36. 表皮水疱症 Epidermolysis bullosa Clinical trials / Disease details


臨床試験数 : 160 薬物数 : 195 - (DrugBank : 47) / 標的遺伝子数 : 50 - 標的パスウェイ数 : 124

  
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04173650
(ClinicalTrials.gov)
April 202218/11/2019MSC EVs in Dystrophic Epidermolysis BullosaA Safety Study of the Administration of Mesenchymal Stem Cell Extracellular Vesicles in the Treatment of Dystrophic Epidermolysis Bullosa WoundsDystrophic Epidermolysis BullosaDrug: AGLE 102Aegle TherapeuticsNULLNot yet recruiting6 YearsN/AAll10Phase 1/Phase 2NULL
2EUCTR2021-000214-42-ES
(EUCTR)
08/03/202217/12/2021INM-755 cream for patients with EB.A Randomised, Double-Blind, Vehicle-Controlled Phase 2 Study of Topically Applied INM-755 (cannabinol) Cream in Patients with Epidermolysis Bullosa. Epidermolysis 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: Cannabinol (CBN)
Product Code: INM-755
INN or Proposed INN: CANNABINOL
InMed Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2France;Serbia;Greece;Spain;Austria;Israel;Germany;Italy
3NCT05111600
(ClinicalTrials.gov)
March 20221/9/2021Open-label, Pivotal Clinical Trial to Confirm Efficacy and Safety of Autologous Grafts Containing Stem Cells Genetically Modified for Epidermis Restoration in Patients With Junctional Epidermolysis BullosaMulticentre, Open-label, Uncontrolled, Pivotal Clinical Trial to Confirm the Efficacy and Safety of Autologous Fibrin-cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified for Restoration of Epidermis in Patients With Junctional Epidermolysis BullosaJunctional Epidermolysis Bullosa Non-Herlitz TypeDrug: TransplantationHolostem Terapie Avanzate s.r.l.IRCCS San Raffaele;University of Modena and Reggio EmiliaNot yet recruiting6 Months65 YearsAll6Phase 2/Phase 3France;Germany;Italy
4NCT05062070
(ClinicalTrials.gov)
November 15, 202121/9/2021Safety and Efficacy of Topical TolaSure Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa SimplexA Phase I/II, Closed Label, Randomized, Pilot Study for the Safety and Efficacy of TolaSure Gel, 5% w/w Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa Simplex (TAMES)Epidermolysis Bullosa SimplexDrug: TolaSure Topical Gel;Drug: Vehicle Topical GelBioMendics, LLCNULLNot yet recruiting18 YearsN/AAll5Phase 1United States
5EUCTR2018-000261-36-IT
(EUCTR)
07/09/202107/10/2020Gene Therapy for patient with Junctional Epidermolysis BullosaMULTICENTRE, OPEN-LABEL, UNCONTROLLED, PIVOTAL CLINICAL TRIAL TO CONFIRM THE EFFICACY AND SAFETY OF AUTOLOGOUS FIBRIN-CULTURED EPIDERMAL GRAFTS CONTAINING EPIDERMAL STEM CELLS GENETICALLY MODIFIED FOR RESTORATION OF EPIDERMIS IN PATIENTS WITH JUNCTIONAL EPIDERMOLYSIS BULLOSA (HOLOGENE 5) - Hologene 5 Inherited Epidermolysis Bullosa (EB) is a group of rare, devastating genetic disorders characterized by structural and mechanical fragility ofskin and mucosal membranes, impairing the patient's quality of life. Generalized JEB is a chronic, life-threatening condition caused bymutations in genes– encoding different chains of laminin 332. All of these mutations hamper hemidesmosome formation, causing blisters. The most frequent, and perhaps most severe, JEB is due to mutations in LAMB3.
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: Hologene 5
Product Code: [Hologene 5 DS]
Other descriptive name: 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.
HOLOSTEM TERAPIE AVANZATE S.R.L.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
6Phase 2;Phase 3France;Germany;Italy
6NCT04177498
(ClinicalTrials.gov)
August 24, 20217/11/2019Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCCA Pilot, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell CarcinomaRecessive Dystrophic Epidermolysis BullosaDrug: Rigosertib Sodium;Other: Quality-of-Life AssessmentThomas Jefferson UniversityOnconova Therapeutics, Inc.Recruiting18 Years79 YearsAll6Early Phase 1United States
7EUCTR2021-000214-42-AT
(EUCTR)
01/08/202129/04/2021INM-755 cream for patients with EB.A Randomised, Double-Blind, Vehicle-Controlled Phase 2 Study of Topically Applied INM-755 (cannabinol) Cream in Patients with Epidermolysis Bullosa. Epidermolysis 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: Cannabinol (CBN)
Product Code: INM-755
INN or Proposed INN: CANNABINOL
InMed Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2France;Serbia;Greece;Austria;Israel;Germany;Italy
8NCT04908215
(ClinicalTrials.gov)
August 202126/5/2021INM-755 (Cannabinol) Cream for Treatment of Epidermolysis BullosaA Randomised, Double-Blind, Vehicle-Controlled Phase 2 Study of Topically Applied INM-755 (Cannabinol) Cream in Patients With Epidermolysis BullosaEpidermolysis Bullosa Simplex;Epidermolysis Bullosa, Junctional;Epidermolysis Bullosa Dystrophica;Kindler SyndromeDrug: INM-755 (cannabinol) cream;Drug: Vehicle CreamInMed Pharmaceuticals Inc.NULLNot yet recruiting12 YearsN/AAll20Phase 2NULL
9EUCTR2021-000214-42-GR
(EUCTR)
22/07/202122/06/2021INM-755 cream for patients with EB.A Randomised, Double-Blind, Vehicle-Controlled Phase 2 Study of Topically Applied INM-755 (cannabinol) Cream in Patients with Epidermolysis Bullosa. Epidermolysis 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: Cannabinol (CBN)
Product Code: INM-755
INN or Proposed INN: CANNABINOL
InMed Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2France;Serbia;Greece;Austria;Israel;Germany;Italy
10NCT04917874
(ClinicalTrials.gov)
May 25, 20212/6/2021A Long-term Treatment With B-VEC for Dystrophic Epidermolysis BullosaOpen Label Treatment of Beremagene Geperpavec (B-VEC)Dystrophic Epidermolysis Bullosa;DEB - Dystrophic Epidermolysis Bullosa;Recessive Dystrophic Epidermolysis Bullosa;Dominant Dystrophic Epidermolysis BullosaBiological: Open Label Topical Beremagene Geperpavec (B-VEC)Krystal Biotech, Inc.NULLRecruiting6 MonthsN/AAll30Phase 3United States
11NCT03786237
(ClinicalTrials.gov)
April 12, 202114/11/2018Rigosertib 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 IntravenousProf. Johann BauerNULLRecruiting18 Years79 YearsAll12Phase 1/Phase 2Austria
12NCT04644627
(ClinicalTrials.gov)
