerein
Product Code: Diacerein
Pharmaceutical Form: Ointment
INN or Proposed INN: DIACEREIN
CAS Number: 13739-02-1
Current Sponsor code: CCP-020
Other descriptive name: Diacerein
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 1-
Castle Creek Pharmaceuticals, LLCAuthorisedFemale: yes
Male: yes
84Phase 2France;United States;Australia;Austria;Israel;Netherlands;Germany;Italy;United Kingdom
19EUCTR2018-000439-29-NL1 October 2018A Multi-center Study to Evaluate How CCP-020 (Diacerein 1% ointment) is Absorbed in the Blood and Removed from the Body in Patients with Epidermolysis Bullosa (EB)A Multi-center Study to Evaluate the Pharmacokinetics of Diacerein and Rhein and the Safety of Diacerein after Maximum Use, Topical Administration of CCP-020 (Diacerein 1% ointment) to Patients with Epidermolysis Bullosa (EB)Epidermolysis Bullosa (EB)
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: Diacerein
Product Code: CCP-020
Pharmaceutical Form: Ointment
INN or Proposed INN: DIACEREIN
CAS Number: 13739-02-1
Current Sponsor code: CCP-020
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 1-
Castle Creek Pharmaceuticals, LLCNot AvailableFemale: yes
Male: yes
20Phase 1France;United States;Germany;Netherlands;United Kingdom
20EUCTR2016-003832-19-AT11/12/201712 November 2018Clinical 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-SCCRecessive 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
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: rigosertib
CAS Number: 592542-60-4
Current Sponsor code: ON 01910.NA
Other descriptive name: ON 01910.NA
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 70-
Product Name: Rigosertib
Product Code: ON 01910.Na
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: rigosertib
CAS Number: 592542-60-4
Current Sponsor code: ON 01910.NA
Other descriptive name: ON 01910.NA
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 280-
Product Name: Rigosertib
Product Code: ON 01910.Na
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: rigosertib
CAS Number: 592542-60-4
Current Sponsor code: ON 01910.NA
Other descriptive name: ON 01910.NA
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 75-
Product Name: Rigosertib
Product Code: ON 01910.Na
Pharmaceutical Form: Concentrate for oral solution
INN or Proposed INN: rigosertib
CAS Number: 592542-60-4
Current Sponsor code: ON 01910.NA
Other descriptive name: ON 01910.NA
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 75-
Gemeinn. Salzburger Landeskliniken BetriebsGesmbH, University Hospital for Dermatology, EB-House AustriaAuthorisedFemale: yes
Male: yes
12Phase 2Austria
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT03389308December 1, 20172 July 2018Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa SimplexAn International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex (EBS)Epidermolysis Bullosa SimplexDrug: Diacerein 1% OintmentCastle Creek Pharmaceuticals, LLCRecruiting6 Months99 YearsAll80Phase 2United States
22EUCTR2017-000606-37-ES16/11/201726 February 2018Safety study of mesenchymal stem cells in the treatment of Recessive Dystrophic Epidermolysis Bullosa.Safety and preliminary efficacy study of infusing mesenchymal stem cells derived from bone marrow for treating Recessive Dystrophic Epidermolysis Bullosa.Recessive Dystrophic Epidermolysis Bullosa;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Product Name: Mesenchymal Stem Cells extracted from bone marrow
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Células madre mesenquimales haploidénticas derivadas de médula ósea
Current Sponsor code: MSCs
Other descriptive name: MESENCHYMAL STEM CELLS (MSCS)
Concentration unit: Other
Concentration type: range
Concentration number: 2000000-4000000
Fundación para la Investigación Biomédica Hospital Universitario La PazAuthorisedFemale: yes
Male: yes
9Phase 1Spain
23EUCTR2016-004427-24-NL06/09/201728 February 2019An International Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS) [DELIVERS Study]Epidermolysis Bullosa Simplex (EBS)
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: Diacerein
Product Code: Diacerein
Pharmaceutical Form: Ointment
INN or Proposed INN: DIACEREIN
CAS Number: 13739-02-1
Current Sponsor code: CCP-020, AC-203
Other descriptive name: Diacerein
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 1-
Pharmaceutical form of the placebo: Ointment
Route of administration of the placebo: Topical use (Noncurrent)
Castle Creek Pharmaceuticals, LLCNot Recruiting Female: yes
Male: yes
80Phase 2France;United States;Austria;Australia;Israel;Germany;Netherlands;Italy;United Kingdom
24EUCTR2016-004427-24-DE24/08/201722 October 2018An International Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS) [DELIVERS Study]Epidermolysis Bullosa Simplex (EBS)
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: Diacerein
Product Code: Diacerein
Pharmaceutical Form: Ointment
