36. 表皮水疱症
［臨床試験数：147，薬物数：170（DrugBank：40），標的遺伝子数：32，標的パスウェイ数：113］
Searched query = "Epidermolysis bullosa", "EBS", "JEB", "DDEB", "RDEB", "Kindler syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2018-003890-91-PL (EUCTR) | 29/09/2020 | 13/11/2018 | Treatment of wound in the course of Epidermolysis Bullosa, chronic venous leg ulceration and thermal injury by biological dressing made of mesynchemal stem cells seeded on acellular human skin. | The development of innovative advanced therapy medicinal product (biological dressing of the human race) in the treatment of Epidermolysis Bullosa (EB) and other chronic wounds. | BIOOPA dressing will be used in the treatment of wounds in the course of Epidermolysis Bullosa, chronic venous leg ulceration and thermal injury (second degree burn). MedDRA version: 20.0;Level: PT;Classification code 10014989;Term: Epidermolysis bullosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Name: BIOOPA dressing INN or Proposed INN: Human Allogeneic WJ-MSCs Other descriptive name: WHARTON’S JELLY DERIVED MESENCHYMAL STEM CELLS | Medical University of Warsaw | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 1;Phase 2 | Poland | ||
| 2 | EUCTR2020-002337-15-NO (EUCTR) | 16/09/2020 | 29/06/2020 | TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA | TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS (THE GENTELBULL STUDY) - GENTELBULL | Epidermolysis bullosa caused by nonsense mutations or splice site mutations;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Trade Name: Infectogenta Product Name: Gentamicin sulfate 0.1% ointment | Oslo University Hospital | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 6 | Phase 2 | Norway | ||
| 3 | EUCTR2020-001542-19-FR (EUCTR) | 22/07/2020 | 03/04/2020 | Comparing the Efficacy and Safety of Ixekizumab to Placebo in Patients > 6 years of age with EB simplex generalized severe | A 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 NICE | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 0 | Phase 2 | France | ||
| 4 | NCT04186650 (ClinicalTrials.gov) | January 10, 2020 | 25/11/2019 | Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent Grafts | 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 | Epidermolysis Bullosa Dystrophica, Recessive | Biological: COL7A1-SIN retroviral vector engineered autologous tissue-engineered skin | Institut National de la Santé Et de la Recherche Médicale, France | NULL | Enrolling by invitation | 18 Years | N/A | All | 3 | Phase 1;Phase 2 | France |
| 5 | EUCTR2018-001009-98-IT (EUCTR) | 11/10/2019 | 10/11/2020 | Study 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. KG | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 1 | United States;France;Austria;Germany;United Kingdom;Italy | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2018-001009-98-DE (EUCTR) | 01/03/2019 | 03/05/2018 | Study 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. KG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 1;Phase 2 | United States;France;Austria;Germany;United Kingdom;Italy | ||
| 7 | EUCTR2018-001009-98-AT (EUCTR) | 05/11/2018 | 24/05/2018 | Study 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. KG | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 1;Phase 2 | United States;France;Austria;Germany;United Kingdom;Italy | ||
| 8 | EUCTR2018-001009-98-GB (EUCTR) | 18/09/2018 | 25/10/2018 | Study 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. KG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 1;Phase 2 | United States;France;Austria;Germany;Italy;United Kingdom | ||
| 9 | EUCTR2017-004806-17-ES (EUCTR) | 06/09/2018 | 11/04/2018 | Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene | A 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 Theraputics | NULL | Not Recruiting | Female: yes Male: yes | 8 | Phase 1;Phase 2 | France;United States;Czech Republic;Canada;Spain;Austria;Germany;United Kingdom | ||
| 10 | EUCTR2018-000439-29-GB (EUCTR) | 04/06/2018 | 22/03/2018 | A 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, LLC | NULL | Not Recruiting | Female: yes Male: yes | 4 | Phase 1 | United States;France;Netherlands;Germany;United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2017-003757-41-DE (EUCTR) | 19/04/2018 | 21/02/2018 | An 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, LLC | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | United States;France;Austria;Australia;Israel;Netherlands;Germany;United Kingdom;Italy | ||
| 12 | EUCTR2017-003757-41-NL (EUCTR) | 11/04/2018 | 06/12/2017 | An 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, LLC | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | United States;France;Austria;Australia;Israel;Germany;Netherlands;United Kingdom;Italy | ||
| 13 | EUCTR2016-002066-32-DK (EUCTR) | 05/04/2018 | 17/01/2018 | A 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 Limited | NULL | Not Recruiting | Female: yes Male: yes | 250 | Phase 3 | Serbia;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 | ||