December 1, 202020/11/2020Topical Gentamicin Nonsense Suppression Therapy of EBTOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONSEpidermolysis BullosaDrug: Gentamicin SulfateOslo University HospitalNULLRecruitingN/AN/AAll6Phase 1/Phase 2Norway
13NCT04599881
(ClinicalTrials.gov)
October 15, 20208/10/2020A Study of PTR-01 in Recessive Dystrophic Epidermolysis BullosaA Phase 2 Open-Label Study of PTR-01 in Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)Recessive Dystrophic Epidermolysis BullosaDrug: PTR-01Phoenix Tissue Repair, Inc.NULLCompleted12 YearsN/AAll6Phase 2United States
14EUCTR2018-003890-91-PL
(EUCTR)
29/09/202013/11/2018Treatment 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 WarsawNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 1;Phase 2Poland
15EUCTR2020-002337-15-NO
(EUCTR)
16/09/202029/06/2020TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSATOPICAL 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 HospitalNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
6Phase 2Norway
16NCT04491604
(ClinicalTrials.gov)
August 17, 202022/7/2020The Objective of This Study is to Compare the Efficacy and Safety of Beremagene Geperpavec (B-VEC) Topical Gel With That of Placebo for the Treatment of Dystrophic Epidermolysis Bullosa (DEB).A Phase III Double Blinded, Placebo-Controlled, Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, Previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)Dystrophic Epidermolysis Bullosa;Recessive Dystrophic Epidermolysis Bullosa;Dominant Dystrophic Epidermolysis BullosaBiological: Topical Beremagene Geperpavec;Other: PlaceboKrystal Biotech, Inc.NULLActive, not recruiting6 MonthsN/AAll31Phase 3United States
17NCT04613102
(ClinicalTrials.gov)
August 1, 202027/10/2020The Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III TrialThe Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III TrialEpidermolysis Bullosa;Pain;ItchDrug: AVCN583601 (3% Cannabidiol cream)Elena PopeAvicanna IncWithdrawn4 Years50 YearsAll0Phase 2/Phase 3Canada
18EUCTR2020-001542-19-FR
(EUCTR)
22/07/202003/04/2020Comparing the Efficacy and Safety of Ixekizumab to Placebo in Patients > 6 years of age with EB simplex generalized severeA 12-Week Multicentre, Randomized, Blinded, Parallel-Group Study Comparing the Efficacy and Safety of Ixekizumab to Placebo in Patients > 6 years of age with EB simplex generalized severe - EB Epidermolysis bullosa simplex
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]
Trade Name: Ixekizumab
Product Name: Taltz 80mg
Product Code: 3400930060797
CHU NICENULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
0Phase 2France
19NCT04213261
(ClinicalTrials.gov)
June 9, 20202/12/2019A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis BullosaA Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis BullosaRecessive Dystrophic Epidermolysis BullosaBiological: FCX-007 (dabocemagene autoficel; see below for FCX-007 description)Castle Creek Biosciences, LLC.NULLRecruiting2 YearsN/AAll24Phase 3United States
20NCT04227106
(ClinicalTrials.gov)
January 10, 202010/1/2020Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)VIITAL: A Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)Epidermolysis Bullosa;Recessive Dystrophic Epidermolysis BullosaBiological: EB-101Abeona Therapeutics, IncNULLRecruiting6 YearsN/AAll15Phase 3United States
21NCT04186650
(ClinicalTrials.gov)
January 10, 202025/11/2019Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent GraftsPhase I/II ex Vivo Gene Therapy Clinical Trial for RDEB Using Autologous Skin Equivalent Grafts Genetically Corrected With a COL7A1-encoding SIN Retroviral VectorEpidermolysis Bullosa Dystrophica, RecessiveBiological: COL7A1-SIN retroviral vector engineered autologous tissue-engineered skinInstitut National de la Santé Et de la Recherche Médicale, FranceNULLActive, not recruiting18 YearsN/AAll3Phase 1/Phase 2France
22NCT04171661
(ClinicalTrials.gov)
December 10, 201919/11/2019Self-Assembled Skin Substitute for the Treatment of Epidermolysis BullosaSASS 3: Self-Assembled Skin Substitute (SASS) for the Treatment of Epidermolysis BullosaEpidermolysis Bullosa DystrophicaBiological: SASSCHU de Quebec-Universite LavalThe Hospital for Sick ChildrenActive, not recruitingN/AN/AAll1Early Phase 1Canada
23NCT04140786
(ClinicalTrials.gov)
October 31, 201923/10/2019Optimizing 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 CaliforniaNULLRecruitingN/AN/AAll6Phase 1/Phase 2United States
24EUCTR2018-001009-98-IT
(EUCTR)
11/10/201910/11/2020Study 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). - allo-APZ2-EB-II-01 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]
Other descriptive name: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells
RHEACELL GmbH & Co. KGNULLNot RecruitingFemale: yes
Male: yes
18Phase 1United States;France;Austria;Germany;United Kingdom;Italy
25NCT03928093
(ClinicalTrials.gov)
August 7, 201927/3/2019Pregabalin 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
26NCT03578029
(ClinicalTrials.gov)
May 22, 201914/6/2018Evaluation 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, LLCNULLRecruiting4 YearsN/AAll15Phase 2United States
27NCT03836001
(ClinicalTrials.gov)
April 18, 20197/2/2019A 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
28EUCTR2018-001009-98-DE
(EUCTR)
01/03/201903/05/2018Study 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
Other descriptive name: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells
RHEACELL GmbH & Co. KGNULLNot RecruitingFemale: yes
Male: yes
16Phase 1;Phase 2United States;France;Austria;Germany;United Kingdom;Italy
29EUCTR2016-002790-35-FR
(EUCTR)
07/02/201917/01/2018 Phase I/II ex vivo gene therapy clinical trial for RDEB using autologous skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector - EBGraft Phase I/II ex vivo gene therapy clinical trial for RDEB using autologous skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector - EBGraft The trial aims to treat the recessive dystrophic epidermolysis bullosa (RDEB) by grafting one to three subjects with RDEB with autologous COL7A1-modified skin equivalents, using SIN-RV encoding COL7A1 cDNA.
MedDRA version: 20.0;Level: LLT;Classification code 10074980;Term: Epidermolysis bullosa aquisita;System Organ Class: 100000004858 ;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
Product Name: SIN RV-mediated COL7A1 corrected autologous skin equivalentINSERMNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
3 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance
30NCT03529877
(ClinicalTrials.gov)
January 16, 201924/4/2018Allogeneic 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 GmbHActive, not recruitingN/A55 YearsAll16Phase 1/Phase 2United States;Austria;France;Germany;United Kingdom;Italy
31NCT03752905
(ClinicalTrials.gov)
January 9, 201919/11/2018A 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.NULLCompleted16 YearsN/AAll12Phase 1/Phase 2United States
32EUCTR2018-001009-98-FR
(EUCTR)
07/01/201930/10/2018Study 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
Other descriptive name: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells
RHEACELL GmbH & Co. KGNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
18 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;France;Austria;Germany;United Kingdom
33EUCTR2018-001009-98-AT
(EUCTR)
05/11/201824/05/2018Study 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
Other descriptive name: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells
RHEACELL GmbH & Co. KGNULLNot RecruitingFemale: yes
Male: yes
16Phase 1;Phase 2United States;France;Austria;Germany;United Kingdom;Italy
34NCT03468322
(ClinicalTrials.gov)
October 20, 20188/3/2018A 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.NULLCompleted2 YearsN/AAll9Phase 2Taiwan
35NCT03632265
(ClinicalTrials.gov)
October 1, 201817/7/2018Study 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 yet recruiting6 YearsN/AAll15Phase 3NULL
36EUCTR2017-004806-17-DE
(EUCTR)
27/09/201808/02/2018Study 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 - WINGS Recessive 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
INN or Proposed INN: unavailable
Wings Therapeutics Inc.NULLNot Recruiting Female: yes
Male: yes
8 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Czech Republic;Spain;Germany;United Kingdom
37EUCTR2018-001009-98-GB
(EUCTR)
18/09/201825/10/2018Study 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
Other descriptive name: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells
RHEACELL GmbH & Co. KGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 1;Phase 2United States;France;Austria;Germany;Italy;United Kingdom
38EUCTR2017-004806-17-ES
(EUCTR)
06/09/201811/04/2018Study 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 - WINGS Recessive 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
INN or Proposed INN: unavailable
ProQR TheraputicsNULLNot RecruitingFemale: yes
Male: yes
8Phase 1;Phase 2France;United States;Czech Republic;Canada;Spain;Austria;Germany;United Kingdom
39NCT03392909
(ClinicalTrials.gov)
July 5, 20182/1/2018Intravenous 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 CaliforniaNULLRecruiting7 YearsN/AAll9Phase 1/Phase 2United States
40NCT03605069
(ClinicalTrials.gov)
July 2, 201825/6/2018A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in 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 DDEB or RDEB Due to Mutation(s) in Exon 73 of the COL7A1 GeneEpidermolysis Bullosa Dystrophica, Recessive;Epidermolysis Bullosa Dystrophica, DominantDrug: QR-313;Drug: PlaceboPhoenicis TherapeuticsNULLTerminated4 YearsN/AAll2Phase 1/Phase 2United States;France;Spain
41NCT03453632
(ClinicalTrials.gov)
June 14, 20186/2/2018Injections 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, ToulouseNULLRecruiting18 YearsN/AAll25Phase 2/Phase 3France
42EUCTR2018-000439-29-GB
(EUCTR)
04/06/201822/03/2018A 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
INN or Proposed INN: DIACEREIN
Castle Creek Pharmaceuticals, LLCNULLNot RecruitingFemale: yes
Male: yes
4Phase 1United States;France;Netherlands;Germany;United Kingdom
43NCT03526159
(ClinicalTrials.gov)
June 1, 201817/4/2018Gentamicin 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 CaliforniaNULLRecruitingN/AN/AAll6Phase 1/Phase 2United States
44NCT03472287
(ClinicalTrials.gov)
May 18, 201814/3/2018To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use 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);Epidermolysis Bullosa Simplex;Dystrophic Epidermolysis Bullosa;Junctional Epidermolysis BullosaDrug: Diacerein 1% OintmentCastle Creek Pharmaceuticals, LLCNULLCompleted4 YearsN/AAll11Phase 1United States;France;Netherlands;United Kingdom
45EUCTR2017-004806-17-GB
(EUCTR)
18/05/201824/01/2018Study 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 - WINGS Recessive 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
INN or Proposed INN: unavailable
Wings Therapeutics Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
8 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Czech Republic;Spain;Germany;United Kingdom
46NCT04153630
(ClinicalTrials.gov)
May 17, 20186/8/2019Safety 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 rarasActive, not recruiting12 Months18 YearsAll9Phase 1/Phase 2Spain
47NCT03536143
(ClinicalTrials.gov)
May 7, 201820/4/2018Topical Beremagene Geperpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis BullosaA Phase II Study of Beremagene Geperpavec (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 beremagene geperpavecKrystal Biotech, Inc.NULLActive, not recruiting5 YearsN/AAll4Phase 2United States
48EUCTR2017-003757-41-DE
(EUCTR)
19/04/201821/02/2018An 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
INN or Proposed INN: DIACEREIN
Other descriptive name: Diacerein
Castle Creek Pharmaceuticals, LLCNULLNot RecruitingFemale: yes
Male: yes
80Phase 2United States;France;Austria;Australia;Israel;Netherlands;Germany;United Kingdom;Italy
49EUCTR2017-003757-41-NL
(EUCTR)
11/04/201806/12/2017An 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
INN or Proposed INN: DIACEREIN
Other descriptive name: Diacerein
Castle Creek Pharmaceuticals, LLCNULLNot RecruitingFemale: yes
Male: yes
80Phase 2United States;France;Austria;Australia;Israel;Germany;Netherlands;United Kingdom;Italy
50EUCTR2016-002066-32-DK
(EUCTR)
05/04/201817/01/2018A 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLNot RecruitingFemale: yes
Male: yes
250Phase 3Serbia;Hong Kong;Greece;Spain;Ukraine;Ireland;Chile;Israel;Russian Federation;Colombia;Italy;Switzerland;France;Australia;Denmark;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Brazil;Belgium;Singapore;Croatia;Romania;Germany
51EUCTR2016-002066-32-BE
(EUCTR)
23/03/201817/01/2018A 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLNot RecruitingFemale: yes
Male: yes
192Phase 3Serbia;Hong Kong;Greece;Spain;Ukraine;Ireland;Chile;Israel;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Brazil;Belgium;Singapore;Croatia;Romania;Germany
52EUCTR2016-002066-32-HR
(EUCTR)
26/02/201812/04/2018A 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
192Phase 3Serbia;Hong Kong;Greece;Spain;Ukraine;Ireland;Chile;Israel;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Austria;United Kingdom;Czech Republic;Hungary;Mexico;Argentina;Brazil;Belgium;Singapore;Croatia;Romania;Germany
53EUCTR2017-003757-41-GB
(EUCTR)
19/02/201813/11/2017An 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
INN or Proposed INN: DIACEREIN
Other descriptive name: Diacerein
Castle Creek Pharmaceuticals, LLCNULLNot RecruitingFemale: yes
Male: yes
80Phase 2United States;France;Austria;Australia;Israel;Netherlands;Germany;Italy;United Kingdom
54EUCTR2017-003757-41-FR
(EUCTR)
07/02/201817/10/2018An 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
INN or Proposed INN: DIACEREIN
Other descriptive name: Diacerein
Castle Creek Pharmaceuticals, LLCNULLNot RecruitingFemale: yes
Male: yes
80Phase 2United States;France;Austria;Australia;Israel;Netherlands;Germany;United Kingdom
55EUCTR2016-002066-32-HU
(EUCTR)
08/01/201829/11/2017A 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLNot RecruitingFemale: yes
Male: yes
164Phase 3Hong Kong;Greece;Spain;Ireland;Austria;Israel;Chile;Colombia;Italy;Switzerland;United Kingdom;France;Hungary;Czech Republic;Mexico;Argentina;Belgium;Brazil;Singapore;Romania;Croatia;Denmark;Australia;Germany
56EUCTR2017-003757-41-AT
(EUCTR)
04/01/201829/11/2017An 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
INN or Proposed INN: DIACEREIN
Other descriptive name: Diacerein
Castle Creek Pharmaceuticals, LLCNULLNot RecruitingFemale: yes
Male: yes
84Phase 2France;United States;Australia;Austria;Israel;Netherlands;Germany;Italy;United Kingdom
57EUCTR2016-002066-32-IE
(EUCTR)
22/12/201707/03/2017A 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLNot RecruitingFemale: yes
Male: yes
250Phase 3Serbia;Greece;Spain;Ukraine;Ireland;Turkey;Austria;Israel;Russian Federation;United Kingdom;Switzerland;Italy;France;Czech Republic;Hungary;Mexico;Belgium;Croatia;Denmark;Australia;Germany
58EUCTR2016-003832-19-AT
(EUCTR)
11/12/201709/08/2017Clinical 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 AustriaNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 2Austria
59NCT03389308
(ClinicalTrials.gov)
December 1, 201719/12/2017Long 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;Epidermolysis Bullosa SimplexDrug: diacerein 1% ointmentCastle Creek Pharmaceuticals, LLCNULLCompleted6 Months99 YearsAll51Phase 2United States
60EUCTR2017-000606-37-ES
(EUCTR)
16/11/201718/09/2017Safety 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
INN or Proposed INN: Células madre mesenquimales haploidénticas derivadas de médula ósea
Other descriptive name: MESENCHYMAL STEM CELLS (MSCS)
Fundación para la Investigación Biomédica Hospital Universitario La PazNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
9Phase 1Spain
61EUCTR2016-002066-32-GB
(EUCTR)
10/11/201713/02/2017A 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
192 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSerbia;Greece;Spain;Ukraine;Ireland;Turkey;Austria;Russian Federation;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Hungary;Mexico;Belgium;Croatia;Denmark;Australia;Germany
62EUCTR2016-002066-32-IT
(EUCTR)
18/10/201730/01/2018A 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
164 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Greece;Spain;Ireland;Turkey;Austria;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Hungary;Mexico;Belgium;Croatia;Denmark;Australia;Germany
63EUCTR2016-002066-32-GR
(EUCTR)
16/10/201705/05/2017A Clinical Trial which compare the safety and efficacy of a wound gel, the study treatment, or a sunflower oil-based vehicle (placebo) 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
250 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSerbia;Hong Kong;Greece;Spain;Ukraine;Ireland;Chile;Israel;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Brazil;Belgium;Singapore;Croatia;Romania;Germany
64EUCTR2016-004427-24-AT
(EUCTR)
10/10/201730/05/2017An 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
INN or Proposed INN: DIACEREIN
Other descriptive name: Diacerein
Castle Creek Pharmaceuticals, LLCNULLNot Recruiting Female: yes
Male: yes
80 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Australia;Austria;Israel;Netherlands;Germany;Italy;United Kingdom
65EUCTR2015-003670-32-AT
(EUCTR)
05/10/201707/08/2017Phase I/II trial to establish safety, tolerability and efficacy of losartan in children with epidermolysis bullosaA dual-center prospective phase I/II trial to establish safety, tolerability and to obtain first data on efficacy of losartan in children with recessive dystrophic epidermolysis bullosa (RDEB) - REFLECT 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]
Trade Name: Losartan HEXAL
INN or Proposed INN: losartan potassium
Other descriptive name: LOSARTAN POTASSIUM
Medical Center - University of FreiburgNULLNot RecruitingFemale: yes
Male: yes
30Phase 2Austria;Germany
66EUCTR2016-002066-32-AT
(EUCTR)
26/09/201708/03/2017A 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLNot RecruitingFemale: yes
Male: yes
250Phase 3Serbia;Hong Kong;Greece;Spain;Ukraine;Ireland;Chile;Israel;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Brazil;Belgium;Singapore;Croatia;Romania;Germany
67EUCTR2016-004427-24-NL
(EUCTR)
06/09/201723/06/2017An 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
INN or Proposed INN: DIACEREIN
Other descriptive name: Diacerein
Castle Creek Pharmaceuticals, LLCNULLNot Recruiting Female: yes
Male: yes
80 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Austria;Australia;Israel;Germany;Netherlands;Italy;United Kingdom
68EUCTR2016-004427-24-DE
(EUCTR)
24/08/201723/05/2017An 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
INN or Proposed INN: DIACEREIN
Other descriptive name: Diacerein
Castle Creek Pharmaceuticals, LLCNULLNot Recruiting Female: yes
Male: yes
80 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Austria;Australia;Israel;Netherlands;Germany;Italy;United Kingdom
69EUCTR2016-000095-17-AT
(EUCTR)
17/08/201707/06/2017Gene 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 17 JEB 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
INN or Proposed INN: Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL17A1-encoding retroviral vector
Holostem Terapie Avanzate s.r.l.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 1;Phase 2Austria
70EUCTR2016-004427-24-GB
(EUCTR)
21/07/201706/07/2017An 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
INN or Proposed INN: DIACEREIN
Other descriptive name: Diacerein
Castle Creek Pharmaceuticals, LLCNULLNot RecruitingFemale: yes
Male: yes
80Phase 2France;United States;Austria;Australia;Israel;Netherlands;Germany;Italy;United Kingdom
71NCT03183934
(ClinicalTrials.gov)
July 20178/6/2017A 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.NULLNot yet recruiting2 Years60 YearsAll5Phase 1/2Korea, Republic of
72JPRN-UMIN000028366
2017/06/2125/07/2017A clinical study to evaluate efficacy, safety and tolerability of ISN001 in dystrophic epidermolysis bullosa patients.A clinical study to evaluate efficacy, safety and tolerability of ISN001 in dystrophic epidermolysis bullosa patients. - A clinical study to evaluate efficacy, safety and tolerability of ISN001 in dystrophic epidermolysis bullosa patients. dystrophic epidermolysis bullosaISN001, the sheet containing allogenic adipose derived mesenchymal stromal cells (1000000/sheet), is applied to the lesion(s) once a week up to 8 timesIshin Pharmaceutical Co. Ltd.NULLComplete: follow-up complete6years-old70years-oldMale and Female5Phase 1,2Japan
73NCT03154333
(ClinicalTrials.gov)
June 1, 201710/5/2017Safety and Efficacy of Diacerein 1% Ointment 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 SimplexEpidermolysis Bullosa SimplexDrug: diacerein 1% ointment;Drug: A placebo ointmentCastle Creek Pharmaceuticals, LLCNULLTerminated4 YearsN/AAll54Phase 2United States;Australia;Austria;France;Germany;Israel;Netherlands;United Kingdom
74NCT03068780
(ClinicalTrials.gov)
March 29, 201727/2/2017Phase 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 LimitedNULLActive, not recruitingN/AN/AAll223Phase 3United States;Argentina;Australia;Austria;Brazil;Chile;Colombia;Czechia;Denmark;France;Georgia;Germany;Greece;Hong Kong;Hungary;Ireland;Israel;Italy;Romania;Russian Federation;Serbia;Singapore;Spain;Switzerland;Ukraine;United Kingdom;Belgium;Croatia
75EUCTR2016-002066-32-ES
(EUCTR)
16/03/201710/03/2017A 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 study Inherited 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
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
164Phase 3United States;Greece;Spain;Ireland;Turkey;Austria;Israel;Italy;Switzerland;United Kingdom;France;Mexico;Croatia;Australia;Germany
76EUCTR2014-005679-96-LT
(EUCTR)
06/03/201709/02/2017An 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 Bullosa Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, INC, An Amicus Therapeutics CompanyNULLNot RecruitingFemale: yes
Male: yes
150Phase 3Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands
77NCT03730584
(ClinicalTrials.gov)
February 27, 20172/11/2018Evaluation 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 ParisNULLCompletedN/A21 YearsAll10N/AFrance
78NCT03012191
(ClinicalTrials.gov)
February 2, 20174/1/2017Gentamicin for RDEBGentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa Patients With Nonsense MutationsRecessive Dystrophic Epidermolysis BullosaDrug: Gentamicin SulfateUniversity of Southern CaliforniaNULLCompletedN/AN/AAll6Phase 1/Phase 2United States
79NCT02984085
(ClinicalTrials.gov)
January 30, 20172/12/2016Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With 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.Recessive Dystrophic Epidermolysis BullosaDrug: Genetically corrected cultured epidermal autograft (ATMP)Holostem Terapie Avanzate s.r.l.Paracelsus Medical UniversityTerminated6 Years54 YearsAll3Phase 1/Phase 2Austria
80NCT03942250
(ClinicalTrials.gov)
January 201722/2/2018Uses 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 AuthorityNULLCompleted12 Years45 YearsAll8N/AEgypt
81EUCTR2014-002288-14-LT
(EUCTR)
07/12/201608/11/2016An 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 Study Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, An Amicus Therapeutics CompanyNULLNot RecruitingFemale: yes
Male: yes
150Phase 3United States;Serbia;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Belgium;Poland;Romania;Australia;Germany;Netherlands
82EUCTR2014-005679-96-ES
(EUCTR)
14/11/201609/09/2016An 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 Bullosa Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, INCNULLNot RecruitingFemale: yes
Male: yes
130Phase 3France;United States;Poland;Spain;Lithuania;Austria;Netherlands;Germany;Italy;United Kingdom
83NCT04520022
(ClinicalTrials.gov)
October 13, 201613/8/2020Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEBSingle Center, Single Group Assignment, Open Label Trial to Assess Safety and Effectiveness of Intravenous Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With Recessive Dystrophic Epidermolysis BullosaRecessive Dystrophic Epidermolysis BullosaDrug: Human Umbilical Cord Blood-derived Mesenchymal Stem CellsGangnam Severance HospitalDaewoong Pharmaceutical Co. LTD.Completed10 Years60 YearsAll5Phase 1/Phase 2Korea, Republic of
84EUCTR2014-002288-14-ES
(EUCTR)
19/09/201605/08/2016An 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 Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, INC.NULLNot RecruitingFemale: yes
Male: yes
90Phase 3France;United States;Belgium;Poland;Spain;Lithuania;Austria;Germany;Netherlands;Italy;United Kingdom
85NCT02654483
(ClinicalTrials.gov)
August 31, 201611/1/2016Neurokinin-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;PruritusDrug: VPD-737;Other: PlaceboJean Yuh TangEpidermolysis Bullosa Research Partnership;Menlo Therapeutics Inc.Completed13 YearsN/AAll14Phase 2United States
86NCT02793960
(ClinicalTrials.gov)
August 10, 201618/5/2016Topical 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, LLCCompleted12 YearsN/AAll11Phase 1United States
87EUCTR2016-001967-35-AT
(EUCTR)
19/07/201628/06/2016A 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 DEB Subjects 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
INN or Proposed INN: CALCIPOTRIOL
Universitätsklinik für Dermatologie, Paracelsus medizinische Privatuniversität SalzburgNULLNot RecruitingFemale: yes
Male: yes
15Phase 2Austria
88EUCTR2015-004592-74-AT
(EUCTR)
01/06/201619/01/2016Gene 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. - HOLOGENE7 RDEB 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
INN or Proposed INN: Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL7A1-encoding retroviral vector
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.
Holostem Terapie Avanzate s.r.l.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 1;Phase 2Austria
89EUCTR2014-004884-19-GB
(EUCTR)
24/05/201618/02/2015Gene 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-F Recessive 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
King's College LondonGuy's and St Thomas' NHS Foundation TrustNot RecruitingFemale: yes
Male: yes
10Phase 1United Kingdom
90NCT03016715
(ClinicalTrials.gov)
May 20169/1/2017Using 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, AustraliaNULLRecruiting5 YearsN/AAll8Phase 2Australia
91NCT02960997
(ClinicalTrials.gov)
May 201615/6/2016Using 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 UniversityNULLActive, not recruiting4 YearsN/AAll8Phase 2United States
92NCT02582775
(ClinicalTrials.gov)
March 201616/10/2015MT2015-20: 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 MinnesotaNULLRecruitingN/A25 YearsAll84Phase 2United States
93NCT02698735
(ClinicalTrials.gov)
February 25, 201619/2/2016Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation PatientsRecessive Dystrophic Epidermolysis BullosaDrug: Gentamicin;Drug: PlaceboUniversity of Southern CaliforniaNULLCompletedN/AN/AAll5Phase 1/Phase 2NULL
94EUCTR2014-005679-96-NL
(EUCTR)
07/09/201502/03/2015An 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 Bullosa 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: Zorblisa
Product Code: SD-101
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, INCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
130France;United States;Poland;Spain;Austria;Australia;Germany;Netherlands;Italy;United Kingdom
95NCT02592954
(ClinicalTrials.gov)
September 201529/10/2015Effect 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 UniversityNULLCompleted18 YearsN/AAll5Phase 1United States
96NCT02493816
(ClinicalTrials.gov)
September 201521/5/2015Safety 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, LondonCompleted17 YearsN/AAll5Phase 1United Kingdom
97EUCTR2014-002288-14-PL
(EUCTR)
10/08/201511/06/2015An 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 Study Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, An Amicus Therapeutics CompanyNULLNot RecruitingFemale: yes
Male: yes
150Phase 3Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Belgium;Poland;Romania;Australia;Germany;Netherlands
98NCT02670330
(ClinicalTrials.gov)
June 9, 201528/7/2015Open 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 TherapeuticsTerminated1 MonthN/AAll152Phase 3United States;Australia;Austria;France;Germany;Israel;Lithuania;Netherlands;Poland;Serbia;Spain;United Kingdom;Italy
99NCT02470689
(ClinicalTrials.gov)
June 201527/5/2015Diacerin for the Treatment of Epidermolysis Bullosa SimplexDiacerin for the Treatment of Epidermolysis Bullosa SimplexEpidermolysis Bullosa SimplexDrug: Diacerin creamTel-Aviv Sourasky Medical CenterNULLNot yet recruiting6 Years19 YearsBoth50Phase 2NULL
100NCT02323789
(ClinicalTrials.gov)
June 201518/12/2014Mesenchymal 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 LondonNULLActive, not recruiting18 Years65 YearsAll10Phase 1/Phase 2United Kingdom
101EUCTR2014-005679-96-DE
(EUCTR)
22/05/201511/03/2015An 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 Bullosa Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, INC, An Amicus Therapeutics CompanyNULLNot Recruiting Female: yes
Male: yes
150 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSerbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands
102EUCTR2014-005679-96-GB
(EUCTR)
22/04/201504/03/2015An 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 Bullosa 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: Zorblisa
Product Code: SD-101
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, INCNULLNot RecruitingFemale: yes
Male: yes
130Phase 3France;United States;Poland;Spain;Lithuania;Austria;Australia;Netherlands;Germany;Italy;United Kingdom
103EUCTR2014-002288-14-DE
(EUCTR)
31/03/201519/12/2014An 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 Study Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, An Amicus Therapeutics CompanyNULLNot RecruitingFemale: yes
Male: yes
150Phase 3Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Belgium;Poland;Romania;Australia;Germany;Netherlands
104EUCTR2014-005679-96-AT
(EUCTR)
16/03/201512/02/2015An 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 Bullosa Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, INCNULLNot RecruitingFemale: yes
Male: yes
150Phase 3Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands
105NCT02384460
(ClinicalTrials.gov)
March 11, 201513/2/2015ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants 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 BullosaEpidermolysis BullosaDrug: SD-101-6.0 cream;Drug: Placebo (SD-101-0.0) creamScioderm, Inc.Amicus TherapeuticsCompleted1 MonthN/AAll169Phase 3United States;Australia;Austria;France;Germany;Israel;Italy;Lithuania;Netherlands;Poland;Serbia;Spain;United Kingdom
106EUCTR2014-004500-30-GB
(EUCTR)
13/02/201522/12/2014A 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 bullosa A phase I/II study evaluating allogeneic mesenchymal stromal cells in adults with recessive dystrophic epidermolysis bullosa - ADSTEM Recessive dystrophic epidermolysis bullosa;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Product Name: Allogeneic mesenchymal stromal cells
Product Code: TC-MSC
King's College LondonGuy's and St Thomas NHS Foundation TrustNot Recruiting Female: yes
Male: yes
10 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
107EUCTR2014-002288-14-NL
(EUCTR)
23/12/201403/07/2014An 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 Study 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]
Product Name: Zorblisa
Product Code: SD-101
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, An Amicus Therapeutics CompanyNULLNot RecruitingFemale: yes
Male: yes
150Phase 3Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Belgium;Poland;Romania;Australia;Netherlands;Germany
108EUCTR2014-002288-14-IT
(EUCTR)
18/12/201415/07/2014An 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 Epidermolysis Bullosa
MedDRA version: 17.0;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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, INC.NULLNot RecruitingFemale: yes
Male: yes
90Phase 3France;United States;Belgium;Spain;Poland;Lithuania;Austria;Germany;Netherlands;United Kingdom;Italy
109EUCTR2014-002288-14-GB
(EUCTR)
26/09/201411/07/2014An 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 Study 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]
Product Name: Zorblisa
Product Code: SD-101
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, An Amicus Therapeutics CompanyNULLNot Recruiting Female: yes
Male: yes
150 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Serbia;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Belgium;Poland;Romania;Australia;Germany;Netherlands
110EUCTR2014-002288-14-AT
(EUCTR)
08/08/201402/07/2014An 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 Study Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, An Amicus Therapeutics CompanyNULLNot RecruitingFemale: yes
Male: yes
150Phase 3Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands
111NCT02090283
(ClinicalTrials.gov)
March 26, 201412/3/2014Open-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)Terminated6 MonthsN/AAll42Phase 2United States
112EUCTR2012-000605-72-NL
(EUCTR)
06/02/201412/08/2013Stem 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+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 UtrechtNULLNot RecruitingFemale: yes
Male: yes
11Phase 2Netherlands
113NCT02014376
(ClinicalTrials.gov)
January 6, 201412/12/2013Study of Effectiveness and Safety of SD-101 in Participants 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: SD-101 dermal cream (3%);Drug: SD-101 Dermal Cream (6%);Drug: Vehicle (SD-101 0%)Scioderm, Inc.Amicus TherapeuticsCompleted6 MonthsN/AAll48Phase 2United States
114EUCTR2013-002034-21-AT
(EUCTR)
16/12/201301/08/2013Diacerein for butterfly childrenDiacerein for the treatment of Epidermolysis bullosa simplex - Diacerein for EBS Epidermolysis bullosa simplex - type Dowling-Meara;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Product Name: SimpleCare
INN or Proposed INN: DIACEREIN
EB House AustriaNULLNot RecruitingFemale: yes
Male: yes
Phase 3Austria
115EUCTR2012-001815-21-PT
(EUCTR)
07/06/201328/03/2013A 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 Bullosa Epidermolysis bullosa
MedDRA version: 14.1;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: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh
Product Code: ABH001
INN or Proposed INN: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh
Other descriptive name: Human fibroblast-derived dermal substitute, Dermagraft
Shire Regenerative Medicine, Inc.NULLNot RecruitingFemale: yes
Male: yes
26France;United States;Portugal;Canada;Spain;Poland;Austria;Germany
116EUCTR2012-001815-21-DE
(EUCTR)
22/05/201301/11/2012A 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 Bullosa Epidermolysis bullosa
MedDRA version: 14.1;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: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh
Product Code: ABH001
INN or Proposed INN: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh
Other descriptive name: Human fibroblast-derived dermal substitute, Dermagraft
Shire Regenerative Medicine, Inc.NULLNot RecruitingFemale: yes
Male: yes
26Portugal;United States;Spain;Austria;Australia;Germany
117EUCTR2012-001815-21-AT
(EUCTR)
24/01/201325/01/2013A 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 Bullosa Epidermolysis bullosa
MedDRA version: 16.0;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
INN or Proposed INN: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh
Other descriptive name: Human fibroblast-derived dermal substitute, Dermagraft
Shire Regenerative Medicine, Inc.NULLNot RecruitingFemale: yes
Male: yes