INN or Proposed INN: DIACEREIN
CAS Number: 13739-02-1
Current Sponsor code: CCP-020, AC-203
Other descriptive name: Diacerein
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 1-
Pharmaceutical form of the placebo: Ointment
Route of administration of the placebo: Topical use (Noncurrent)
Castle Creek Pharmaceuticals, LLCNot RecruitingFemale: yes
Male: yes
80Phase 2France;United States;Austria;Australia;Israel;Netherlands;Germany;Italy;United Kingdom
25EUCTR2016-000095-17-AT17/08/20172 October 2017Gene therapy for patients with Junctional Epidermolysis Bullosa (JEB)PROSPECTIVE, OPEN-LABEL, UNCONTROLLED CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF AUTOLOGOUS CULTURED EPIDERMAL GRAFTS CONTAINING EPIDERMAL STEM CELLS GENETICALLY MODIFIED WITH A GAMMA-RETROVIRAL (RV) VECTOR CARRYING COL17A1 cDNA FOR RESTORATION OF EPIDERMIS IN PATIENTS WITH JUNCTIONAL EPIDERMOLYSIS BULLOSA - HOLOGENE 17JEB is genetically and clinically heterogeneous characterized by mutations in COL17A1,encoding for Collagen17 protein. COLXVII is a collagenous transmembrane type II protein component of the hemidesmosomes and plays a key role in the adhesion of epidermis to the basement membrane. JEB is characterized by a wide range of severity: skin blistering, which can be generalized or localized, different degrees of mucosal involvement, enamel defects, dystrophy or loss of nails, and alopecia can occur.;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Product Name: HOLOGENE17
Product Code: HOLOGENE17
Pharmaceutical Form: Living tissue equivalent
INN or Proposed INN: Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL17A1-encoding retroviral vector
Current Sponsor code: Hologene17 DS
Concentration unit: % percent
Concentration type: range
Concentration number: 50-100
Holostem Terapie Avanzate s.r.l.AuthorisedFemale: yes
Male: yes
Phase 1;Phase 2Austria
No.TrialIDDate_
enrollement
Last_Refreshed_
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agemin
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26NCT03183934July 201716 December 2017A Follow-up Study to Evaluate the Efficacy and Safety of ALLO-ASC-DFU in ALLO-ASC-EB-101 Clinical TrialA Follow-up Study to Evaluate the Efficacy and Safety for the Patients With ALLO-ASC-DFU Treatment in Phase 1/2 Clinical Trial of ALLO-ASC-EB-101Dystrophic Epidermolysis BullosaBiological: ALLO-ASC-DFUAnterogen Co., Ltd.Not recruiting2 Years60 YearsAll5N/AKorea, Republic of
27NCT03154333June 1, 20175 November 2018Safety and Efficacy of Diacerein 1% Ointment Topical Formulation Compared to Placebo for Subjects With Epidermolysis Bullosa Simplex (EBS)An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex (EBS) [DELIVERS Study]Epidermolysis Bullosa SimplexDrug: Diacerein 1% Ointment Topical Formulation;Drug: VehicleCastle Creek Pharmaceuticals, LLCNot recruiting4 YearsN/AAll80Phase 2United States;Australia;Austria;France;Germany;Israel;Netherlands;United Kingdom
28EUCTR2016-002066-32-GB24/05/201724 July 2017A Clinical Trial which compare the safety and efficacy of a wound gel, the study treatment, or a sunflower oil-based vehicle gel in patients with Inherited Epidermolysis Bullosa (EB)Double-blind, Randomised, Vehicle-controlled, Phase III, Efficacy and Safety Study with 24-month Open-label Follow up of Oleogel S10 in Patients with Inherited Epidermolysis Bullosa - EASE studyInherited Epidermolysis Bullosa
MedDRA version: 19.1 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
Pharmaceutical Form: Gel
INN or Proposed INN: Birch bark extract
CAS Number: 1640971-03-4
Current Sponsor code: Dry extract from betulae cortex
Other descriptive name: BIRCH BARK EXTRACT
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Gel
Route of administration of the placebo: Cutaneous use
Amryt Research LimitedAuthorisedFemale: yes
Male: yes
164Phase 3Turkey;Austria;Israel;Italy;United Kingdom;Switzerland;France;Mexico;Croatia;Australia;Germany;Ireland;Spain;Greece;United States
29NCT03068780March 29, 20174 November 2019Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis BullosaDouble Blind, Randomised, Vehicle Controlled, Phase III, Efficacy and Safety Study With 24-month Open-label Follow up of Oleogel-S10 in Patients With Inherited Epidermolysis BullosaEpidermolysis BullosaDrug: Oleogel-S10;Drug: PlaceboAmryt Research LimitedRecruitingN/AN/AAll250Phase 3United States;Argentina;Australia;Austria;Brazil;Chile;Colombia;Croatia;Czechia;Denmark;France;Georgia;Germany;Greece;Hong Kong;Hungary;Ireland;Israel;Italy;Romania;Russian Federation;Serbia;Singapore;Spain;Switzerland;Ukraine;United Kingdom;Belgium
30NCT03730584February 27, 20176 May 2019Evaluation of the Efficacy of ROPIVACAINE in Children and Young Adults With Hereditary Epidermolysis BullosaEvaluation of the Efficacy of a Topical Analgesic Treatment With ROPIVACAINE in Children and Young Adults With Hereditary Epidermolysis BullosaEpidermolysis BullosaDrug: Ropivacaine;Biological: Blood testAssistance Publique - Hôpitaux de ParisRecruitingN/A21 YearsAll10N/AFrance
