36. Epidermolysis bullosa Clinical trials / Disease details
Clinical trials : 160 / Drugs : 195 - (DrugBank : 47) / Drug target genes : 50 - Drug target pathways : 124
| 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 | NCT05111600 (ClinicalTrials.gov) | March 2022 | 1/9/2021 | Open-label, Pivotal Clinical Trial to Confirm Efficacy and Safety of Autologous Grafts Containing Stem Cells Genetically Modified for Epidermis Restoration in Patients With Junctional Epidermolysis 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 | Junctional Epidermolysis Bullosa Non-Herlitz Type | Drug: Transplantation | Holostem Terapie Avanzate s.r.l. | IRCCS San Raffaele;University of Modena and Reggio Emilia | Not yet recruiting | 6 Months | 65 Years | All | 6 | Phase 2/Phase 3 | France;Germany;Italy |
| 2 | 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 |
| 3 | 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 | ||
| 4 | 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 |
| 5 | 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 |
| 6 | NCT00881556 (ClinicalTrials.gov) | August 20, 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: Palifermin;Drug: Fludarabine;Drug: Busulfan;Drug: Lorazepam;Drug: Alemtuzumab;Drug: Tacrolimus | Columbia University | NULL | Terminated | N/A | 21 Years | All | 3 | Early Phase 1 | United States |
| 7 | 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 |