36. 表皮水疱症 ［臨床試験数：80，薬物数：118（DrugBank：31），標的遺伝子数：27，標的パスウェイ数：94］
Searched query = "Epidermolysis bullosa", "Kindler syndrome"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04140786 | October 31, 2019 | 11 November 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 | Recruiting | N/A | N/A | All | 6 | Phase 1/Phase 2 | United States | |
| 2 | NCT03786237 | September 2019 | 30 September 2019 | 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 Oral Liquid Solution / Rigosertib Intravenous | Prof. Johann Bauer | Recruiting | 18 Years | 79 Years | All | 12 | Phase 1/Phase 2 | Austria | |
| 3 | NCT03928093 | August 7, 2019 | 2 September 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 |
| 4 | NCT03578029 | May 22, 2019 | 9 September 2019 | 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 | Recruiting | 4 Years | N/A | All | 15 | Phase 2 | United States | |
| 5 | NCT03836001 | April 18, 2019 | 27 May 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 |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03529877 | January 16, 2019 | 30 September 2019 | 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 | Recruiting | 12 Months | 55 Years | All | 18 | Phase 1/Phase 2 | United States;Austria;France;Germany;Italy;United Kingdom |
| 7 | NCT03752905 | January 9, 2019 | 25 February 2019 | 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. | Recruiting | 18 Years | N/A | All | 14 | Phase 1/Phase 2 | United States | |
| 8 | EUCTR2018-001009-98-AT | 05/11/2018 | 28 February 2019 | 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 Pharmaceutical Form: Suspension for injection Current Sponsor code: T202-3 Other descriptive name: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells Concentration unit: million organisms/ml million organisms/millilitre Concentration type: equal Concentration number: 10- | RHEACELL GmbH & Co. KG | Authorised | Female: yes Male: yes | 18 | Phase 1;Phase 2 | France;United States;Austria;Germany;Italy;United Kingdom | |||
| 9 | NCT03468322 | October 20, 2018 | 29 April 2019 | 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. | Not recruiting | 2 Years | N/A | All | 9 | Phase 2 | Taiwan | |
| 10 | NCT03632265 | October 1, 2018 | 20 August 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 recruiting | 6 Years | N/A | All | 15 | Phase 3 | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT03605069 | July 2, 2018 | 26 August 2019 | Topical QR-313 in 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 RDEB Due to Mutation(s) in Exon 73 of the COL7A1 Gene | Epidermolysis Bullosa Dystrophica, Recessive | Drug: QR-313;Drug: Placebo | Wings Therapeutics Inc. | Recruiting | 6 Years | N/A | All | 8 | Phase 1/Phase 2 | United States;France;Spain | |
| 12 | NCT03526159 | July 1, 2018 | 25 June 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 | Not recruiting | N/A | N/A | All | 6 | Phase 1/Phase 2 | United States | |
| 13 | NCT03392909 | July 2018 | 25 June 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 | Not recruiting | 7 Years | N/A | All | 9 | Phase 1/Phase 2 | United States | |
| 14 | NCT04153630 | May 17, 2018 | 11 November 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 | Not recruiting | 12 Months | 18 Years | All | 9 | Phase 1/Phase 2 | Spain |
| 15 | NCT03472287 | May 10, 2018 | 25 February 2019 | Study of Diacerein After Maximum Use in Patients With 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 | Drug: CCP-020 (Diacerein 1% Ointment) | Castle Creek Pharmaceuticals, LLC | Recruiting | 6 Months | N/A | All | 20 | Phase 1 | United States;Netherlands;United Kingdom;France | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT03536143 | May 7, 2018 | 11 February 2019 | Topical Bercolagene Telserpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa | A Phase II Study of Bercolagene Telserpavec (KB103), a Non-Integrating, Replication-Incompetent Herpes Simplex Virus 1 (HSV-1) Vector Expressing the Human Collagen VII (COL7) Protein, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) | Dystrophic Epidermolysis Bullosa | Biological: Topical bercolagene telserpavec | Krystal Biotech, Inc. | Recruiting | 5 Years | N/A | All | 4 | Phase 2 | United States | |
| 17 | NCT03453632 | March 1, 2018 | 12 March 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 | Not recruiting | 18 Years | N/A | All | 25 | Phase 2/Phase 3 | France | |