| 14 | EUCTR2016-002066-32-BE (EUCTR) | 23/03/2018 | 17/01/2018 | A 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 Limited | NULL | Not Recruiting | Female: yes Male: yes | 192 | Phase 3 | Serbia;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 | ||
| 15 | EUCTR2016-002066-32-HR (EUCTR) | 26/02/2018 | 12/04/2018 | A 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 192 | Phase 3 | Serbia;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 | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | EUCTR2017-003757-41-GB (EUCTR) | 19/02/2018 | 13/11/2017 | An 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, LLC | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | United States;France;Austria;Australia;Israel;Netherlands;Germany;Italy;United Kingdom | ||
| 17 | EUCTR2017-003757-41-FR (EUCTR) | 07/02/2018 | 17/10/2018 | An 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, LLC | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | United States;France;Austria;Australia;Israel;Netherlands;Germany;United Kingdom | ||
| 18 | EUCTR2016-002066-32-HU (EUCTR) | 08/01/2018 | 29/11/2017 | A 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 Limited | NULL | Not Recruiting | Female: yes Male: yes | 164 | Phase 3 | Hong 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 | ||
| 19 | EUCTR2017-003757-41-AT (EUCTR) | 04/01/2018 | 29/11/2017 | An 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, LLC | NULL | Not Recruiting | Female: yes Male: yes | 84 | Phase 2 | France;United States;Australia;Austria;Israel;Netherlands;Germany;Italy;United Kingdom | ||
| 20 | EUCTR2016-002066-32-IE (EUCTR) | 22/12/2017 | 07/03/2017 | A 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 Limited | NULL | Not Recruiting | Female: yes Male: yes | 250 | Phase 3 | Serbia;Greece;Spain;Ukraine;Ireland;Turkey;Austria;Israel;Russian Federation;United Kingdom;Switzerland;Italy;France;Czech Republic;Hungary;Mexico;Belgium;Croatia;Denmark;Australia;Germany | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | EUCTR2016-003832-19-AT (EUCTR) | 11/12/2017 | 09/08/2017 | Clinical 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 Austria | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2 | Austria | ||
| 22 | EUCTR2017-000606-37-ES (EUCTR) | 16/11/2017 | 18/09/2017 | Safety 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 Paz | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 9 | Phase 1 | Spain | ||
| 23 | EUCTR2015-003670-32-AT (EUCTR) | 05/10/2017 | 07/08/2017 | Phase I/II trial to establish safety, tolerability and efficacy of losartan in children with epidermolysis bullosa | A 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 Freiburg | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | Austria;Germany | ||
| 24 | EUCTR2016-002066-32-AT (EUCTR) | 26/09/2017 | 08/03/2017 | A 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 Limited | NULL | Not Recruiting | Female: yes Male: yes | 250 | Phase 3 | 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;Germany | ||
| 25 | EUCTR2016-000095-17-AT (EUCTR) | 17/08/2017 | 07/06/2017 | Gene 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 1;Phase 2 | Austria | |||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | EUCTR2016-004427-24-GB (EUCTR) | 21/07/2017 | 06/07/2017 | An 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, LLC | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | France;United States;Austria;Australia;Israel;Netherlands;Germany;Italy;United Kingdom | ||
| 27 | EUCTR2016-002066-32-ES (EUCTR) | 16/03/2017 | 10/03/2017 | A 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 164 | Phase 3 | United States;Greece;Spain;Ireland;Turkey;Austria;Israel;Italy;Switzerland;United Kingdom;France;Mexico;Croatia;Australia;Germany | ||
| 28 | EUCTR2014-005679-96-LT (EUCTR) | 06/03/2017 | 09/02/2017 | An 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 Company | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands | ||
| 29 | EUCTR2014-002288-14-LT (EUCTR) | 07/12/2016 | 08/11/2016 | An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa | A 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 Company | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | United States;Serbia;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Belgium;Poland;Romania;Australia;Germany;Netherlands | ||
| 30 | EUCTR2014-005679-96-ES (EUCTR) | 14/11/2016 | 09/09/2016 | An 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, INC | NULL | Not Recruiting | Female: yes Male: yes | 130 | Phase 3 | France;United States;Poland;Spain;Lithuania;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | EUCTR2014-002288-14-ES (EUCTR) | 19/09/2016 | 05/08/2016 | An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 90 | Phase 3 | France;United States;Belgium;Poland;Spain;Lithuania;Austria;Germany;Netherlands;Italy;United Kingdom | ||
| 32 | EUCTR2016-001967-35-AT (EUCTR) | 19/07/2016 | 28/06/2016 | A study to assess the efficacy of a vitamin D-containing ointment applied on the skin to improve wound healing in butterfly children | A 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 Salzburg | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Austria | ||