26United States;Portugal;France;Canada;Spain;Poland;Austria;Germany
118EUCTR2012-001815-21-ES
(EUCTR)
18/01/201326/09/2012A 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 Bullosa Epidermolysis 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
INN or Proposed INN: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh
Other descriptive name: Human fibroblast-derived dermal substitute, Dermagraft
Shire Regenerative Medicine, Inc.NULLNot RecruitingFemale: yes
Male: yes
25Portugal;United States;Spain;Austria;Australia;Germany
119NCT01749306
(ClinicalTrials.gov)
December 201211/12/2012A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not HealingA Multicenter, Prospective, Randomized, Open-label, Intra-subject Controlled Study of the Efficacy and Safety of ABH001 for the Treatment of Stalled Chronic Cutaneous Wounds Associated With Generalized Epidermolysis BullosaEpidermolysis BullosaBiological: ABH001;Other: Control wound treatmentShire Regenerative Medicine, Inc.NULLTerminatedN/AN/ABoth1Phase 3United States;Austria;Canada;France;Germany;Poland;Portugal;Spain
120EUCTR2012-001394-87-GB
(EUCTR)
16/11/201222/08/2012A prospective phase I/II study to evaluate allogeneic mesenchymal stromal cells for the treatment of skin disease in children with recessive dystrophic epidermolysis bullosa. A prospective phase I/II study to evaluate allogeneic mesenchymal stromal cells for the treatment of skin disease in children with recessive dystrophic epidermolysis bullosa. - EBSTEM Recessive Dystrophic Epidermolysis Bullosa
MedDRA version: 17.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
Product Name: Allogenic mesenchymal stromal cells
Product Code: TC-MSC
King's College LondonNULLNot RecruitingFemale: yes
Male: yes
10Phase 1;Phase 2United Kingdom
121NCT01528306
(ClinicalTrials.gov)
March 201231/1/2012A 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: PlaceboHealthpointNULLWithdrawn2 YearsN/ABoth0Phase 2United States
122NCT01538862
(ClinicalTrials.gov)
February 201220/2/2012Efficacy 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 CenterNULLCompletedN/AN/AAll7Phase 2United States
123NCT01340235
(ClinicalTrials.gov)
June 201111/4/2011Treatment 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 NiceNULLRecruiting6 Months8 YearsBoth8Phase 3France
124EUCTR2010-023121-38-GB
(EUCTR)
13/01/201126/11/2010A 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 trial Recessive dystropic epidermolysis bullosaTrade Name: ICX-RHY-013
Product Name: Cultured allogeneic fibroblasts
Product Code: ICX-RHY-013
INN or Proposed INN: Fibroblasts
Intercytex LtdNULLNot RecruitingFemale: yes
Male: yes
25Phase 2United Kingdom
125JPRN-UMIN000004428
2010/11/0101/11/2010Exploratory 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 disorderNULLComplete: follow-up complete20years-oldNot applicableMale and Female20Phase 2Japan
126NCT01263379
(ClinicalTrials.gov)
October 5, 201015/12/2010Gene 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, IncActive, not recruiting13 YearsN/AAll10Phase 1/Phase 2United States
127NCT01294241
(ClinicalTrials.gov)
October 201027/10/2010Case 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 AGNULLCompleted1 Year95 YearsBoth10Phase 2Germany
128NCT00951964
(ClinicalTrials.gov)
October 20103/8/2009Treatment 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 NiceNULLCompleted2 YearsN/ABoth18Phase 2France
129EUCTR2010-019945-24-DE
(EUCTR)
24/09/201013/07/2010Open, 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-trial This 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
INN or Proposed INN: triterpene dry extract from birch cork
Birken GmbHNULLNot RecruitingFemale: yes
Male: yes
Germany
130NCT01908088
(ClinicalTrials.gov)
July 201023/7/2013Autologous 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 HospitalCompleted5 Years25 YearsBoth6Phase 1Iran, Islamic Republic of
131EUCTR2009-012750-21-FR
(EUCTR)
20/01/201027/11/2009Traitement des épidermolyses bulleuses dystrophiques héréditaires parl’épigallocatéchine-3-gallate oral (Polyphenon E®)Traitement des épidermolyses bulleuses dystrophiques héréditaires parl’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
CHU de NICENULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
France
132NCT01033552
(ClinicalTrials.gov)
January 201014/12/2009Biochemical 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 MinnesotaNULLRecruitingN/A25 YearsAll75Phase 2United States
133NCT00881556
(ClinicalTrials.gov)
August 20, 200914/4/2009Allogeneic 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: Palifermin;Drug: Fludarabine;Drug: Busulfan;Drug: Lorazepam;Drug: Alemtuzumab;Drug: TacrolimusColumbia UniversityNULLTerminatedN/A21 YearsAll3Early Phase 1United States
134EUCTR2009-010763-17-SE
(EUCTR)
26/05/200920/03/2009Botulinumtoxin 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 study Epidermolysis Bullosa Simplex and Pachyonychia CongenitaTrade Name: DysportSophiahemmetNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2Sweden
135NCT00936533
(ClinicalTrials.gov)
May 20098/7/2009Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia CongenitaBotulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita - a Double-blind Placebo-controlled Phase II Proof of Concept StudyEpidermolysis Bullosa SimplexDrug: Dysport® (Botulinumtoxin A (Btx A));Drug: PlaceboUppsala UniversityNULLRecruiting16 YearsN/ABoth40Phase 2Sweden
136NCT00825565
(ClinicalTrials.gov)
February 200919/1/2009Study 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 ChicagoCompleted6 MonthsN/AAll8Phase 2United States
137NCT00478244
(ClinicalTrials.gov)
April 200723/5/2007Allogeneic 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 MinnesotaNULLTerminatedN/A25 YearsAll7N/AUnited States
138NCT00987142
(ClinicalTrials.gov)
December 200629/9/2009Trial 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.NULLCompletedN/AN/AAll12Phase 2Spain
139NCT00380640
(ClinicalTrials.gov)
September 200625/9/2006The 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 ChildrenNULLCompletedN/A20 YearsAll10Phase 2Canada
140EUCTR2005-002329-30-ES
(EUCTR)
09/08/200615/03/2010ENSAYO CLINICO COMPARATIVO, ABIERTO, MULTICENTRICO, PARA EVALUAR LA EFICACIA Y SEGURIDAD DE UNA NUEVA TERAPIA CON PIEL QUIMÉRICA CULTIVADA PARA EL TRATAMIENTO DE LAS LESIONES CUTÁNEAS DE LOS PACIENTES CON EPIDERMOLISIS BULLOSAENSAYO CLINICO COMPARATIVO, ABIERTO, MULTICENTRICO, PARA EVALUAR LA EFICACIA Y SEGURIDAD DE UNA NUEVA TERAPIA CON PIEL QUIMÉRICA CULTIVADA PARA EL TRATAMIENTO DE LAS LESIONES CUTÁNEAS DE LOS PACIENTES CON EPIDERMOLISIS BULLOSA Epidermolisis Bullosa distrófica recesiva con sindactilia cutánea severa
MedDRA version: 9.1;Level: LLT;Classification code 10014989;Term: Epidermolysis bullosa
Product Code: CX501CELLERIX, S.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Spain
141NCT00336154
(ClinicalTrials.gov)
March 3, 200612/6/2006Study 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 CampusNULLWithdrawn13 Years60 YearsAll0Phase 4Israel
142NCT00311766
(ClinicalTrials.gov)
February 20064/4/2006A 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.NULLTerminated2 YearsN/AAll30Phase 2United States
143NCT00231517
(ClinicalTrials.gov)
October 20053/10/2005Randomised 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 HealthNULLCompleted4 Years18 YearsBothPhase 4United Kingdom
144NCT00014729
(ClinicalTrials.gov)
October 200010/4/2001Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis BullosaEpidermolysis BullosaDrug: isotretinoinUniversity of North CarolinaNULLCompleted15 YearsN/ABoth20Phase 1NULL