| 18 | EUCTR2017-003757-41-AT | 04/01/2018 | 30 April 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 Pharmaceutical Form: Ointment INN or Proposed INN: DIACEREIN CAS Number: 13739-02-1 Current Sponsor code: CCP-020 Other descriptive name: Diacerein Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 1- | Castle Creek Pharmaceuticals, LLC | Authorised | Female: yes Male: yes | 84 | Phase 2 | France;United States;Australia;Austria;Israel;Netherlands;Germany;Italy;United Kingdom | |||
| 19 | EUCTR2018-000439-29-NL | 1 October 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 Pharmaceutical Form: Ointment INN or Proposed INN: DIACEREIN CAS Number: 13739-02-1 Current Sponsor code: CCP-020 Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 1- | Castle Creek Pharmaceuticals, LLC | Not Available | Female: yes Male: yes | 20 | Phase 1 | France;United States;Germany;Netherlands;United Kingdom | ||||
| 20 | EUCTR2016-003832-19-AT | 11/12/2017 | 12 November 2018 | 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 Pharmaceutical Form: Capsule, soft INN or Proposed INN: rigosertib CAS Number: 592542-60-4 Current Sponsor code: ON 01910.NA Other descriptive name: ON 01910.NA Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 70- Product Name: Rigosertib Product Code: ON 01910.Na Pharmaceutical Form: Capsule, soft INN or Proposed INN: rigosertib CAS Number: 592542-60-4 Current Sponsor code: ON 01910.NA Other descriptive name: ON 01910.NA Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 280- Product Name: Rigosertib Product Code: ON 01910.Na Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: rigosertib CAS Number: 592542-60-4 Current Sponsor code: ON 01910.NA Other descriptive name: ON 01910.NA Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 75- Product Name: Rigosertib Product Code: ON 01910.Na Pharmaceutical Form: Concentrate for oral solution INN or Proposed INN: rigosertib CAS Number: 592542-60-4 Current Sponsor code: ON 01910.NA Other descriptive name: ON 01910.NA Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 75- | Gemeinn. Salzburger Landeskliniken BetriebsGesmbH, University Hospital for Dermatology, EB-House Austria | Authorised | Female: yes Male: yes | 12 | Phase 2 | Austria | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT03389308 | December 1, 2017 | 2 July 2018 | 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 Simplex | Drug: Diacerein 1% Ointment | Castle Creek Pharmaceuticals, LLC | Recruiting | 6 Months | 99 Years | All | 80 | Phase 2 | United States | |
| 22 | EUCTR2017-000606-37-ES | 16/11/2017 | 26 February 2018 | 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 Pharmaceutical Form: Solution for infusion INN or Proposed INN: Células madre mesenquimales haploidénticas derivadas de médula ósea Current Sponsor code: MSCs Other descriptive name: MESENCHYMAL STEM CELLS (MSCS) Concentration unit: Other Concentration type: range Concentration number: 2000000-4000000 | Fundación para la Investigación Biomédica Hospital Universitario La Paz | Authorised | Female: yes Male: yes | 9 | Phase 1 | Spain | |||
| 23 | EUCTR2016-004427-24-NL | 06/09/2017 | 28 February 2019 | 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 Pharmaceutical Form: Ointment INN or Proposed INN: DIACEREIN CAS Number: 13739-02-1 Current Sponsor code: CCP-020, AC-203 Other descriptive name: Diacerein Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 1- Pharmaceutical form of the placebo: Ointment Route of administration of the placebo: Topical use (Noncurrent) | Castle Creek Pharmaceuticals, LLC | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | France;United States;Austria;Australia;Israel;Germany;Netherlands;Italy;United Kingdom | |||
| 24 | EUCTR2016-004427-24-DE | 24/08/2017 | 22 October 2018 | 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 Pharmaceutical Form: Ointment INN or Proposed INN: DIACEREIN CAS Number: 13739-02-1 Current Sponsor code: CCP-020, AC-203 Other descriptive name: Diacerein Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 1- Pharmaceutical form of the placebo: Ointment Route of administration of the placebo: Topical use (Noncurrent) | Castle Creek Pharmaceuticals, LLC | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | France;United States;Austria;Australia;Israel;Netherlands;Germany;Italy;United Kingdom | |||
| 25 | EUCTR2016-000095-17-AT | 17/08/2017 | 2 October 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 Pharmaceutical Form: Living tissue equivalent INN or Proposed INN: Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL17A1-encoding retroviral vector Current Sponsor code: Hologene17 DS Concentration unit: % percent Concentration type: range Concentration number: 50-100 | Holostem Terapie Avanzate s.r.l. | Authorised | Female: yes Male: yes | Phase 1;Phase 2 | Austria | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | NCT03183934 | July 2017 | 16 December 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. | Not recruiting | 2 Years | 60 Years | All | 5 | N/A | Korea, Republic of | |
| 27 | NCT03154333 | June 1, 2017 | 5 November 2018 | Safety and Efficacy of Diacerein 1% Ointment Topical Formulation Compared to Placebo for Subjects With Epidermolysis Bullosa Simplex (EBS) | An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex (EBS) [DELIVERS Study] | Epidermolysis Bullosa Simplex | Drug: Diacerein 1% Ointment Topical Formulation;Drug: Vehicle | Castle Creek Pharmaceuticals, LLC | Not recruiting | 4 Years | N/A | All | 80 | Phase 2 | United States;Australia;Austria;France;Germany;Israel;Netherlands;United Kingdom | |
| 28 | EUCTR2016-002066-32-GB | 24/05/2017 | 24 July 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 Pharmaceutical Form: Gel INN or Proposed INN: Birch bark extract CAS Number: 1640971-03-4 Current Sponsor code: Dry extract from betulae cortex Other descriptive name: BIRCH BARK EXTRACT Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Gel Route of administration of the placebo: Cutaneous use | Amryt Research Limited | Authorised | Female: yes Male: yes | 164 | Phase 3 | Turkey;Austria;Israel;Italy;United Kingdom;Switzerland;France;Mexico;Croatia;Australia;Germany;Ireland;Spain;Greece;United States | |||
| 29 | NCT03068780 | March 29, 2017 | 4 November 2019 | 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 | Recruiting | N/A | N/A | All | 250 | Phase 3 | United States;Argentina;Australia;Austria;Brazil;Chile;Colombia;Croatia;Czechia;Denmark;France;Georgia;Germany;Greece;Hong Kong;Hungary;Ireland;Israel;Italy;Romania;Russian Federation;Serbia;Singapore;Spain;Switzerland;Ukraine;United Kingdom;Belgium | |
| 30 | NCT03730584 | February 27, 2017 | 6 May 2019 | 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 | Recruiting | N/A | 21 Years | All | 10 | N/A | France | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | NCT03012191 | February 2, 2017 | 26 August 2019 | 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 | Not recruiting | N/A | N/A | All | 6 | Phase 1/Phase 2 | United States | |
| 32 | NCT03942250 | January 2017 | 15 July 2019 | 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 | Not recruiting | 12 Years | 45 Years | All | 8 | N/A | Egypt | |
| 33 | EUCTR2017-004806-17-FR | 23 July 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 Pharmaceutical Form: Gel INN or Proposed INN: unavailable CAS Number: unavailable Current Sponsor code: QR-313 Concentration unit: mg/g milligram(s)/gram Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Gel Route of administration of the placebo: Cutaneous use | ProQR Therapeutics | Not Available | Female: yes Male: yes | 14 | Phase 1;Phase 2 | United States;France;Czech Republic;Canada;Austria;Germany;United Kingdom | ||||
| 34 | NCT02654483 | August 31, 2016 | 12 November 2018 | 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 | Other: Placebo;Drug: VPD-737 | Jean Yuh Tang | Epidermolysis Bullosa Research Partnership;Menlo Therapeutics Inc. | Not recruiting | 13 Years | N/A | All | 14 | Phase 2 | United States |
| 35 | NCT02793960 | August 10, 2016 | 22 July 2019 | 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 | Not recruiting | 12 Years | N/A | All | 11 | Phase 1 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | EUCTR2016-001967-35-AT | 19/07/2016 | 10 December 2018 | 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 Pharmaceutical Form: Ointment INN or Proposed INN: CALCIPOTRIOL CAS Number: 112828-00-9 Concentration unit: µg microgram(s) Concentration type: equal Concentration number: 0.05- Pharmaceutical form of the placebo: Ointment Route of administration of the placebo: Topical use (Noncurrent) | Universitätsklinik für Dermatologie, Paracelsus medizinische Privatuniversität Salzburg | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Austria | |||
| 37 | EUCTR2015-004592-74-AT | 01/06/2016 | 11 June 2018 | 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 Pharmaceutical Form: Living tissue equivalent INN or Proposed INN: Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL7A1-encoding retroviral vector Current Sponsor code: Hologene7 DS Other descriptive name: Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL7A1-encoding retroviral vector, obtained from secondary culture of ex vivo expanded autologous human keratinocytes. Concentration unit: % percent Concentration type: range Concentration number: 50-100 | Holostem Terapie Avanzate s.r.l. | Authorised | Female: yes Male: yes | 12 | Phase 1;Phase 2 | Austria | |||
| 38 | EUCTR2014-004884-19-GB | 24/05/2016 | 20 August 2018 | 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 Pharmaceutical Form: Suspension for injection in pre-filled syringe | King's College London | Guy's and St Thomas' NHS Foundation Trust | Not Recruiting | Female: yes Male: yes | 10 | Phase 1 | United Kingdom | ||
| 39 | NCT02960997 | May 2016 | 7 October 2019 | 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 | Not recruiting | 4 Years | N/A | All | 8 | Phase 2 | United States | |
| 40 | NCT03016715 | May 2016 | 16 December 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 | Recruiting | 5 Years | N/A | All | 8 | Phase 2 | Australia | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 41 | NCT02582775 | March 2016 | 8 April 2019 | 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 | Recruiting | N/A | 25 Years | All | 84 | Phase 2 | United States | |
| 42 | NCT02698735 | February 25, 2016 | 26 August 2019 | Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients | Recessive Dystrophic Epidermolysis Bullosa | Drug: Gentamicin;Drug: Placebo | University of Southern California | Not recruiting | N/A | N/A | All | 5 | Phase 1/Phase 2 | |||
| 43 | NCT02493816 | September 2015 | 7 October 2019 | 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 | Not recruiting | 17 Years | N/A | All | 5 | Phase 1 | United Kingdom |
| 44 | NCT02592954 | September 2015 | 16 December 2017 | 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 | Not recruiting | 18 Years | N/A | All | 5 | Phase 1 | United States | |
| 45 | NCT02670330 | June 9, 2015 | 30 September 2019 | 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 | Not recruiting | 1 Month | N/A | All | 152 | Phase 3 | United States;Australia;Austria;France;Germany;Israel;Lithuania;Netherlands;Poland;Serbia;Spain;United Kingdom;Italy |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 46 | NCT02323789 | June 2015 | 16 December 2017 | 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 | Not recruiting | 18 Years | 65 Years | All | 10 | Phase 1/Phase 2 | United Kingdom | |
| 47 | NCT02470689 | June 2015 | 29 June 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 | Not recruiting | 6 Years | 19 Years | Both | 50 | Phase 2 | ||
| 48 | EUCTR2014-005679-96-AT | 16/03/2015 | 24 September 2018 | 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 Pharmaceutical Form: Cream INN or Proposed INN: ALLANTOIN CAS Number: 97-59-6 Other descriptive name: ALLANTOIN Concentration unit: % (W/V) percent weight/volume Concentration type: equal Concentration number: 6- | Scioderm, INC | 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 | |||
| 49 | EUCTR2014-004500-30-GB | 13/02/2015 | 30 April 2019 | 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] | Product Name: Allogeneic mesenchymal stromal cells Product Code: TC-MSC Pharmaceutical Form: Injection | 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 | ||
| 50 | EUCTR2014-002288-14-GB | 26/09/2014 | 28 February 2019 | 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 Pharmaceutical Form: Cream INN or Proposed INN: ALLANTOIN CAS Number: 97-59-6 Other descriptive name: ALLANTOIN Concentration unit: % (W/V) percent weight/volume Concentration type: equal Concentration number: 6- Pharmaceutical form of the placebo: Cream Route of administration of the placebo: Topical use (Noncurrent) | Scioderm, An Amicus Therapeutics Company | 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_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 51 | EUCTR2014-002288-14-AT | 08/08/2014 | 2 October 2017 | 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 Pharmaceutical Form: Cream INN or Proposed INN: ALLANTOIN CAS Number: 97-59-6 Other descriptive name: ALLANTOIN Concentration unit: % (W/V) percent weight/volume Concentration type: equal Concentration number: 6- Pharmaceutical form of the placebo: Cream Route of administration of the placebo: Topical use (Noncurrent) | Scioderm, An Amicus Therapeutics Company | 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 | |||