No.TrialIDDate_
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31NCT03012191February 2, 201726 August 2019Gentamicin for RDEBGentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa Patients With Nonsense MutationsRecessive Dystrophic Epidermolysis BullosaDrug: Gentamicin SulfateUniversity of Southern CaliforniaNot recruitingN/AN/AAll6Phase 1/Phase 2United States
32NCT03942250January 201715 July 2019Uses of Irradiated Human Amniotic Membrane in the Treatment of Dystrophic Epidermolysis Bullosa PatientsUses of Irradiated Human Amniotic Membrane in the Treatment of Dystrophic Epidermolysis Bullosa PatientsEpidermolysis Bullosa;Chronic Skin UlcerBiological: REGE pro dressingEgyptian Atomic Energy AuthorityNot recruiting12 Years45 YearsAll8N/AEgypt
33EUCTR2017-004806-17-FR23 July 2018Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 geneA first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene - WINGSRecessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene
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: QR-313 Gel for Topical (Cutaneous) Administration
Product Code: QR-313
Pharmaceutical Form: Gel
INN or Proposed INN: unavailable
CAS Number: unavailable
Current Sponsor code: QR-313
Concentration unit: mg/g milligram(s)/gram
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Gel
Route of administration of the placebo: Cutaneous use
ProQR TherapeuticsNot AvailableFemale: yes
Male: yes
14Phase 1;Phase 2United States;France;Czech Republic;Canada;Austria;Germany;United Kingdom
34NCT02654483August 31, 201612 November 2018Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB PatientsA Phase 2 Trial of Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Epidermolysis Bullosa PatientsEpidermolysis Bullosa;PruritusOther: Placebo;Drug: VPD-737Jean Yuh TangEpidermolysis Bullosa Research Partnership;Menlo Therapeutics Inc.Not recruiting13 YearsN/AAll14Phase 2United States
35NCT02793960August 10, 201622 July 2019Topical BPM31510 3.0% Cream in Patients With Epidermolysis BullosaA Phase 1 Study to Evaluate the Safety, Pharmacokinetics and Therapeutic Effect of Topical BPM31510 3.0% Cream in Patients With Epidermolysis BullosaEpidermolysis BullosaDrug: topical BPM31510 3.0% CreamShasa HuBerg, LLCNot recruiting12 YearsN/AAll11Phase 1United States
No.TrialIDDate_
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PhaseCountries
36EUCTR2016-001967-35-AT19/07/201610 December 2018A study to assess the efficacy of a vitamin D-containing ointment applied on the skin to improve wound healing in butterfly childrenA double-blind, placebo-controlled cross-over study to assess the efficacy of topical calcipotriol (Psorcutan®-ointment containing 0.05 µg/g calcipotriol) to improve wound healing in dystrophic epidermolysis bullosa (DEB) - Topical calcipotriol in DEBSubjects with dystrophic epidermolysis bullosa (DEB) with a known mutation in the type VII collagen gene.
MedDRA version: 19.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Psorcutan-Ointment
Pharmaceutical Form: Ointment
INN or Proposed INN: CALCIPOTRIOL
CAS Number: 112828-00-9
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 0.05-
Pharmaceutical form of the placebo: Ointment
Route of administration of the placebo: Topical use (Noncurrent)
Universitätsklinik für Dermatologie, Paracelsus medizinische Privatuniversität SalzburgNot RecruitingFemale: yes
Male: yes
15Phase 2Austria
37EUCTR2015-004592-74-AT01/06/201611 June 2018Gene Therapy for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB).PROSPECTIVE, OPEN LABEL, UNCONTROLLED CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF AUTOLOGOUS CULTURED EPIDERMAL GRAFTS CONTAINING EPIDERMAL STEM CELLS GENETICALLY MODIFIED WITH A GAMMA-RETROVIRAL (RV) VECTOR CARRYING COL7A1 cDNA FOR RESTORATION OF EPIDERMIS IN PATIENTS WITH RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA. - HOLOGENE7RDEB is characterized by generalized skin blistering, erosions, crusts, atrophic scarring, onychodystrophy and loss of nails, mutilating pseudosyndactyly of hands and feet, as well as oral cavity lesions, esophageal strictures and eye and genitourinary tract lesions, all of which can also lead to anemia, iron deficiency and growth delay. Aggressive metastasizing squamous cell carcinomas are a common complication of RDEB, which reduce patients’ average life expectancy to less than 40 years.;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Product Name: HOLOGENE7
Product Code: HOLOGENE7
Pharmaceutical Form: Living tissue equivalent
INN or Proposed INN: Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL7A1-encoding retroviral vector
Current Sponsor code: Hologene7 DS
Other descriptive name: 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.