| 33 | EUCTR2015-004592-74-AT (EUCTR) | 01/06/2016 | 19/01/2016 | Gene 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 1;Phase 2 | Austria | ||
| 34 | EUCTR2014-004884-19-GB (EUCTR) | 24/05/2016 | 18/02/2015 | Gene Therapy Trial in adults with Inherited Blistering Skin Disease | Phase 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 London | Guy's and St Thomas' NHS Foundation Trust | Not Recruiting | Female: yes Male: yes | 10 | Phase 1 | United Kingdom | ||
| 35 | EUCTR2014-005679-96-NL (EUCTR) | 07/09/2015 | 02/03/2015 | An 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, INC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 130 | France;United States;Poland;Spain;Austria;Australia;Germany;Netherlands;Italy;United Kingdom | |||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | EUCTR2014-002288-14-PL (EUCTR) | 10/08/2015 | 11/06/2015 | An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa | A 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 Company | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Belgium;Poland;Romania;Australia;Germany;Netherlands | ||
| 37 | EUCTR2014-005679-96-GB (EUCTR) | 22/04/2015 | 04/03/2015 | An 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, INC | NULL | Not Recruiting | Female: yes Male: yes | 130 | Phase 3 | France;United States;Poland;Spain;Lithuania;Austria;Australia;Netherlands;Germany;Italy;United Kingdom | ||
| 38 | EUCTR2014-002288-14-DE (EUCTR) | 31/03/2015 | 19/12/2014 | An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa | A 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 Company | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Belgium;Poland;Romania;Australia;Germany;Netherlands | ||
| 39 | EUCTR2014-005679-96-AT (EUCTR) | 16/03/2015 | 12/02/2015 | An 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 | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands | ||
| 40 | EUCTR2014-002288-14-NL (EUCTR) | 23/12/2014 | 03/07/2014 | An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa | A 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 Company | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Belgium;Poland;Romania;Australia;Netherlands;Germany | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 41 | EUCTR2014-002288-14-IT (EUCTR) | 18/12/2014 | 15/07/2014 | An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 90 | Phase 3 | France;United States;Belgium;Spain;Poland;Lithuania;Austria;Germany;Netherlands;United Kingdom;Italy | ||
| 42 | EUCTR2014-002288-14-AT (EUCTR) | 08/08/2014 | 02/07/2014 | An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa | A 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 Company | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands | ||
| 43 | EUCTR2012-000605-72-NL (EUCTR) | 06/02/2014 | 12/08/2013 | Stem cell transplantation with cord blood and mesenchymal stem cells after reduced intensity conditioning for severe forms of the blistering disease epidermolysis bullosa | Unrelated cord blood transplantation after reduced toxicity conditioning with mesenchymal stromal cell co-infusion in patients with severe epidermolysis bullosa - CB+MSCforEB | The source population consists of patients referred to or within the UMC Groningen because they have diagnosed clinically and genetically severe generalized RDEB.;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Name: TC-MSC INN or Proposed INN: Mesenchymal stromal cells Trade Name: Busilvex Trade Name: Fludarabine Product Name: FLudarabine Trade Name: THYMOGLOBULINE Product Name: Thymoglobuline | Universitair Medisch Centrum Utrecht | NULL | Not Recruiting | Female: yes Male: yes | 11 | Phase 2 | Netherlands | ||
| 44 | EUCTR2013-002034-21-AT (EUCTR) | 16/12/2013 | 01/08/2013 | Diacerein for butterfly children | Diacerein 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 Austria | NULL | Not Recruiting | Female: yes Male: yes | Phase 3 | Austria | |||
| 45 | EUCTR2012-001815-21-PT (EUCTR) | 07/06/2013 | 28/03/2013 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 26 | France;United States;Portugal;Canada;Spain;Poland;Austria;Germany | |||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 46 | EUCTR2012-001815-21-DE (EUCTR) | 22/05/2013 | 01/11/2012 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 26 | Portugal;United States;Spain;Austria;Australia;Germany | |||
| 47 | EUCTR2012-001815-21-AT (EUCTR) | 24/01/2013 | 25/01/2013 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 26 | United States;Portugal;France;Canada;Spain;Poland;Austria;Germany | |||
| 48 | EUCTR2012-001815-21-ES (EUCTR) | 18/01/2013 | 26/09/2012 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 25 | Portugal;United States;Spain;Austria;Australia;Germany | |||
| 49 | EUCTR2012-001394-87-GB (EUCTR) | 16/11/2012 | 22/08/2012 | 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. | 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 London | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 1;Phase 2 | United Kingdom | ||
