The Latest on EB Clinical Research

According to the U.S. National Institutes of Health (NIH), there are currently ten active EB research studies in the U.S. and abroad still in the recruiting stage. Each study focuses on a specific goal in the treatment of epidermolysis bullosa, such as testing the effectiveness of a new or modified medication or implementing a procedure such as stem-cell transplantation. The methods and organizations behind the studies may vary, but ultimately we hope that each of these studies will offer EB patients and their families some answers in the future.

If you are planning to participate in a clinical study, be sure to get all of the facts regarding eligibility and the trial’s ongoing requirements beforehand. And if you’ve participated in a study or are currently taking part and would like to share your own feedback about the experience, we’d love to hear from you. Just email us at

Here is some information about some of the trials currently recruiting in the U.S. A few of the universities referenced below who are currently involved in these clinical studies, such as Stanford and the University of Minnesota, have additional EB-focused studies going on simultaneously. Click here for the complete list of all EB studies both in the U.S. and internationally that are currently recruiting participants.

  • Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa. Dr. Alfred T. Lane of Stanford University’s School of Medicine heads up the study, one of the most recently active trials, in collaboration with the National Institute of Arthritis and Musculoskeletal and Skin Diseases. In this Phase 1 study, launched last December, doctors will create a graft of each participating RDEB patient’s skin and attempt to correct the cellular protein deficiency which causes RDEB in a culture, then transplant the corrected cells back onto the patient’s skin. Click here to learn more about this study at
  • Allogeneic Hematopoietic Stem Cell Transplant for Epidermolysis Bullosa. The team at the Masonic Cancer Center at the University of Minnesota is testing the safety and effectiveness of stem cell infusion for treatments of RDEB, based on animal models that have shown that stem cells can home in on the skin and repair biochemical and structural abnormalities associated with RDEB and its collagen 7 deficiency. Click here for more details about this study from
  • Effect of Thymosin Beta 4 on Wound Healing in Patients with Epidermolysis Bullosa. RegeneRx Biopharmaceuticals, Inc. is continuing to recruit Junctional EB and Dystrophic EB patients across ten locations nationwide for this study slated to continue through end of this year. The trial is designed to test the safety and tolerability of the Thymosin Beta 4 treatment, administered topically, as well as its lesion healing effectiveness. Click here to learn more about this study from
  • Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa. The team behind this Columbia University-sponsored study is seeking qualified patients to participate in trials at three locations. The study is trying to assess the event-free survival and overall survival for RDEB patients following Reduced Intensity Condition consisting of busulfan/flurdarabine/alemtuzumad (BFA) and Allogeneic Stem Cell Transplantation (ALLOSCT). To learn more about this study and its eligibility requirement, click here.

DebRA U.K., together with Intercytex and King’s College London, also announced the start of a Phase II trial with the Intercytex therapy ICX-RHY to treat skin erosions on patients diagnosed with severe RDEB. ICX-RHY is a suspension of human dermal fibroblasts (HDFs, which are naturally derived skin cells) in cell storage medium, for injection into the skin. The newly introduced HDFs are thought to repair the extracellular matrix to improve skin structure and function. Click here to read more about this recent news. As we learn more about the status of this clinical study and patient recruitment, we’ll be sure to include in future updates.


Epidermolysis Bullosa, Open Studies (U.S. National Institutes of Health)

Research Funding Announcement from DebRA U.K.

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