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 | NCT04177498 (ClinicalTrials.gov) | January 1, 2021 | 7/11/2019 | Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC | A Pilot, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma | Recessive Dystrophic Epidermolysis Bullosa | Drug: Rigosertib Sodium;Other: Quality-of-Life Assessment | Thomas Jefferson University | Onconova Therapeutics, Inc. | Not yet recruiting | 18 Years | 79 Years | All | 6 | Early Phase 1 | United States |
| 2 | NCT03786237 (ClinicalTrials.gov) | January 2021 | 14/11/2018 | Rigosertib for RDEB-SCC | 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 | Epidermolysis Bullosa Dystrophica;Squamous Cell Carcinoma | Drug: Rigosertib Oral Capsules / Rigosertib Intravenous | Prof. Johann Bauer | NULL | Recruiting | 18 Years | 79 Years | All | 12 | Phase 1;Phase 2 | Austria |
| 3 | NCT04644627 (ClinicalTrials.gov) | December 1, 2020 | 20/11/2020 | Topical Gentamicin Nonsense Suppression Therapy of EB | TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS | Epidermolysis Bullosa | Drug: Gentamicin Sulfate | Oslo University Hospital | NULL | Not yet recruiting | N/A | N/A | All | 6 | Phase 1;Phase 2 | Norway |
| 4 | NCT04599881 (ClinicalTrials.gov) | October 15, 2020 | 8/10/2020 | A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa | A Phase 2 Open-Label Study of PTR-01 in Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) | Recessive Dystrophic Epidermolysis Bullosa | Drug: PTR-01 | Phoenix Tissue Repair, Inc. | NULL | Recruiting | 12 Years | N/A | All | 6 | Phase 2 | United States |
| 5 | 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 | ||
| 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 | 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 | ||
| 7 | NCT04173650 (ClinicalTrials.gov) | September 2020 | 18/11/2019 | MSC EVs in Dystrophic Epidermolysis Bullosa | A Safety Study of the Administration of MSC Extracellular Vesicles in the Treatment of Dystrophic Epidermolysis Bullosa Wounds | Dystrophic Epidermolysis Bullosa | Drug: AGLE 102 | Aegle Therapeutics | NULL | Not yet recruiting | N/A | N/A | All | 10 | Phase 1;Phase 2 | NULL |
| 8 | NCT04613102 (ClinicalTrials.gov) | August 1, 2020 | 27/10/2020 | The Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III Trial | The Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III Trial | Epidermolysis Bullosa;Pain;Itch | Drug: AVCN583601 (3% Cannabidiol cream) | Elena Pope | Avicanna Inc | Withdrawn | 4 Years | 50 Years | All | 0 | Phase 2;Phase 3 | Canada |
| 9 | NCT04491604 (ClinicalTrials.gov) | August 2020 | 22/7/2020 | The 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 Bullosa | Biological: Topical Beremagene Geperpavec;Other: Placebo | Krystal Biotech, Inc. | NULL | Recruiting | 6 Months | N/A | All | 30 | Phase 3 | United States |
| 10 | 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 | ||
| 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 | NCT04213261 (ClinicalTrials.gov) | June 9, 2020 | 2/12/2019 | A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa | A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Biological: FCX-007 (debcoemagene autoficel; see below for FCX-007 description) | Fibrocell Technologies, Inc. | NULL | Recruiting | 2 Years | N/A | All | 24 | Phase 3 | United States |
| 12 | 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 |
| 13 | NCT04227106 (ClinicalTrials.gov) | January 10, 2020 | 10/1/2020 | Phase 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 Bullosa | Biological: EB-101 | Abeona Therapeutics, Inc | NULL | Recruiting | 6 Years | N/A | All | 15 | Phase 3 | United States |
| 14 | NCT04140786 (ClinicalTrials.gov) | October 31, 2019 | 23/10/2019 | Optimizing IV Gentamicin in JEB | Optimization of Intravenous Gentamicin Treatment to Restore Functional Laminin 332 in JEB Patients With Nonsense Mutations | Junctional Epidermolysis Bullosa | Drug: Gentamicin Sulfate, Injectable | University of Southern California | NULL | Recruiting | N/A | N/A | All | 6 | Phase 1;Phase 2 | United States |
| 15 | 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 |
| 16 | NCT03928093 (ClinicalTrials.gov) | August 7, 2019 | 27/3/2019 | Pregabalin Treatment for RDEB Pain and Itch | A Double-blind, Randomized, Cross-over, Feasibility Trial of Pregabalin for the Treatment of Recessive Dystrophic Epidermolysis Bullosa-associated Neuropathic Pain and Itch | Pain, Neuropathic;Itch;Epidermolysis Bullosa | Drug: Pregabalin | The Hospital for Sick Children | Epidemolysis Bullosa Research Partnership | Recruiting | 8 Years | 40 Years | All | 15 | Phase 3 | Canada |