| 50 | EUCTR2010-023121-38-GB (EUCTR) | 13/01/2011 | 26/11/2010 | A 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 | A 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 bullosa | Trade Name: ICX-RHY-013 Product Name: Cultured allogeneic fibroblasts Product Code: ICX-RHY-013 INN or Proposed INN: Fibroblasts | Intercytex Ltd | NULL | Not Recruiting | Female: yes Male: yes | 25 | Phase 2 | United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 51 | NCT01263379 (ClinicalTrials.gov) | October 5, 2010 | 15/12/2010 | Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa | A 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 Bullosa | Biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets | Stanford University | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);Abeona Therapeutics, Inc | Active, not recruiting | 13 Years | N/A | All | 10 | Phase 1;Phase 2 | United States |
| 52 | EUCTR2010-019945-24-DE (EUCTR) | 24/09/2010 | 13/07/2010 | Open, 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 | Open, 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 GmbH | NULL | Not Recruiting | Female: yes Male: yes | Germany | ||||
| 53 | EUCTR2009-012750-21-FR (EUCTR) | 20/01/2010 | 27/11/2009 | Traitement 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 NICE | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | France | ||||
| 54 | EUCTR2009-010763-17-SE (EUCTR) | 26/05/2009 | 20/03/2009 | Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia congenita-a double-blind placebo-controlled phase II proof of concept study | Botulinumtoxin 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 Congenita | Trade Name: Dysport | Sophiahemmet | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 2 | Sweden | |||
| 55 | NCT00004359 (ClinicalTrials.gov) | February 1996 | 18/10/1999 | Phase II Pilot Study of Extracorporeal Phototherapy for Epidermolysis Bullosa Acquisita | Epidermolysis Bullosa Acquisita | Drug: methoxsalen | National Center for Research Resources (NCRR) | Northwestern University | Completed | 18 Years | N/A | Both | 10 | Phase 2 | NULL | |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 56 | EUCTR2014-005679-96-PL (EUCTR) | 11/12/2015 | An 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 Company | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Romania;Australia;Bulgaria;Germany;Netherlands | |||
| 57 | EUCTR2016-002066-32-CZ (EUCTR) | 29/11/2017 | A 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 Limited | NULL | NA | Female: yes Male: yes | 250 | Phase 3 | United 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 | |||
| 58 | EUCTR2016-002066-32-DE (EUCTR) | 09/03/2017 | A 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 Limited | NULL | NA | Female: yes Male: yes | 192 | Phase 3 | Serbia;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 | |||
| 59 | EUCTR2018-000439-29-NL (EUCTR) | 27/09/2018 | A 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, LLC | NULL | NA | Female: yes Male: yes | 20 | Phase 1 | France;United States;Germany;Netherlands;United Kingdom | |||
| 60 | EUCTR2017-004806-17-CZ (EUCTR) | 21/03/2018 | Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene | A 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. | NULL | NA | Female: yes Male: yes | 8 | Phase 1;Phase 2 | France;United States;Czech Republic;Spain;Germany;United Kingdom | |||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 61 | EUCTR2017-004806-17-FR (EUCTR) | 05/02/2018 | Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene | A 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 Therapeutics | NULL | NA | Female: yes Male: yes | 14 | Phase 1;Phase 2 | United States;France;Czech Republic;Canada;Austria;Germany;United Kingdom | |||
| 62 | EUCTR2018-000261-36-IT (EUCTR) | 07/10/2020 | Gene Therapy for patient with Junctional Epidermolysis Bullosa | MULTICENTRE, 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. | NULL | NA | Female: yes Male: yes | 6 | Phase 2;Phase 3 | France;Germany;Italy | |||
| 63 | EUCTR2015-003670-32-DE (EUCTR) | 07/04/2016 | Phase I/II trial to establish safety, tolerability and efficacy of losartan in children with epidermolysis bullosa | A 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 Freiburg | NULL | NA | Female: yes Male: yes | 30 | Phase 2 | Austria;Germany | |||
| 64 | EUCTR2010-024428-10-FR (EUCTR) | 03/02/2011 | Traitement des épidermolyses bulleuses simples de type Dowling Maera par l'érythromicine orale | Traitement 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 NICE | NULL | NA | Female: yes Male: yes | 8 | Phase 2 | France | |||
| 65 | EUCTR2014-002288-14-BE (EUCTR) | 14/12/2016 | An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa | A 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 Company | NULL | NA | Female: yes Male: yes | 150 | Phase 3 | Serbia;United States;Spain;Lithuania;Austria;Israel;United Kingdom;Italy;France;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands |