Latest EB Clinical Trials

The U.S. National Institutes of Health (NIH) currently reflects eleven EB clinical trials that are listed as “recruiting.” These include seven active EB research studies in the U.S. and four clinical trials abroad. We have compiled brief summaries highlighting specific aspects of the U.S. studies below. For complete information about all eleven of the EB studies recruiting internationally, visit the NIH’s Clinical Trials page by clicking here.

  • Allogeneic Hematopoietic Stem Cell Transplant for Epidermolysis Bullosa. Led by the team at the Masonic Cancer Center at the University of Minnesota, this trial is testing the safety and effectiveness of stem cell infusion for treatments of RDEB. Results based on animal models have indicated that stem cells may be able to home in on the skin and repair biochemical and structural abnormalities associated with RDEB and its collagen 7 deficiency. For complete information about this study, click here to link to ClinicalTrials.gov.
  • Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa. Dr. Alfred T. Lane of Stanford University’s School of Medicine is the primary investigator for this study, which first launched in December 2010. The trial, being conducted in collaboration with the National Institute of Arthritis and Musculoskeletal and Skin Diseases, involves creating a graft of the participant’s skin to attempt to correct the cellular protein deficiency that causes RDEB in a culture. Once this is completed, doctors then transplant the “corrected” cells back onto the patient’s skin. For complete information about this study, click here to link to ClinicalTrials.gov.
  • Stem Cell Transplant for Epidermolysis Bullosa. This study, entering Phase 2 of research by the Masonic Cancer Center at the University of Minnesota, is attempting to test its main hypothesis that the infusion of bone marrow or umbilical cord blood from a healthy donor can correct the cellular protein deficiency of several severe forms of EB and that this infusion may reduce skin fragility in participants. The study is also testing a secondary hypothesis that mesenchymal stem cells from a healthy donor will enhance the safety and effectiveness of the allogeneic hematopoietic stem cell transplant to serve as a renewable cell source to treat specific areas of blistering. For complete information about this study, click here to link to ClinicalTrials.gov.
  • Characteristics of Patients with Dystrophic Epidermolysis Bullosa. Stanford University is also conducting a screening of subjects with DEB to evaluate the characteristics of the patients, who usually develop severely painful blistering and open wounds, as well as their cells as a means to support the development of future therapies. For complete information about this study, click here to link to ClinicalTrials.gov.
  • Characteristics of Adult Patients with Recessive Dystrophic Epidermolysis Bullosa. Similarly, Stanford University is conducting a screening to evaluate characteristics of patients diagnosed with RDEB who survive to adulthood. For complete information about this study, click here to link to ClinicalTrials.gov.
  • Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa. This Columbia University-sponsored study continues to seek qualified patients at three locations, including The Children’s Hospital in Aurora, CO, Children’s Memorial Hospital in Chicago, IL, and Morgan Stanley Children’s Hospital of NYP in New York City. The study is trying to determine the event-free survival and overall survival for RDEB patients after undergoing Reduced Intensity Conditioning by a combination of busulfan/flurdarabine/alemtuzumad (BFA) and Allogeneic Stem Cell Transplantation (ALLOSCT). For complete information about this study, click here to link to ClinicalTrials.gov.
  • Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes. One of the most recently launched clinical trials that began in October of last year, this study led by Dr. Lane and the Stanford University team is listed as open to EB patients 18 and older (simplex, junctional or dystrophic). The trial will attempt to culture cells from EB patients’ non-fragile skin patches and graft them on to wounded areas for the same patient. For complete information about this study, click here to link to ClinicalTrials.gov.