| 17 | NCT03578029 (ClinicalTrials.gov) | May 22, 2019 | 14/6/2018 | Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa | A 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 Bullosa | Drug: RGN-137;Drug: Placebo | Lenus Therapeutics, LLC | NULL | Recruiting | 4 Years | N/A | All | 15 | Phase 2 | United States |
| 18 | NCT03836001 (ClinicalTrials.gov) | April 18, 2019 | 7/2/2019 | A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa | A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: Serlopitant Tablet;Drug: Placebo Oral Tablet | Stanford University | Epidermolysis Bullosa Research Partnership;Menlo Therapeutics | Recruiting | 13 Years | N/A | All | 40 | Phase 2 | United States |
| 19 | 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 | ||
| 20 | EUCTR2016-002790-35-FR (EUCTR) | 07/02/2019 | 17/01/2018 | 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] | INSERM | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 3 | Phase 1;Phase 2 | France | |||
| 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 | NCT03529877 (ClinicalTrials.gov) | January 16, 2019 | 24/4/2018 | Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa | 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 | Biological: allo-APZ2-EB | RHEACELL GmbH & Co. KG | FGK Clinical Research GmbH;Granzer Regulatory Consulting & Services;Ticeba GmbH | Active, not recruiting | N/A | 55 Years | All | 16 | Phase 1;Phase 2 | United States;Austria;France;Germany;United Kingdom;Italy |
| 22 | NCT03752905 (ClinicalTrials.gov) | January 9, 2019 | 19/11/2018 | A 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 Bullosa | Drug: PTR-01;Drug: Normal saline | Phoenix Tissue Repair, Inc. | NULL | Active, not recruiting | 16 Years | N/A | All | 12 | Phase 1;Phase 2 | United States |
| 23 | EUCTR2018-001009-98-FR (EUCTR) | 07/01/2019 | 30/10/2018 | 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] | RHEACELL GmbH & Co. KG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Phase 1;Phase 2 | United States;France;Austria;Germany;United Kingdom | |||
| 24 | 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 | ||
| 25 | NCT03468322 (ClinicalTrials.gov) | October 20, 2018 | 8/3/2018 | A Double-blind, Intra-individual Comparison, POC Trial of AC-203 in EB Patients | A Double-blind, Intra-individual Comparison, Proof-of-concept Trial of Topical AC-203 in Patients With Inherited Epidermolysis Bullosa | Inherited Epidermolysis Bullosa | Drug: AC-203;Drug: Vehicle | TWi Biotechnology, Inc. | NULL | Completed | 2 Years | N/A | All | 9 | Phase 2 | Taiwan |
| 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 | NCT03632265 (ClinicalTrials.gov) | October 1, 2018 | 17/7/2018 | Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa | VITAL: A Pivotal Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) (GENE TRANSFER) | Recessive Dystrophic Epidermolysis Bullosa | Biological: EB-101 | Jean Yuh Tang | Abeona Therapeutics, Inc | Not yet recruiting | 6 Years | N/A | All | 15 | Phase 3 | NULL |
| 27 | EUCTR2017-004806-17-DE (EUCTR) | 27/09/2018 | 08/02/2018 | 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] | Wings Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 8 | Phase 1;Phase 2 | France;United States;Czech Republic;Spain;Germany;United Kingdom | |||
| 28 | 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 | ||
| 29 | 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 | ||
| 30 | NCT03392909 (ClinicalTrials.gov) | July 5, 2018 | 2/1/2018 | Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) | Restoration of Full-Length Type VII Collagen in RDEB Patients With Nonsense Mutations After Intravenous Gentamicin Treatment | Recessive Dystrophic Epidermolysis Bullosa | Drug: Gentamicin | University of Southern California | NULL | Recruiting | 7 Years | N/A | All | 9 | Phase 1;Phase 2 | United States |
| 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 | NCT03605069 (ClinicalTrials.gov) | July 2, 2018 | 25/6/2018 | Topical QR-313 in Dominant Dystrophic Epidermolysis Bullosa (DDEB) or Recessive Dystrophic Epidermolysis Bullosa (RDEB) Due to Mutation(s) in Exon 73 of the COL7A1gene | 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 DDEB or RDEB Due to Mutation(s) in Exon 73 of the COL7A1 Gene | Epidermolysis Bullosa Dystrophica, Recessive;Epidermolysis Bullosa Dystrophica, Dominant | Drug: QR-313;Drug: Placebo | Wings Therapeutics Inc. | NULL | Recruiting | 4 Years | N/A | All | 14 | Phase 1;Phase 2 | United States;France;Spain |