| 52 | NCT02090283 | March 26, 2014 | 11 November 2019 | 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) | Not recruiting | 6 Months | N/A | All | 42 | Phase 2 | United States |
| 53 | EUCTR2012-000605-72-NL | 06/02/2014 | 10 September 2018 | 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 Pharmaceutical Form: Infusion INN or Proposed INN: Mesenchymal stromal cells Current Sponsor code: TC-MSC Concentration unit: thousand organisms/ml thousand organisms/millilitre Concentration type: range Concentration number: 1000-2000 Trade Name: Busilvex Pharmaceutical Form: Concentrate for solution for infusion Trade Name: Fludarabine Product Name: FLudarabine Pharmaceutical Form: Concentrate for solution for injection/infusion Trade Name: THYMOGLOBULINE Product Name: Thymoglobuline Pharmaceutical Form: Powder and solvent for solution for infusion | Universitair Medisch Centrum Utrecht | Not Recruiting | Female: yes Male: yes | 11 | Phase 2 | Netherlands | |||
| 54 | NCT02014376 | January 2014 | 16 December 2017 | Study of Effectiveness and Safety of SD-101 in Subjects 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: 3% SD-101 dermal cream;Drug: 6% SD-101 dermal cream;Drug: Vehicle (0% SD-101) | Scioderm, Inc. | Not recruiting | 6 Months | N/A | All | 48 | Phase 2 | United States | |
| 55 | EUCTR2013-002034-21-AT | 16/12/2013 | 13 March 2017 | 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 Pharmaceutical Form: Cream INN or Proposed INN: DIACEREIN CAS Number: 13739-02-1 Concentration unit: g gram(s) Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Cream Route of administration of the placebo: Topical use (Noncurrent) | EB House Austria | Not Recruiting | Female: yes Male: yes | Phase 3 | Austria | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 56 | EUCTR2012-001815-21-ES | 18/01/2013 | 21 October 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: 15.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Name: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh Product Code: ABH001 Pharmaceutical Form: Living tissue equivalent INN or Proposed INN: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh Current Sponsor code: ABH001 Other descriptive name: Human fibroblast-derived dermal substitute, Dermagraft Concentration unit: U unit(s) Concentration type: equal Concentration number: 1- | Shire Regenerative Medicine, Inc. | Not Recruiting | Female: yes Male: yes | 25 | Portugal;United States;Spain;Austria;Australia;Germany | ||||
| 57 | NCT01528306 | March 2012 | 19 February 2015 | 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 | Not recruiting | 2 Years | N/A | Both | 0 | Phase 2 | United States | |
| 58 | NCT01538862 | February 2012 | 16 December 2017 | 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 | Not recruiting | N/A | N/A | All | 7 | Phase 2 | United States | |
| 59 | NCT01340235 | June 2011 | 19 February 2015 | 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 | Recruiting | 6 Months | 8 Years | Both | 8 | Phase 3 | France | |
| 60 | EUCTR2010-023121-38-GB | 13/01/2011 | 19 March 2012 | 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 Pharmaceutical Form: Injection INN or Proposed INN: Fibroblasts Current Sponsor code: ICX-DEBRHY-2 Concentration unit: ml millilitre(s) Concentration type: equal Pharmaceutical form of the placebo: Injection Route of administration of the placebo: Intradermal use | Intercytex Ltd | Authorised | Female: yes Male: yes | 25 | Phase 2 | United Kingdom | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 61 | JPRN-UMIN000004428 | 2010/11/01 | 2 April 2019 | 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 | Not Recruiting | 20years-old | Not applicable | Male and Female | 20 | Phase 2 | Japan | ||
| 62 | NCT01263379 | October 5, 2010 | 30 September 2019 | 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 | Not recruiting | 13 Years | N/A | All | 10 | Phase 1/Phase 2 | United States |
| 63 | NCT00951964 | October 2010 | 19 February 2015 | 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 | Not recruiting | 2 Years | N/A | Both | 18 | Phase 2 | France | |