Concentration unit: % percent
Concentration type: range
Concentration number: 50-100
Holostem Terapie Avanzate s.r.l.AuthorisedFemale: yes
Male: yes
12Phase 1;Phase 2Austria
38EUCTR2014-004884-19-GB24/05/201620 August 2018Gene Therapy Trial in adults with Inherited Blistering Skin DiseasePhase I study of lentiviral-mediated COL7A1 gene-modified autologous fibroblasts in adults with recessive dystrophic epidermolysis bullosa (RDEB) - LENTICOL-FRecessive Dystrophic Epidermolysis Bullosa
MedDRA version: 18.1 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: SIN LV Mediated ex vivo transduced autologous fibroblasts expressing codon-optimised COL7A1
Product Code: Not applicable
Pharmaceutical Form: Suspension for injection in pre-filled syringe
King's College LondonGuy's and St Thomas' NHS Foundation TrustNot RecruitingFemale: yes
Male: yes
10Phase 1United Kingdom
39NCT02960997May 20167 October 2019Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) StudyA Prospective, Double-Blind, Cross-Over, Pilot Study to Assess Safety and Efficacy of Topical Sirolimus 2% in the Treatment of Plantar Blistering in Patients With Epidermolysis Bullous Simplex (EBS)Epidermolysis Bullosa Simplex;Epidermolysis Bullosa Simplex Kobner;Weber-Cockayne SyndromeDrug: Sirolimus, 2%;Drug: VehicleStanford UniversityNot recruiting4 YearsN/AAll8Phase 2United States
40NCT03016715May 201616 December 2017Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) StudyA Prospective, Double-Blind, Cross-Over, Pilot Study to Assess Safety and Efficacy of Topical Sirolimus 2% in the Treatment of Plantar Blistering in Patients With Epidermolysis Bullous Simplex (EBS)Epidermolysis Bullosa Simplex;Epidermolysis Bullosa Simplex Kobner;Weber-Cockayne SyndromeDrug: Sirolimus 2%;Drug: VehiclePremier Specialists, AustraliaRecruiting5 YearsN/AAll8Phase 2Australia
No.TrialIDDate_
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41NCT02582775March 20168 April 2019MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCsMT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell InfusionsEpidermolysis BullosaDrug: 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: BusulfanMasonic Cancer Center, University of MinnesotaRecruitingN/A25 YearsAll84Phase 2United States
42NCT02698735February 25, 201626 August 2019Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation PatientsRecessive Dystrophic Epidermolysis BullosaDrug: Gentamicin;Drug: PlaceboUniversity of Southern CaliforniaNot recruitingN/AN/AAll5Phase 1/Phase 2
43NCT02493816September 20157 October 2019Safety Study of Gene-modified Autologous Fibroblasts in Recessive Dystrophic Epidermolysis BullosaPhase I Study of Lentiviral-mediated COL7A1 Gene-modified Autologous Fibroblasts in Adults With Recessive Dystrophic Epidermolysis Bullosa.Recessive Dystrophic Epidermolysis BullosaDrug: Gene-modified autologous fibroblastsKing's College LondonUniversity College, LondonNot recruiting17 YearsN/AAll5Phase 1United Kingdom
44NCT02592954September 201516 December 2017Effect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal SkinEffect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal SkinEpidermolysis Bullosa Simplex;Pachyonychia CongenitaDrug: Jojoba oil with broccoli sprout extract;Drug: Jojoba oil (placebo)Johns Hopkins UniversityNot recruiting18 YearsN/AAll5Phase 1United States
45NCT02670330June 9, 201530 September 2019Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis BullosaAn Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of Zorblisa™ (SD-101-6.0) in Patients With Epidermolysis BullosaEpidermolysis BullosaDrug: SD-101-6.0 creamScioderm, Inc.Amicus TherapeuticsNot recruiting1 MonthN/AAll152Phase 3United States;Australia;Austria;France;Germany;Israel;Lithuania;Netherlands;Poland;Serbia;Spain;United Kingdom;Italy
No.TrialIDDate_
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46NCT02323789June 201516 December 2017Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis BullosaA Phase I/II Study Evaluating Allogeneic Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis BullosaRecessive Dystrophic Epidermolysis BullosaDrug: Mesenchymal stromal cellsKing's College LondonNot recruiting18 Years65 YearsAll10Phase 1/Phase 2United Kingdom
47NCT02470689June 201529 June 2015Diacerin for the Treatment of Epidermolysis Bullosa SimplexDiacerin for the Treatment of Epidermolysis Bullosa SimplexEpidermolysis Bullosa SimplexDrug: Diacerin creamTel-Aviv Sourasky Medical CenterNot recruiting6 Years19 YearsBoth50Phase 2