| 32 | NCT03453632 (ClinicalTrials.gov) | June 14, 2018 | 6/2/2018 | Injections of Botulinic Toxin in Plantar Lesions of Localized Epidermolysis Bullosa Simplex | Evaluation 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 Simplex | Drug: Botulinic toxin;Drug: Placebo | University Hospital, Toulouse | NULL | Recruiting | 18 Years | N/A | All | 25 | Phase 2;Phase 3 | France |
| 33 | 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 | ||
| 34 | NCT03526159 (ClinicalTrials.gov) | June 1, 2018 | 17/4/2018 | Gentamicin for Junctional Epidermolysis Bullosa | A Pilot Study of the Restoration of Functional Laminin 332 in JEB Patients With Nonsense Mutations After Topical and Intravenous Gentamicin Treatment | Junctional Epidermolysis Bullosa | Drug: Gentamicin Sulfate | University of Southern California | NULL | Recruiting | N/A | N/A | All | 6 | Phase 1;Phase 2 | United States |
| 35 | EUCTR2017-004806-17-GB (EUCTR) | 18/05/2018 | 24/01/2018 | 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] | Wings Therapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | 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 |
| 36 | NCT03472287 (ClinicalTrials.gov) | May 18, 2018 | 14/3/2018 | To 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 Bullosa | Drug: Diacerein 1% Ointment | Castle Creek Pharmaceuticals, LLC | NULL | Completed | 4 Years | N/A | All | 11 | Phase 1 | United States;France;Netherlands;United Kingdom |
| 37 | NCT04153630 (ClinicalTrials.gov) | May 17, 2018 | 6/8/2019 | Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa | Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa | Epidermolysis Bullosa Dystrophica, Recessive | Biological: mesenchymal stem cells derived from bone marrow (BM-MSCs) | Instituto de Investigación Hospital Universitario La Paz | Universidad Carlos III Madrid (TERMeG);St John's Institute of Dermatology Kings College London;Instituto de Salud Carlos III;DEBRA;CIBER Enfermedades raras | Active, not recruiting | 12 Months | 18 Years | All | 9 | Phase 1;Phase 2 | Spain |
| 38 | NCT03536143 (ClinicalTrials.gov) | May 7, 2018 | 20/4/2018 | Topical Beremagene Geperpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa | A 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 Bullosa | Biological: Topical beremagene geperpavec | Krystal Biotech, Inc. | NULL | Active, not recruiting | 5 Years | N/A | All | 4 | Phase 2 | United States |
| 39 | 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 | ||
| 40 | 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 | ||
| 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 | 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 | ||
| 42 | 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 | ||
| 43 | 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 | ||
| 44 | 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 | ||
| 45 | 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 | ||
| 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 | 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 | ||
| 47 | 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 | ||
| 48 | 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 | ||
| 49 | 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 | ||
| 50 | NCT03389308 (ClinicalTrials.gov) | December 1, 2017 | 19/12/2017 | Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex | 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;Epidermolysis Bullosa Simplex | Drug: diacerein 1% ointment | Castle Creek Pharmaceuticals, LLC | NULL | Completed | 6 Months | 99 Years | All | 51 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 51 | 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 | ||
| 52 | EUCTR2016-002066-32-GB (EUCTR) | 10/11/2017 | 13/02/2017 | 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] | Amryt Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 192 | Phase 3 | Serbia;Greece;Spain;Ukraine;Ireland;Turkey;Austria;Russian Federation;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Hungary;Mexico;Belgium;Croatia;Denmark;Australia;Germany | |||
| 53 | EUCTR2016-002066-32-IT (EUCTR) | 18/10/2017 | 30/01/2018 | 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] | 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;Czech Republic;Hungary;Mexico;Belgium;Croatia;Denmark;Australia;Germany | |||
| 54 | EUCTR2016-002066-32-GR (EUCTR) | 16/10/2017 | 05/05/2017 | A 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] | Amryt Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | 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 | |||
| 55 | EUCTR2016-004427-24-AT (EUCTR) | 10/10/2017 | 30/05/2017 | 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] | Castle Creek Pharmaceuticals, LLC | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | France;United States;Australia;Austria;Israel;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 |