| 64 | NCT01294241 | October 2010 | 19 February 2015 | 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 | Not recruiting | 1 Year | 95 Years | Both | 10 | Phase 2 | Germany | |
| 65 | EUCTR2010-019945-24-DE | 24/09/2010 | 19 March 2012 | 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 Pharmaceutical Form: Cutaneous suspension INN or Proposed INN: triterpene dry extract from birch cork Current Sponsor code: TE Concentration unit: % percent Concentration type: equal Concentration number: 10- | Birken GmbH | Not Recruiting | Female: yes Male: yes | Germany | |||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 66 | NCT01908088 | July 2010 | 19 February 2015 | 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 | Not recruiting | 5 Years | 25 Years | Both | 6 | Phase 1 | Iran, Islamic Republic of |
| 67 | EUCTR2009-012750-21-FR | 20/01/2010 | 19 March 2012 | Traitement des épidermolyses bulleuses dystrophiques héréditaires par l’épigallocatéchine-3-gallate oral (Polyphenon E®) | Traitement des épidermolyses bulleuses dystrophiques héréditaires par l’épigallocatéchine-3-gallate oral (Polyphenon E®) | Dystrophic epidermolysis bullosa hereditaria MedDRA version: 12.0 Level: LLT Classification code 10056508 Term: Acquired epidermolysis bullosa | Trade Name: POLYPHENON E Product Name: POLYPHENON E Pharmaceutical Form: Capsule* Pharmaceutical form of the placebo: Capsule* Route of administration of the placebo: Oral use | CHU de NICE | Authorised | Female: yes Male: yes | France | |||||
| 68 | NCT01033552 | January 2010 | 7 October 2019 | 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 | Recruiting | N/A | 25 Years | All | 75 | Phase 2 | United States | |
| 69 | EUCTR2010-024428-10-FR | 2 October 2017 | 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 Pharmaceutical Form: Granules for syrup Trade Name: erythromycine Product Name: erythrocine oral Pharmaceutical Form: Granules for syrup | CHU de NICE | Not Available | Female: yes Male: yes | 8 | Phase 2 | France | ||||
| 70 | EUCTR2009-010763-17-SE | 26/05/2009 | 19 March 2012 | 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 Pharmaceutical Form: Powder for solution for injection Pharmaceutical form of the placebo: Powder for solution for injection Route of administration of the placebo: Subcutaneous use | Sophiahemmet | Authorised | Female: yes Male: yes | Phase 2 | Sweden | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 71 | NCT00881556 | March 2009 | 19 February 2015 | 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 | Not recruiting | N/A | 21 Years | Both | 20 | Phase 0 | United States | |
| 72 | NCT00825565 | February 2009 | 16 December 2017 | 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 | Not recruiting | 6 Months | N/A | All | 8 | Phase 2 | United States |
| 73 | NCT00478244 | April 2007 | 19 October 2017 | 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 | Not recruiting | N/A | 25 Years | All | 7 | N/A | United States | |
| 74 | NCT00987142 | December 2006 | 26 August 2019 | 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. | Not recruiting | N/A | N/A | All | 12 | Phase 2 | Spain | |
| 75 | NCT00380640 | September 2006 | 11 June 2018 | 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 | Not recruiting | N/A | 20 Years | All | 10 | Phase 2 | Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 76 | NCT00336154 | June 2006 | 19 February 2015 | Randomised Double Blind Crossover Placebo Controlled 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 | Not recruiting | 13 Years | 60 Years | Both | 20 | Phase 4 | Israel | |
| 77 | NCT00311766 | February 2006 | 19 October 2017 | 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. | Not recruiting | 2 Years | N/A | All | 30 | Phase 2 | United States | |
| 78 | NCT00231517 | October 2005 | 19 February 2015 | 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 | Not recruiting | 4 Years | 18 Years | Both | Phase 4 | United Kingdom | |||
| 79 | NCT00014729 | October 2000 | 7 April 2015 | Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: isotretinoin | University of North Carolina | Not recruiting | 15 Years | N/A | Both | 20 | Phase 1 | |||
| 80 | NCT00004359 | February 1996 | 19 February 2015 | Phase II Pilot Study of Extracorporeal Phototherapy for Epidermolysis Bullosa Acquisita | Epidermolysis Bullosa Acquisita | Drug: methoxsalen | National Center for Research Resources (NCRR) | Northwestern University | Not recruiting | 18 Years | N/A | Both | 10 | Phase 2 |