A number of clinical trials are presently recruiting or already in progress that may have a tremendous impact on the EB community. By accessing the U.S. National Institutes of Health (NIH) website, we are able to offer a useful summary of these research trials that have been logged by the NIH.
Currently, there are 12 open, international, EB clinical research trials listed as active, with three of the studies not yet recruiting and three of the other international studies listed with unknown recruiting status. Of the remaining six EB research studies which are open and recruiting, four of them are being run within the United States and two are active in other internationally-based locations.
The following summary spotlights key background information related to the four open and recruiting U.S. studies. For the complete, up-to-date details about each of the EB studies open and recruiting here in the U.S. as well as internationally, please visit the NIH’s Clinical Trials page here: http://clinicaltrials.gov/ct2/results?term=epidermolysis+bullosa&recr=Open.
The following is not an endorsement but a brief summary of EB clinical trial background information available at this time and is strictly for informational purposes only. For more information, visit the links provided. You should also consult your health care professional and thoroughly understand the potential risks involved with a clinical trial before you participate in any clinical trial. We take no responsibility for any results or outcomes associated with the following clinical trials.
- Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa. Led by principal investigator and associate professor at Stanford University Jean Yuh Tang, this study launched in December 2010 and was last verified in February 2015. The study is a collaboration between the university and the National Institute of Arthritis and Musculoskeletal and Skin Diseases. During the study, researchers create a graft of the participant’s skin that has been genetically engineered in a culture to attempt correcting the cellular protein deficiency that led to the RDEB. Investigators then transplant the “corrected” cells back onto the patient’s skin. For more information about this study, click here to link to ClinicalTrials.gov.
- Stem Cell Transplant for Epidermolysis Bullosa. Last verified in April 2015, this study by the Masonic Cancer Center at the University of Minnesota attempts to test a primary hypothesis that the infusion of bone marrow or umbilical cord blood from a healthy donor will correct a collagen, laminin, integrin or plakin deficiency and reduce the skin’s fragility caused by severe forms of EB. A secondary hypothesis also examines the impact of mesenchymal stem cells from a healthy donor on the safety and efficacy of an allogeneic hematopoietic stem cell transplant. For more information about this study, click here to link to ClinicalTrials.gov.
Characteristics of Patients with Dystrophic Epidermolysis Bullosa. Jean Yuh Tang at Stanford University is also conducting a strictly observational study by screening subjects with DEB to evaluate the characteristics of these patients, who may develop severely painful blistering and open wounds. Last verified in February 2015, this trial involves the study of cells to support the development of future therapy strategies. For more information about this study, click here to link to ClinicalTrials.gov.
- Study of Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa. Sponsored by Scioderm, Inc., the target of this study is to evaluate the efficacy and safety of SD-101-6.0 cream versus a placebo in the treatment of EB. Launched in November 2009 and last verified in May 2015, this study is currently recruiting participants in various locations across the U.S. including the University of Colorado School of Medicine (Aurora, CO), Ann & Robert H. Lurie Children's Hospital of Chicago (Chicago, IL), Washington University School of Medicine (St. Louis, MO), University of North Carolina School of Medicine (Chapel Hill, NC), Cincinnati Children's Hospital Medical Center (Cincinnati, OH), and Texas Dermatology and Laser Specialists (San Antonio, TX), as well as in France, Italy, Austria, and the U.K. For more information about this study, click here to link to ClinicalTrials.gov.