48EUCTR2014-005679-96-AT16/03/201524 September 2018An Investigation into the Long-term Safety of Zorblisa (SD-101-6.0) cream used by Patients with Epidermolysis Bullosa.An Open Label, Multi-center, Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients with Epidermolysis Bullosa - Study to Evaluate the Long-term Safety of Zorblisa in Patients with Epidermolysis BullosaEpidermolysis Bullosa
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: Zorblisa
Product Code: SD-101
Pharmaceutical Form: Cream
INN or Proposed INN: ALLANTOIN
CAS Number: 97-59-6
Other descriptive name: ALLANTOIN
Concentration unit: % (W/V) percent weight/volume
Concentration type: equal
Concentration number: 6-
Scioderm, INCNot RecruitingFemale: yes
Male: yes
150Phase 3Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands
49EUCTR2014-004500-30-GB13/02/201530 April 2019A prospective phase I/II study to evaluate the use of mesenchymal stromal (stem) cells for the treatment of skin disease in adults with recessive dystrophic epidermolysis bullosaA phase I/II study evaluating allogeneic mesenchymal stromal cells in adults with recessive dystrophic epidermolysis bullosa - ADSTEMRecessive dystrophic epidermolysis bullosa;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Product Name: Allogeneic mesenchymal stromal cells
Product Code: TC-MSC
Pharmaceutical Form: Injection
King's College LondonGuy's and St Thomas NHS Foundation TrustNot Recruiting Female: yes
Male: yes
10Phase 1;Phase 2United Kingdom
50EUCTR2014-002288-14-GB26/09/201428 February 2019An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis BullosaA Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa - Study of Effectiveness and Safety of SD-101 in Subjects with Epidermolysis Bullosa/ESSENCE StudyEpidermolysis Bullosa
MedDRA version: 19.1 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: Zorblisa
Product Code: SD-101
Pharmaceutical Form: Cream
INN or Proposed INN: ALLANTOIN
CAS Number: 97-59-6
Other descriptive name: ALLANTOIN
Concentration unit: % (W/V) percent weight/volume
Concentration type: equal
Concentration number: 6-
Pharmaceutical form of the placebo: Cream
Route of administration of the placebo: Topical use (Noncurrent)
Scioderm, An Amicus Therapeutics CompanyNot Recruiting Female: yes
Male: yes
150Phase 3United States;Serbia;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Belgium;Poland;Romania;Australia;Germany;Netherlands
No.TrialIDDate_
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51EUCTR2014-002288-14-AT08/08/20142 October 2017An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis BullosaA Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa - Study of Effectiveness and Safety of SD-101 in Subjects with Epidermolysis Bullosa/ESSENCE StudyEpidermolysis Bullosa
MedDRA version: 19.0 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: Zorblisa
Product Code: SD-101
Pharmaceutical Form: Cream
INN or Proposed INN: ALLANTOIN
CAS Number: 97-59-6
Other descriptive name: ALLANTOIN
Concentration unit: % (W/V) percent weight/volume
Concentration type: equal
Concentration number: 6-
Pharmaceutical form of the placebo: Cream
Route of administration of the placebo: Topical use (Noncurrent)
Scioderm, An Amicus Therapeutics CompanyNot RecruitingFemale: yes
Male: yes
150Phase 3Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands
52NCT02090283March 26, 201411 November 2019Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis BullosaAn Open Label Extension, Multi-Center, Study to Evaluate the Safety of SD-101 Cream in Subjects With Epidermolysis BullosaEpidermolysis BullosaDrug: SD-101 dermal cream (6%)Scioderm, Inc.Amicus Therapeutics;Food and Drug Administration (FDA)Not recruiting6 MonthsN/AAll42Phase 2United States
53EUCTR2012-000605-72-NL06/02/201410 September 2018Stem cell transplantation with cord blood and mesenchymal stem cells after reduced intensity conditioning for severe forms of the blistering disease epidermolysis bullosaUnrelated cord blood transplantation after reduced toxicity conditioning with mesenchymal stromal cell co-infusion in patients with severe epidermolysis bullosa - CB+MSCforEBThe 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
Pharmaceutical Form: Infusion
INN or Proposed INN: Mesenchymal stromal cells
Current Sponsor code: TC-MSC
Concentration unit: thousand organisms/ml thousand organisms/millilitre
Concentration type: range
Concentration number: 1000-2000
Trade Name: Busilvex
Pharmaceutical Form: Concentrate for solution for infusion
Trade Name: Fludarabine
Product Name: FLudarabine
Pharmaceutical Form: Concentrate for solution for injection/infusion
Trade Name: THYMOGLOBULINE
Product Name: Thymoglobuline
Pharmaceutical Form: Powder and solvent for solution for infusion
Universitair Medisch Centrum UtrechtNot RecruitingFemale: yes
Male: yes
11Phase 2Netherlands