| 56 | 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 | ||
| 57 | 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 | ||
| 58 | EUCTR2016-004427-24-NL (EUCTR) | 06/09/2017 | 23/06/2017 | 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] | Castle Creek Pharmaceuticals, LLC | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | France;United States;Austria;Australia;Israel;Germany;Netherlands;Italy;United Kingdom | |||
| 59 | EUCTR2016-004427-24-DE (EUCTR) | 24/08/2017 | 23/05/2017 | 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] | 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 | |||
| 60 | 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 |
| 61 | 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 | ||
| 62 | NCT03183934 (ClinicalTrials.gov) | July 2017 | 8/6/2017 | A Follow-up Study to Evaluate the Efficacy and Safety of ALLO-ASC-DFU in ALLO-ASC-EB-101 Clinical Trial | A 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-101 | Dystrophic Epidermolysis Bullosa | Biological: ALLO-ASC-DFU | Anterogen Co., Ltd. | NULL | Not yet recruiting | 2 Years | 60 Years | All | 5 | Phase 1;Phase 2 | Korea, Republic of |
| 63 | JPRN-UMIN000028366 | 2017/06/21 | 25/07/2017 | 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. - A clinical study to evaluate efficacy, safety and tolerability of ISN001 in dystrophic epidermolysis bullosa patients. | dystrophic epidermolysis bullosa | ISN001, the sheet containing allogenic adipose derived mesenchymal stromal cells (1000000/sheet), is applied to the lesion(s) once a week up to 8 times | Ishin Pharmaceutical Co. Ltd. | NULL | Recruiting | 6years-old | 70years-old | Male and Female | 5 | Phase 1;Phase 2 | Japan |
| 64 | NCT03154333 (ClinicalTrials.gov) | June 1, 2017 | 10/5/2017 | Safety 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 Simplex | Epidermolysis Bullosa Simplex | Drug: diacerein 1% ointment;Drug: A placebo ointment | Castle Creek Pharmaceuticals, LLC | NULL | Terminated | 4 Years | N/A | All | 54 | Phase 2 | United States;Australia;Austria;France;Germany;Israel;Netherlands;United Kingdom |
| 65 | NCT03068780 (ClinicalTrials.gov) | March 29, 2017 | 27/2/2017 | Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa | 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 | Epidermolysis Bullosa | Drug: Oleogel-S10;Drug: Placebo | Amryt Research Limited | NULL | Active, not recruiting | N/A | N/A | All | 223 | Phase 3 | United 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 |
| 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 |
| 66 | 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 | ||
| 67 | 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 | ||
| 68 | NCT03730584 (ClinicalTrials.gov) | February 27, 2017 | 2/11/2018 | Evaluation of the Efficacy of ROPIVACAINE in Children and Young Adults With Hereditary Epidermolysis Bullosa | Evaluation of the Efficacy of a Topical Analgesic Treatment With ROPIVACAINE in Children and Young Adults With Hereditary Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: Ropivacaine;Biological: Blood test | Assistance Publique - Hôpitaux de Paris | NULL | Active, not recruiting | N/A | 21 Years | All | 10 | N/A | France |
| 69 | NCT03012191 (ClinicalTrials.gov) | February 2, 2017 | 4/1/2017 | Gentamicin for RDEB | Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa Patients With Nonsense Mutations | Recessive Dystrophic Epidermolysis Bullosa | Drug: Gentamicin Sulfate | University of Southern California | NULL | Completed | N/A | N/A | All | 6 | Phase 1;Phase 2 | United States |
| 70 | NCT02984085 (ClinicalTrials.gov) | January 30, 2017 | 2/12/2016 | Clinical 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 Bullosa | Drug: Genetically corrected cultured epidermal autograft (ATMP) | Holostem Terapie Avanzate s.r.l. | Paracelsus Medical University | Active, not recruiting | 6 Years | 54 Years | All | 3 | 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 |
| 71 | NCT03942250 (ClinicalTrials.gov) | January 2017 | 22/2/2018 | Uses of Irradiated Human Amniotic Membrane in the Treatment of Dystrophic Epidermolysis Bullosa Patients | Uses of Irradiated Human Amniotic Membrane in the Treatment of Dystrophic Epidermolysis Bullosa Patients | Epidermolysis Bullosa;Chronic Skin Ulcer | Biological: REGE pro dressing | Egyptian Atomic Energy Authority | NULL | Completed | 12 Years | 45 Years | All | 8 | N/A | Egypt |
| 72 | 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 | ||
| 73 | 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 | ||
| 74 | NCT04520022 (ClinicalTrials.gov) | October 13, 2016 | 13/8/2020 | Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB | Single 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 Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Drug: Human Umbilical Cord Blood-derived Mesenchymal Stem Cells | Gangnam Severance Hospital | Daewoong Pharmaceutical Co. LTD. | Completed | 10 Years | 60 Years | All | 5 | Phase 1;Phase 2 | Korea, Republic of |
| 75 | 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 | ||
| 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 |
| 76 | NCT02654483 (ClinicalTrials.gov) | August 31, 2016 | 11/1/2016 | Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients | A Phase 2 Trial of Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Epidermolysis Bullosa Patients | Epidermolysis Bullosa;Pruritus | Drug: VPD-737;Other: Placebo | Jean Yuh Tang | Epidermolysis Bullosa Research Partnership;Menlo Therapeutics Inc. | Completed | 13 Years | N/A | All | 14 | Phase 2 | United States |
| 77 | NCT02793960 (ClinicalTrials.gov) | August 10, 2016 | 18/5/2016 | Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa | A Phase 1 Study to Evaluate the Safety, Pharmacokinetics and Therapeutic Effect of Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: topical BPM31510 3.0% Cream | Shasa Hu | Berg, LLC | Completed | 12 Years | N/A | All | 11 | Phase 1 | United States |
| 78 | 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 | ||
| 79 | 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 | ||
| 80 | 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 | ||
| 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 |
| 81 | NCT03016715 (ClinicalTrials.gov) | May 2016 | 9/1/2017 | Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study | A 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 Syndrome | Drug: Sirolimus 2%;Drug: Vehicle | Premier Specialists, Australia | NULL | Recruiting | 5 Years | N/A | All | 8 | Phase 2 | Australia |
| 82 | NCT02960997 (ClinicalTrials.gov) | May 2016 | 15/6/2016 | Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study | A 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 Syndrome | Drug: Sirolimus, 2%;Drug: Vehicle | Stanford University | NULL | Active, not recruiting | 4 Years | N/A | All | 8 | Phase 2 | United States |
| 83 | NCT02582775 (ClinicalTrials.gov) | March 2016 | 16/10/2015 | MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs | MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions | Epidermolysis Bullosa | Drug: Thymoglobulin;Drug: Cyclophosphamide;Drug: Fludarabine;Radiation: Total Body Irradiation;Procedure: Bone marrow infusion;Drug: Tacrolimus;Drug: Mycophenolate Mofetil;Biological: Donor mesenchymal stem cell infusions;Drug: Busulfan | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 25 Years | All | 84 | Phase 2 | United States |
| 84 | NCT02698735 (ClinicalTrials.gov) | February 25, 2016 | 19/2/2016 | Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients | Recessive Dystrophic Epidermolysis Bullosa | Drug: Gentamicin;Drug: Placebo | University of Southern California | NULL | Completed | N/A | N/A | All | 5 | Phase 1;Phase 2 | NULL | |
| 85 | 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 |
| 86 | NCT02493816 (ClinicalTrials.gov) | September 2015 | 21/5/2015 | Safety Study of Gene-modified Autologous Fibroblasts in Recessive Dystrophic Epidermolysis Bullosa | Phase I Study of Lentiviral-mediated COL7A1 Gene-modified Autologous Fibroblasts in Adults With Recessive Dystrophic Epidermolysis Bullosa. | Recessive Dystrophic Epidermolysis Bullosa | Drug: Gene-modified autologous fibroblasts | King's College London | University College, London | Completed | 17 Years | N/A | All | 5 | Phase 1 | United Kingdom |
| 87 | NCT02592954 (ClinicalTrials.gov) | September 2015 | 29/10/2015 | Effect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal Skin | Effect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal Skin | Epidermolysis Bullosa Simplex;Pachyonychia Congenita | Drug: Jojoba oil with broccoli sprout extract;Drug: Jojoba oil (placebo) | Johns Hopkins University | NULL | Completed | 18 Years | N/A | All | 5 | Phase 1 | United States |
| 88 | 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 | ||
| 89 | NCT02670330 (ClinicalTrials.gov) | June 9, 2015 | 28/7/2015 | Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in 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 | Epidermolysis Bullosa | Drug: SD-101-6.0 cream | Scioderm, Inc. | Amicus Therapeutics | Terminated | 1 Month | N/A | All | 152 | Phase 3 | United States;Australia;Austria;France;Germany;Israel;Lithuania;Netherlands;Poland;Serbia;Spain;United Kingdom;Italy |
