EB Clinical Trial Updates

Throughout the year, we like to keep the EB community informed about current clinical research trials in place. According to the U.S. National Institutes of Health (NIH) website, there are presently 12 open EB clinical research trials listed as actively recruiting participants.

One study is not yet recruiting and the status of two studies is currently labeled unknown. Of those open, recruiting EB research studies, eight studies are taking place in the U.S. and four are being led outside of the U.S.

The following briefs highlight general aspects of the U.S. studies currently recruiting. For the latest detailed information about all EB studies open and recruiting here in the U.S. and abroad, please visit the NIH’s Clinical Trials page at the following link: http://clinicaltrials.gov/ct2/results?term=epidermolysis+bullosa&recr=Open.

The following is not an endorsement but merely a brief summary of the EB clinical trials available at this time and is for informational purposes only. You should visit the relevant link below for more information. 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. Dr. Alfred T. Lane of Stanford University’s School of Medicine leads this study first launched in December 2010. The trial is being conducted in collaboration with the National Institute of Arthritis and Musculoskeletal and Skin Diseases. This study involves researchers creating a graft of the participant’s skin that has been genetically engineered in a culture, in an attempt to correct its cellular protein deficiency causing 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. This study by the Masonic Cancer Center at the University of Minnesota, is attempting to test its primary hypothesis that the infusion of bone marrow or umbilical cord blood from a healthy donor will correct certain deficiencies and reduce the skin fragility characteristic of several severe forms of EB. EB patients are being treated with a combination of a chemotherapy regimen, a bone marrow or umbilical cord blood stem cell transplant and a mesenchymal stem cell transplant. For more information about this study, click here to link to ClinicalTrials.gov.
     
  • Characteristics of Patients with Dystrophic Epidermolysis Bullosa. In addition to its gene transfer study for RDEB patients, Stanford University is conducting a screening of subjects with DEB to evaluate the characteristics of these patients, who often develop severely painful blistering and open wounds, and examine their cells as a means to support the development of future therapies. For more information about this study, click here to link to ClinicalTrials.gov.
     
  • A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients with Epidermolysis Bullosa Who Have Wounds That Are Not Healing. Launched in December 2012, this trial by Shire Regenerative Medicine will attempt to evaluate the efficacy and safety of ABH001 for treating EB patients with wounds that are not healing. The hypothesis being tested is that ABH001 may initiate and continue the wound healing process for these particular EB patients. Currently, a handful of sites in the U.S. are recruiting for this study including UCSD Children’s Hospital in San Diego, Northwestern University in Chicago and Virginia Clinical Research in Norfolk, Va. For more information about this study, click here to link to ClinicalTrials.gov.
     
  • Treatment of Chronic and Non-Chronic Wounds in Patients with Recessive Dystrophic Epidermolysis Bullosa Using Helicoll Collagen Dressings Versus Standard Care. This trial is being led by Dr. Lane at Stanford University and launched last October. Its purpose is to test the effectiveness of a specific collagen wound dressing (Helicoll) against traditional wound dressing for treating chronic and no-chronic wounds of RDEB patients. For more information about this study, click here to link to ClinicalTrials.gov.
     
  • Characteristics of Adult Patients with Recessive Dystrophic Epidermolysis Bullosa. Stanford University is also conducting a screening of RDEB patients similar to its Dystrophic EB trial, in particular those adults who survive with the diagnosis into adulthood, to evaluate their characteristics. For more information about this study, click here to link to ClinicalTrials.gov.
     
  • Efficacy of Granulocyte Colony Stimulating Factor in Patients with Dystrophic Epidermolysis Bullosa. This feasibility study from Vanderbilt University in Nashville, TN launched in February 2012 and attempts to measure the effectiveness of Granulocyte Colony Stimulating Factor treatment for DEB patients in a seven day course of treatment. Follow-up with the patients will take place after seven days and 30 days following discontinuation of the drug. For more information about this study, click here to link to ClinicalTrials.gov.
     
  • Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes. This is another study being led by Dr. Lane at Stanford that launched in fall 2011. Revertant Mosaicism means that a patient has two different genetically different cell populations due to spontaneous mutations, and for this study, the research team is attempting to use such circumstances to treat a patient with his or her own normal, non-fragile skin patches. That is, the team will try to culture cells from these areas the EB patients’ skin to create grafts for the wounded areas of the same patient. For more information about this study, click here to link to ClinicalTrials.gov.

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