54NCT02014376January 201416 December 2017Study of Effectiveness and Safety of SD-101 in Subjects With Epidermolysis BullosaA Phase 2B, Multi-center, Randomized, Double-Blind, Vehicle Controlled Study of the Effectiveness and Safety of SD-101 Cream in Subjects With Epidermolysis BullosaEpidermolysis BullosaDrug: 3% SD-101 dermal cream;Drug: 6% SD-101 dermal cream;Drug: Vehicle (0% SD-101)Scioderm, Inc.Not recruiting6 MonthsN/AAll48Phase 2United States
55EUCTR2013-002034-21-AT16/12/201313 March 2017Diacerein for butterfly childrenDiacerein for the treatment of Epidermolysis bullosa simplex - Diacerein for EBSEpidermolysis bullosa simplex - type Dowling-Meara;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Product Name: SimpleCare
Pharmaceutical Form: Cream
INN or Proposed INN: DIACEREIN
CAS Number: 13739-02-1
Concentration unit: g gram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Cream
Route of administration of the placebo: Topical use (Noncurrent)
EB House AustriaNot RecruitingFemale: yes
Male: yes
Phase 3Austria
No.TrialIDDate_
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56EUCTR2012-001815-21-ES18/01/201321 October 2013A study of the safety and efficacy of ABH001 in the treatment of patients with epidermolysis bullosa who have wounds that are not healing.A Multicenter, Prospective, Randomized, Open-label, Intra-patient Controlled Study of the Efficacy and Safety of ABH001 for the Treatment of Stalled Chronic Cutaneous Wounds Associated with Generalized Epidermolysis BullosaEpidermolysis bullosa
MedDRA version: 15.0 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: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh
Product Code: ABH001
Pharmaceutical Form: Living tissue equivalent
INN or Proposed INN: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh
Current Sponsor code: ABH001
Other descriptive name: Human fibroblast-derived dermal substitute, Dermagraft
Concentration unit: U unit(s)
Concentration type: equal
Concentration number: 1-
Shire Regenerative Medicine, Inc.Not RecruitingFemale: yes
Male: yes
25Portugal;United States;Spain;Austria;Australia;Germany
57NCT01528306March 201219 February 2015A Pilot Study of HP802-247 in Dystrophic Epidermolysis BullosaAn Exploratory, Cross-Over Study of the Safety of HP802-247 Applied to Open Wounds of Subjects With Dystrophic Epidermolysis BullosaDystrophic Epidermolysis BullosaBiological: HP802-247;Other: PlaceboHealthpointNot recruiting2 YearsN/ABoth0Phase 2United States
58NCT01538862February 201216 December 2017Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis BullosaEfficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis BullosaDystrophic Epidermolysis BullosaDrug: Granulocyte Colony Stimulating Factor (GCSF)Vanderbilt University Medical CenterNot recruitingN/AN/AAll7Phase 2United States
59NCT01340235June 201119 February 2015Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral ErythromycinTreatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral ErythromycinEpidermolysis BullosaDrug: Oral erythromycinCentre Hospitalier Universitaire de NiceRecruiting6 Months8 YearsBoth8Phase 3France
60EUCTR2010-023121-38-GB13/01/201119 March 2012A prospective placebo controlled phase II study to evaluate the use of allogeneic fibroblasts for the treatment of skin erosions in recessive dystrophic epidermolysis bulllosa. - Fibroblast cell therapy for RDEB - a phase II clinical trialA prospective placebo controlled phase II study to evaluate the use of allogeneic fibroblasts for the treatment of skin erosions in recessive dystrophic epidermolysis bulllosa. - Fibroblast cell therapy for RDEB - a phase II clinical trialRecessive dystropic epidermolysis bullosaTrade Name: ICX-RHY-013
Product Name: Cultured allogeneic fibroblasts
Product Code: ICX-RHY-013
Pharmaceutical Form: Injection
INN or Proposed INN: Fibroblasts
Current Sponsor code: ICX-DEBRHY-2
Concentration unit: ml millilitre(s)
Concentration type: equal
Pharmaceutical form of the placebo: Injection
Route of administration of the placebo: Intradermal use
Intercytex LtdAuthorisedFemale: yes
Male: yes
25Phase 2United Kingdom
No.TrialIDDate_
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61JPRN-UMIN0000044282010/11/012 April 2019Exploratory research on effectiveness and safety of Rituximab treatment for steroid intractable pemphygus, bullous pemphigoid and epidermolysis bullosa acquisita.pemphigus, bullous pemphigoid, epidermolysis bullosa acquisitaRituximabResearch group of rare intractable dermatologial disorderNot Recruiting20years-oldNot applicableMale and Female20Phase 2Japan
62NCT01263379October 5, 201030 September 2019Gene Transfer for Recessive Dystrophic Epidermolysis BullosaA Phase 1/2A Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Using the Drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)Epidermolysis Bullosa Dystrophica;Epidermolysis BullosaBiological: LZRSE-Col7A1 Engineered Autologous Epidermal SheetsStanford UniversityNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);Abeona Therapeutics, IncNot recruiting13 YearsN/AAll10Phase 1/Phase 2United States
63NCT00951964October 201019 February 2015Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate)Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate)Epidermolysis Bullosa DystrophicaDrug: Polyphenon E before Placebo;Drug: placebo before treatmentCentre Hospitalier Universitaire de NiceNot recruiting2 YearsN/ABoth18Phase 2France
64NCT01294241October 201019 February 2015Case Series Documentation of Patients With Epidermolysis BullosaOpen, Prospective, Controlled Case Series Documentation to Compare Intra-individually the Efficacy and Tolerance of Sericare® Versus Non-adhesive Wound Dressing Alone in Accelerating the Epithelialization of Skin Lesions of Patients With Epidermolysis Bullosa HereditariaEpidermolysis Bullosa HereditariaDrug: Sericare®Birken AGNot recruiting1 Year95 YearsBoth10Phase 2Germany
65EUCTR2010-019945-24-DE24/09/201019 March 2012Open, Prospective, Controlled Case series Documentation to Compare Intra-individually the Efficacy and Tolerance of Sericare versus Non-adhesive Wound Dressing alone in Accelerating the Epithelialization of Skin lesions of Patients with Epidermolysis bullosa hereditaria - EBCS-trialOpen, Prospective, Controlled Case series Documentation to Compare Intra-individually the Efficacy and Tolerance of Sericare versus Non-adhesive Wound Dressing alone in Accelerating the Epithelialization of Skin lesions of Patients with Epidermolysis bullosa hereditaria - EBCS-trialThis is an open, prospective, controlled case-series documentation to compare intra-individually the efficacy and tolerance of Sericare versus standard treatment in accelerating the epithelialization of skin lesions of patients with Epidermolysis bullosa hereditaria.Product Name: Sericare
Pharmaceutical Form: Cutaneous suspension
INN or Proposed INN: triterpene dry extract from birch cork
Current Sponsor code: TE
Concentration unit: % percent
Concentration type: equal
Concentration number: 10-
Birken GmbHNot RecruitingFemale: yes
Male: yes
Germany
No.TrialIDDate_
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66NCT01908088July 201019 February 2015Autologous Transplantation of Cultured Fibroblast on Amniotic Membrane in Patients With Epidermolysis BullosaAutologous Transplantation of Cultured Fibroblast on Amniotic Membrane for Mitten Hand Deformity in Patients With Epidermolysis BullosaEpidermolysis Bullosa With Mitten HandsBiological: Cell transplantationRoyan InstituteHazrat Fatemeh HospitalNot recruiting5 Years25 YearsBoth6Phase 1Iran, Islamic Republic of
67EUCTR2009-012750-21-FR20/01/201019 March 2012Traitement des épidermolyses bulleuses dystrophiques héréditaires par l’épigallocatéchine-3-gallate oral (Polyphenon E®)Traitement des épidermolyses bulleuses dystrophiques héréditaires par l’épigallocatéchine-3-gallate oral (Polyphenon E®)Dystrophic epidermolysis bullosa hereditaria
MedDRA version: 12.0 Level: LLT Classification code 10056508 Term: Acquired epidermolysis bullosa
Trade Name: POLYPHENON E
Product Name: POLYPHENON E
Pharmaceutical Form: Capsule*
Pharmaceutical form of the placebo: Capsule*
Route of administration of the placebo: Oral use
CHU de NICEAuthorisedFemale: yes
Male: yes
France
68NCT01033552January 20107 October 2019Biochemical Correction of Severe EB by Allo HSCT and Off-the-shelf MSCsMT2009-09: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation and Off-the-shelf Mesenchymal Stem CellsEpidermolysis BullosaDrug: 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 transplantationMasonic Cancer Center, University of MinnesotaRecruitingN/A25 YearsAll75Phase 2United States
69EUCTR2010-024428-10-FR2 October 2017Traitement des épidermolyses bulleuses simples de type Dowling Maera par l'érythromicine oraleTraitement des épidermolyses bulleuses simples de type Dowling Maera par l'érythromicine oraleDowling Maera's bullous epidermolysis is a génodermatose with autosomique dominant transmission owed to transfers of the genes coding for keratins. It results from it a cutaneous fragility very severe especially during the early childhood. Tetracyclines showed a certain efficiency in cases isolated probably by their anti-inflammatory action but cannot be used at the young child's. The érythromycine, used in the other inflammatory dermatosis, seems to be a good candidate for these patients.