| 90 | NCT02323789 (ClinicalTrials.gov) | June 2015 | 18/12/2014 | Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa | A Phase I/II Study Evaluating Allogeneic Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Drug: Mesenchymal stromal cells | King's College London | NULL | Active, not recruiting | 18 Years | 65 Years | All | 10 | Phase 1;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 |
| 91 | NCT02470689 (ClinicalTrials.gov) | June 2015 | 27/5/2015 | Diacerin for the Treatment of Epidermolysis Bullosa Simplex | Diacerin for the Treatment of Epidermolysis Bullosa Simplex | Epidermolysis Bullosa Simplex | Drug: Diacerin cream | Tel-Aviv Sourasky Medical Center | NULL | Not yet recruiting | 6 Years | 19 Years | Both | 50 | Phase 2 | NULL |
| 92 | EUCTR2014-005679-96-DE (EUCTR) | 22/05/2015 | 11/03/2015 | 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: 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] | 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 | |||
| 93 | 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 | ||
| 94 | 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 | ||
| 95 | 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 | ||
| 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 |
| 96 | NCT02384460 (ClinicalTrials.gov) | March 11, 2015 | 13/2/2015 | ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants 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 | Epidermolysis Bullosa | Drug: SD-101-6.0 cream;Drug: Placebo (SD-101-0.0) cream | Scioderm, Inc. | Amicus Therapeutics | Completed | 1 Month | N/A | All | 169 | Phase 3 | United States;Australia;Austria;France;Germany;Israel;Italy;Lithuania;Netherlands;Poland;Serbia;Spain;United Kingdom |
| 97 | EUCTR2014-004500-30-GB (EUCTR) | 13/02/2015 | 22/12/2014 | A 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] | King's College London | Guy's and St Thomas NHS Foundation Trust | Not Recruiting | Female: yes Male: yes | 10 | Phase 1;Phase 2 | United Kingdom | |||
| 98 | 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 | ||
| 99 | 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 | ||
| 100 | EUCTR2014-002288-14-GB (EUCTR) | 26/09/2014 | 11/07/2014 | 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] | 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 | |||
| 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 |
| 101 | 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 | ||
| 102 | NCT02090283 (ClinicalTrials.gov) | March 26, 2014 | 12/3/2014 | Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa | An Open Label Extension, Multi-Center, Study to Evaluate the Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: SD-101 dermal cream (6%) | Scioderm, Inc. | Amicus Therapeutics;Food and Drug Administration (FDA) | Terminated | 6 Months | N/A | All | 42 | Phase 2 | United States |
| 103 | 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 | ||
| 104 | NCT02014376 (ClinicalTrials.gov) | January 6, 2014 | 12/12/2013 | Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa | A Phase 2b, Multi-Center, Randomized, Double-Blind, Vehicle-Controlled Study of the Effectiveness and Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: SD-101 dermal cream (3%);Drug: SD-101 Dermal Cream (6%);Drug: Vehicle (SD-101 0%) | Scioderm, Inc. | Amicus Therapeutics | Completed | 6 Months | N/A | All | 48 | Phase 2 | United States |
| 105 | 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 | |||
| 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 |
| 106 | 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 | |||
| 107 | 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 | |||
| 108 | 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 | |||
| 109 | 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 | |||
| 110 | NCT01749306 (ClinicalTrials.gov) | December 2012 | 11/12/2012 | A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing | A 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 Bullosa | Epidermolysis Bullosa | Biological: ABH001;Other: Control wound treatment | Shire Regenerative Medicine, Inc. | NULL | Terminated | N/A | N/A | Both | 1 | Phase 3 | United States;Austria;Canada;France;Germany;Poland;Portugal;Spain |
| 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 |
| 111 | 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 | ||