145NCT00004359
(ClinicalTrials.gov)
February 199618/10/1999Phase II Pilot Study of Extracorporeal Phototherapy for Epidermolysis Bullosa AcquisitaEpidermolysis Bullosa AcquisitaDrug: methoxsalenNational Center for Research Resources (NCRR)Northwestern UniversityCompleted18 YearsN/ABoth10Phase 2NULL
146EUCTR2016-002066-32-DE
(EUCTR)
09/03/2017A 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLNAFemale: yes
Male: yes
192Phase 3Serbia;Hong Kong;Greece;Spain;Ukraine;Ireland;Chile;Israel;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Brazil;Belgium;Singapore;Croatia;Romania;Germany
147EUCTR2021-000103-20-NL
(EUCTR)
15/07/2021A study on the effect of Transvamix on pain in adults with epidermolysis bullosa.An explorative randomized, placebo-controlled and double-blind intervention crossover study: Transvamix (100mg/mL THC / 50mg/mL CBD) to treat chronic pain in Epidermolysis Bullosa Genetic Epidermolysis Bullosa;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23]Product Name: Transvamix (100mg/mL THC, 50mg/mL CBD)
INN or Proposed INN: THC
Other descriptive name: DELTA-9-TETRAHYDROCANNABINOL
INN or Proposed INN: CBD
Other descriptive name: CANNABIDIOL
University Medical Center GroningenNULLNAFemale: yes
Male: yes
16Phase 2Netherlands
148EUCTR2018-000439-29-NL
(EUCTR)
27/09/2018A 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
INN or Proposed INN: DIACEREIN
Castle Creek Pharmaceuticals, LLCNULLNAFemale: yes
Male: yes
20Phase 1France;United States;Germany;Netherlands;United Kingdom
149EUCTR2010-024428-10-FR
(EUCTR)
03/02/2011Traitement des épidermolyses bulleuses simples de type Dowling Maera par l'érythromicine oraleTraitement des épidermolyses bulleuses simples de type Dowling Maera par l'érythromicine orale Dowling 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
Trade Name: erythromycine
Product Name: erythrocine oral
CHU de NICENULLNAFemale: yes
Male: yes
8Phase 2France
150EUCTR2020-002936-55-CZ
(EUCTR)
03/08/2020Safety and Efficacy of Mesenchymal Stromal Cells Derived from Adipose Tissue of Non-related Donors in Patients with The Butterfly Disease: Clinical Trial Phase I/IISafety and Efficacy of Allogenic Adipose Tissue-derived Mesenchymal Stromal Cells in Patients with Epidermolysis Bullosa: Clinical Trial Phase I/II - MSC-EB Epidermolysis bullosa congenita;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Product Name: allogeneic adipose tissue-derived MSC
INN or Proposed INN: Human allogeneic adipose tissue-derived mesenchymal stem cells expanded
Other descriptive name: ALLOGENEIC ADIPOSE TISSUE-DERIVED MESENCHYMAL STEM CELLS EXPANDED
Masarykova univerzitaNULLNAFemale: yes
Male: yes
15Phase 1;Phase 2Czech Republic
151EUCTR2015-003670-32-DE
(EUCTR)
07/04/2016Phase I/II trial to establish safety, tolerability and efficacy of losartan in children with epidermolysis bullosaA dual-center prospective phase I/II trial to establish safety, tolerability and to obtain first data on efficacy of losartan in children with recessive dystrophic epidermolysis bullosa (RDEB) - REFLECT 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]
Trade Name: Losartan HEXAL
INN or Proposed INN: losartan potassium
Other descriptive name: LOSARTAN POTASSIUM
Medical Center - University of FreiburgNULLNot RecruitingFemale: yes
Male: yes
30Phase 2Austria;Germany
152EUCTR2017-004806-17-CZ
(EUCTR)
21/03/2018Study 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 - WINGS Recessive 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
INN or Proposed INN: unavailable
Wings Therapeutics Inc.NULLNAFemale: yes
Male: yes
8Phase 1;Phase 2France;United States;Czech Republic;Spain;Germany;United Kingdom
153EUCTR2016-004427-24-FR
(EUCTR)
21/08/2017An 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) 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
INN or Proposed INN: DIACEREIN
Other descriptive name: Diacerein
Castle Creek Pharmaceuticals, LLCNULLNot Recruiting Female: yes
Male: yes
80 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;France;Austria;Australia;Israel;Netherlands;Germany;United Kingdom
154EUCTR2014-002288-14-BE
(EUCTR)
14/12/2016An 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 Study Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm INC, An Amicus Therapeutics CompanyNULLNAFemale: yes
Male: yes
150Phase 3Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands
155EUCTR2014-005679-96-IT
(EUCTR)
21/01/2021An Investigation into the Long-term Safety of Zorblisa (SD-101-6.0) creamused by Patients with Epidermolysis Bullosa.An Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of ZorblisaTM (SD-101-6.0) in Patients with Epidermolysis Bullosa - Long-term Safety study of Zorblisa in Patients with Epidermolysis Bullosa Epidermolysis 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]
INN or Proposed INN: ALLANTOINASCIODERM, INCNULLNot RecruitingFemale: yes
Male: yes
150Phase 3Israel;Serbia;United States;Spain;Lithuania;Austria;Netherlands;Germany;Bulgaria;Australia;Romania;Poland;Belgium;France;Italy;United Kingdom
156EUCTR2017-004806-17-FR
(EUCTR)
05/02/2018Study 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 - WINGS Recessive 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
INN or Proposed INN: unavailable
ProQR TherapeuticsNULLNot RecruitingFemale: yes
Male: yes
14Phase 1;Phase 2United States;France;Czech Republic;Canada;Spain;Austria;Germany;United Kingdom
157EUCTR2021-000214-42-FR
(EUCTR)
26/04/2021INM-755 cream for patients with EB.A Randomised, Double-Blind, Vehicle-Controlled Phase 2 Study of Topically Applied INM-755 (cannabinol) Cream in Patients with Epidermolysis Bullosa. Epidermolysis 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: Cannabinol (CBN)
Product Code: INM-755
INN or Proposed INN: CANNABINOL
InMed Pharmaceuticals Inc.NULLNAFemale: yes
Male: yes
20Phase 2Serbia;France;Greece;Austria;Israel;Germany;Italy
158EUCTR2014-005679-96-PL
(EUCTR)
11/12/2015An 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 Bullosa Epidermolysis 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
INN or Proposed INN: ALLANTOIN
Other descriptive name: ALLANTOIN
Scioderm, INC, An Amicus Therapeutics CompanyNULLNot RecruitingFemale: yes
Male: yes
150Phase 3Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Romania;Australia;Bulgaria;Germany;Netherlands
159EUCTR2016-002066-32-CZ
(EUCTR)
29/11/2017A 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 study Inherited Epidermolysis 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]
Trade Name: Episalvan gel
Product Name: Oleogel-S10
INN or Proposed INN: Birch bark extract
Other descriptive name: BIRCH BARK EXTRACT
Amryt Research LimitedNULLNot RecruitingFemale: yes
Male: yes
250Phase 3United States;Serbia;Hong Kong;Greece;Spain;Ukraine;Ireland;Chile;Israel;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Brazil;Belgium;Singapore;Croatia;Romania;Georgia;Germany
160EUCTR2021-000214-42-DE
(EUCTR)
19/04/2021INM-755 cream for patients with EB.A Randomised, Double-Blind, Vehicle-Controlled Phase 2 Study of Topically Applied INM-755 (cannabinol) Cream in Patients with Epidermolysis Bullosa. Epidermolysis 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: Cannabinol (CBN)
Product Code: INM-755
INN or Proposed INN: CANNABINOL
InMed Pharmaceuticals Inc.NULLNAFemale: yes
Male: yes
20Phase 2France;Serbia;Greece;Austria;Israel;Germany;Italy