MedDRA version: 12.1 Level: LLT Classification code 10056508 Term: Acquired epidermolysis bullosa
Trade Name: erythrocine
Product Name: erythrocine oral
Pharmaceutical Form: Granules for syrup
Trade Name: erythromycine
Product Name: erythrocine oral
Pharmaceutical Form: Granules for syrup
CHU de NICENot AvailableFemale: yes
Male: yes
8Phase 2France
70EUCTR2009-010763-17-SE26/05/200919 March 2012Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia congenita-a double-blind placebo-controlled phase II proof of concept studyBotulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia congenita-a double-blind placebo-controlled phase II proof of concept studyEpidermolysis Bullosa Simplex and Pachyonychia CongenitaTrade Name: Dysport
Pharmaceutical Form: Powder for solution for injection
Pharmaceutical form of the placebo: Powder for solution for injection
Route of administration of the placebo: Subcutaneous use
SophiahemmetAuthorisedFemale: yes
Male: yes
Phase 2Sweden
No.TrialIDDate_
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71NCT00881556March 200919 February 2015Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)A Pilot Study of Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (ALLOSCT) In Children With Recessive Dystrophic Epidermolysis Bullosa (RDEB)Epidermolysis BullosaDrug: Reduced Intensity Transplant conditioningColumbia UniversityNot recruitingN/A21 YearsBoth20Phase 0United States
72NCT00825565February 200916 December 2017Study of Alwextin® Cream in Treating Epidermolysis BullosaOpen-label, Pilot Study to Investigate the Safety and Tolerability of Alwextin 3.0% Cream in the Treatment of Epidermolysis BullosaEpidermolysis BullosaDrug: Alwextin creamNorthwestern UniversityAnn & Robert H Lurie Children's Hospital of ChicagoNot recruiting6 MonthsN/AAll8Phase 2United States
73NCT00478244April 200719 October 2017Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis BullosaAllogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis BullosaEpidermolysis BullosaDrug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Procedure: hematopoietic bone marrow transplantationMasonic Cancer Center, University of MinnesotaNot recruitingN/A25 YearsAll7N/AUnited States
74NCT00987142December 200626 August 2019Trial To Assess Efficacy Of A Chimeric Skin In Patients With Epidermolysys BullosaA Comparative Open-Label Multicentre Clinical Trial To Assess The Efficacy And Safety Of A New Therapy With Cultured Chimeric Skin For The Treatment Of Skin Lesions In Patients With Epidermolysis BullosaEPIDERMOLYSIS BULLOSADrug: CX501;Device: Occlusive non adherent dressingTigenix S.A.U.Not recruitingN/AN/AAll12Phase 2Spain
75NCT00380640September 200611 June 2018The Efficacy of Trimethoprim in Wound Healing of Patients With Epidermolysis BullosaThe Efficacy of Trimethoprim in Wound Healing of Patients With Epidermolysis Bullosa: A Randomized, Double Blinded, Placebo Controlled, Cross-over Pilot StudyEpidermolysis BullosaDrug: TrimethoprimThe Hospital for Sick ChildrenNot recruitingN/A20 YearsAll10Phase 2Canada
No.TrialIDDate_
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PhaseCountries
76NCT00336154June 200619 February 2015Randomised Double Blind Crossover Placebo Controlled Study to Evaluate the Efficacy of Tetracycline in Epidermolysis BullosaRandomised Double Blind Crossover Placebo Controlled Study to Evaluate the Efficacy of Tetracycline in Epidermolysis BullosaEpidermolysis BullosaDrug: tetracyclinRambam Health Care CampusNot recruiting13 Years60 YearsBoth20Phase 4Israel
77NCT00311766February 200619 October 2017A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis BullosaA Randomized, Double-Blind, Placebo-Controlled, Dose-Response Study of the Safety and Efficacy of Thymosin Beta 4 in the Treatment of Patients With Epidermolysis BullosaEpidermolysis BullosaDrug: Thymosin Beta 4;Drug: PlaceboRegeneRx Biopharmaceuticals, Inc.Not recruiting2 YearsN/AAll30Phase 2United States
78NCT00231517October 200519 February 2015Randomised Double Blind Placebo Controlled Cross Over Design of the Efficacy of Topical Morphine for Inflammatory Pain in Children With Epidermolysis BullosaEpidermylosis BullosaDrug: topical opiod;Drug: morphine sulphate in intrasite gelInstitute of Child HealthNot recruiting4 Years18 YearsBothPhase 4United Kingdom
79NCT00014729October 20007 April 2015Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis BullosaEpidermolysis BullosaDrug: isotretinoinUniversity of North CarolinaNot recruiting15 YearsN/ABoth20Phase 1
80NCT00004359February 199619 February 2015Phase II Pilot Study of Extracorporeal Phototherapy for Epidermolysis Bullosa AcquisitaEpidermolysis Bullosa AcquisitaDrug: methoxsalenNational Center for Research Resources (NCRR)Northwestern UniversityNot recruiting18 YearsN/ABoth10Phase 2

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