| 112 | NCT01528306 (ClinicalTrials.gov) | March 2012 | 31/1/2012 | A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa | An Exploratory, Cross-Over Study of the Safety of HP802-247 Applied to Open Wounds of Subjects With Dystrophic Epidermolysis Bullosa | Dystrophic Epidermolysis Bullosa | Biological: HP802-247;Other: Placebo | Healthpoint | NULL | Withdrawn | 2 Years | N/A | Both | 0 | Phase 2 | United States |
| 113 | NCT01538862 (ClinicalTrials.gov) | February 2012 | 20/2/2012 | Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa | Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa | Dystrophic Epidermolysis Bullosa | Drug: Granulocyte Colony Stimulating Factor (GCSF) | Vanderbilt University Medical Center | NULL | Completed | N/A | N/A | All | 7 | Phase 2 | United States |
| 114 | NCT01340235 (ClinicalTrials.gov) | June 2011 | 11/4/2011 | Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin | Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin | Epidermolysis Bullosa | Drug: Oral erythromycin | Centre Hospitalier Universitaire de Nice | NULL | Recruiting | 6 Months | 8 Years | Both | 8 | Phase 3 | France |
| 115 | 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 |
| 116 | JPRN-UMIN000004428 | 2010/11/01 | 01/11/2010 | Exploratory research on effectiveness and safety of Rituximab treatment for steroid intractable pemphygus, bullous pemphigoid and epidermolysis bullosa acquisita. | pemphigus, bullous pemphigoid, epidermolysis bullosa acquisita | Rituximab | Research group of rare intractable dermatologial disorder | NULL | Complete: follow-up complete | 20years-old | Not applicable | Male and Female | 20 | Phase 2 | Japan | |
| 117 | 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 |
| 118 | NCT01294241 (ClinicalTrials.gov) | October 2010 | 27/10/2010 | Case Series Documentation of Patients With Epidermolysis Bullosa | 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 | Epidermolysis Bullosa Hereditaria | Drug: Sericare® | Birken AG | NULL | Completed | 1 Year | 95 Years | Both | 10 | Phase 2 | Germany |
| 119 | NCT00951964 (ClinicalTrials.gov) | October 2010 | 3/8/2009 | Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate) | Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate) | Epidermolysis Bullosa Dystrophica | Drug: Polyphenon E before Placebo;Drug: placebo before treatment | Centre Hospitalier Universitaire de Nice | NULL | Completed | 2 Years | N/A | Both | 18 | Phase 2 | France |
| 120 | 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 | ||||
| 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 |
| 121 | NCT01908088 (ClinicalTrials.gov) | July 2010 | 23/7/2013 | Autologous Transplantation of Cultured Fibroblast on Amniotic Membrane in Patients With Epidermolysis Bullosa | Autologous Transplantation of Cultured Fibroblast on Amniotic Membrane for Mitten Hand Deformity in Patients With Epidermolysis Bullosa | Epidermolysis Bullosa With Mitten Hands | Biological: Cell transplantation | Royan Institute | Hazrat Fatemeh Hospital | Completed | 5 Years | 25 Years | Both | 6 | Phase 1 | Iran, Islamic Republic of |
| 122 | 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 | ||||
| 123 | NCT01033552 (ClinicalTrials.gov) | January 2010 | 14/12/2009 | Biochemical Correction of Severe EB by Allo HSCT and Off-the-shelf MSCs | MT2009-09: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation and Off-the-shelf Mesenchymal Stem Cells | Epidermolysis Bullosa | Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Anti-thymocyte globulin;Drug: Cyclosporine A;Drug: Mycophenolate mofetil;Procedure: Mesenchymal stem cell transplantation;Radiation: Total body irradiation;Procedure: Bone marrow or umbilical cord blood (UCG) stem cell transplantation | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 25 Years | All | 75 | Phase 2 | United States |
| 124 | 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 | |||
| 125 | NCT00936533 (ClinicalTrials.gov) | May 2009 | 8/7/2009 | Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita | Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita - a Double-blind Placebo-controlled Phase II Proof of Concept Study | Epidermolysis Bullosa Simplex | Drug: Dysport® (Botulinumtoxin A (Btx A));Drug: Placebo | Uppsala University | NULL | Recruiting | 16 Years | N/A | Both | 40 | Phase 2 | Sweden |
| 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 |
| 126 | NCT00881556 (ClinicalTrials.gov) | March 2009 | 14/4/2009 | Allogeneic 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 Bullosa | Drug: Reduced Intensity Transplant conditioning | Columbia University | NULL | Active, not recruiting | N/A | 21 Years | Both | 20 | Phase 0 | United States |
