A variety of clinical trials are currently recruiting participants or already in progress that could ultimately benefit the EB community with its findings. Using 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 20 open, international, EB clinical research trials listed as active, with four of the studies not yet recruiting and four of the other international studies listed with unknown recruiting status. Of the remaining 12 EB research studies which are open and recruiting, seven of them are being run within the United States and five are active in other internationally-based locations.
The following summary spotlights key background information related to the seven 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.
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 June 2016, this study is currently recruiting participants in various locations across the U.S. including the University of Missouri (St. Louis, MO), Stanford University Dept. of Dermatology (Redwood City, CA), 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), Seattle Children's (Seattle, WA), 'Specially for Children (Austin, TX) and Texas Dermatology and Laser Specialists (San Antonio, TX), as well as in France, Italy, Germany, the Netherlands, Austria, Poland and the U.K. 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. First launched in November 2009 and last verified in March 2016, 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.
Stem Cell Transplant for Epidermolysis Bullosa. Launched in January 2010 and last verified in May 2016, 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.
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa. This study launched in December 2010, led by principal investigator and associate professor at Stanford University Jean Yuh Tang, and was last verified in March 2016. The interventional 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.
Effect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal Skin. Launched in September 2015 and last verified in October 2015, this study led by Johns Hopkins University (Baltimore, MD) features adult participants who will apply a broccoli sprout extract-jojoba oil compound to one arm every night under occlusion for a week with Jojoba oil alone applied to the other arm. At the end of that week, a 6mm punch biopsy will be taken from both arms and analyzed via polymerase chain reaction (PCR) and immunohistochemistry for differences in skin proteins. For more information about this study, click here to link to ClinicalTrials.gov.
MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs. As one of the newest studies launched in March 2016 and last verified in May 2016, this study by the Masonic Cancer Center at the University of Minnesota is a phase II study established to determine the event-free survival at 1-year following an allogeneic transplant and serial mesenchymal stem cell (MSC) infusions from a related donor or matched unrelated donor for the biochemical correction of severe EB. For more information about this study, click here to link to ClinicalTrials.gov.
- A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB). This study, launched and verified in June 2016, is led by Fibrocell Technologies and administered at Stanford University (Stanford, CA). The purpose of the study is to evaluate the safety of FCX-007, C7 expression and the presence of anchoring fibrils resulting from FCX-007 and to analyze wound healing as a result of FCX-007 administration in subjects with RDEB. For more information about this study, click here to link to ClinicalTrials.gov.