| 127 | NCT00825565 (ClinicalTrials.gov) | February 2009 | 19/1/2009 | Study of Alwextin® Cream in Treating Epidermolysis Bullosa | Open-label, Pilot Study to Investigate the Safety and Tolerability of Alwextin 3.0% Cream in the Treatment of Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: Alwextin cream | Northwestern University | Ann & Robert H Lurie Children's Hospital of Chicago | Completed | 6 Months | N/A | All | 8 | Phase 2 | United States |
| 128 | NCT00478244 (ClinicalTrials.gov) | April 2007 | 23/5/2007 | Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa | Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Procedure: hematopoietic bone marrow transplantation | Masonic Cancer Center, University of Minnesota | NULL | Terminated | N/A | 25 Years | All | 7 | N/A | United States |
| 129 | NCT00987142 (ClinicalTrials.gov) | December 2006 | 29/9/2009 | Trial To Assess Efficacy Of A Chimeric Skin In Patients With Epidermolysys Bullosa | A 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 Bullosa | EPIDERMOLYSIS BULLOSA | Drug: CX501;Device: Occlusive non adherent dressing | Tigenix S.A.U. | NULL | Completed | N/A | N/A | All | 12 | Phase 2 | Spain |
| 130 | NCT00380640 (ClinicalTrials.gov) | September 2006 | 25/9/2006 | The Efficacy of Trimethoprim in Wound Healing of Patients With Epidermolysis Bullosa | The Efficacy of Trimethoprim in Wound Healing of Patients With Epidermolysis Bullosa: A Randomized, Double Blinded, Placebo Controlled, Cross-over Pilot Study | Epidermolysis Bullosa | Drug: Trimethoprim | The Hospital for Sick Children | NULL | Completed | N/A | 20 Years | All | 10 | Phase 2 | Canada |
| 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 |
| 131 | EUCTR2005-002329-30-ES (EUCTR) | 09/08/2006 | 15/03/2010 | ENSAYO 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 | ENSAYO 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: CX501 | CELLERIX, S.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Spain | |||
| 132 | NCT00336154 (ClinicalTrials.gov) | March 3, 2006 | 12/6/2006 | Study to Evaluate the Efficacy of Tetracycline in Epidermolysis Bullosa | Randomised Double Blind Crossover Placebo Controlled Study to Evaluate the Efficacy of Tetracycline in Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: tetracyclin | Rambam Health Care Campus | NULL | Withdrawn | 13 Years | 60 Years | All | 0 | Phase 4 | Israel |
| 133 | NCT00311766 (ClinicalTrials.gov) | February 2006 | 4/4/2006 | A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa | A Randomized, Double-Blind, Placebo-Controlled, Dose-Response Study of the Safety and Efficacy of Thymosin Beta 4 in the Treatment of Patients With Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: Thymosin Beta 4;Drug: Placebo | RegeneRx Biopharmaceuticals, Inc. | NULL | Terminated | 2 Years | N/A | All | 30 | Phase 2 | United States |
| 134 | NCT00231517 (ClinicalTrials.gov) | October 2005 | 3/10/2005 | Randomised Double Blind Placebo Controlled Cross Over Design of the Efficacy of Topical Morphine for Inflammatory Pain in Children With Epidermolysis Bullosa | Epidermylosis Bullosa | Drug: topical opiod;Drug: morphine sulphate in intrasite gel | Institute of Child Health | NULL | Completed | 4 Years | 18 Years | Both | Phase 4 | United Kingdom | ||
| 135 | NCT00014729 (ClinicalTrials.gov) | October 2000 | 10/4/2001 | Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: isotretinoin | University of North Carolina | NULL | Completed | 15 Years | N/A | Both | 20 | Phase 1 | 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 |
| 136 | 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 | |
| 137 | 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 | |||
| 138 | 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 | |||
| 139 | 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 | |||
| 140 | 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 | |||
| 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 |
| 141 | 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 | |||
| 142 | 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 | |||
| 143 | 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 | |||
| 144 | EUCTR2016-004427-24-FR (EUCTR) | 21/08/2017 | 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) | 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] | Castle Creek Pharmaceuticals, LLC | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | United States;France;Austria;Australia;Israel;Netherlands;Germany;United Kingdom | ||||
| 145 | 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 | |||
| 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 |
| 146 | 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 | |||
| 147 | 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 |