Latest EB Research Updates

From time to time, we share information about certain clinical trials that are currently in progress and in some cases, still recruiting participants. We highlight trials that could possibly benefit the EB community through its research findings and use of it in creating more powerful treatment for the skin disease. Using the U.S. National Institutes of Health (NIH) website, we have compiled a summary of these research trials that have been logged by the NIH.

 

 

As of June 2017, there are 22 open, international, EB clinical research trials listed as active, with one of the studies not yet recruiting. Of the remaining 21 EB research studies which are open and recruiting, 13 of them are being run within the United States and eight are active in other internationally-based locations.

 

The following summary includes general background information related to the 13 open and recruiting U.S. studies. For complete, up-to-date listings for all 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 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 Shasa Hu, M.D., at the University of Miami, the aim of this study is to investigate the effects of a topical cream on patients 12 years of age and older that have been diagnosed with epidermolysis bullosa. Launched in July 2016 and last verified in May 2017, this study is currently recruiting participants at the University of Miami Department of Dermatology in Miami, Florida. 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 February 2017, this study by the Masonic Cancer Center at the University of Minnesota attempts to test two hypotheses. The underlying hypothesis is that the infusion of bone marrow or umbilical cord blood from a healthy unaffected donor will correct the collagen, laminin, integrin, or plakin deficiency and reduce the skin fragility characteristic of severe forms of EB. A secondary hypothesis is that mesenchymal stem cells from a healthy donor will enhance the safety and efficacy of the allogeneic hematopoietic stem cell transplant as well as serve as a source of renewable cells for the treatment of focal areas of residual blistering For more information about this study, click here to link to ClinicalTrials.gov.
 

· Characteristics of Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB). Jean Yuh Tang at Stanford University is screening subjects with RDEB to evaluate characteristics of the subjects and their cells to develop new strategies of therapy and determine whether subjects could be candidates for treatment studies. This observational study at Stanford University in Stanford, California was launched in November 2009 and last verified in March 2016. For more information about this study, click here to link to ClinicalTrials.gov.
 

· Safety and Efficacy of Diacerein 1% Ointment Topical Formulation Compared to Placebo for Subjects with Epidermolysis Bullosa Simplex (EBS). This study with an anticipated launch date of May 2017 was last verified in June 2017 and is being led by sponsor Castle Creek Pharmaceuticals, LLC. The purpose of this interventional study, being held at Texas Dermatology and Laser Specialists in San Antonio, Texas, is to compare the efficacy of Diacerein 1% Ointment to Control Ointment when applied once daily for 8 weeks in subjects with EBS. For more information about this study, click here to link to ClinicalTrials.gov.
 

· Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients with Nonsense Mutations. Sponsored by the University of Southern California, the target of this study is to evaluate the safety and efficacy of intravenous gentamicin in RDEB patients. In theory, this intravenous administration has the possibility of treating simultaneously all skin wounds of patients. Investigators also propose optimizing the concentration and manner of delivery of topical gentamicin. Launched in January 2017 and last verified then, this study is currently recruiting participants at the University of Southern California in Los Angeles, California. 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 February 2017. The interventional study is a collaboration between the university and the National Institute of Arthritis and Musculoskeletal and Skin Diseases as well as Abeona Therapeutics. This trial, which is being conducted at Stanford University School of Medicine in Stanford, California, will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen. The corrected cells will be transplanted back to the patient. 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. Launched in March 2016 and last verified in February 2017, this study by the Masonic Cancer Center at the University of Minnesota in Minneapolis, Minnesota is a single-institution, 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 in June 2016 and last verified in April 2017, is led by Fibrocell Technologies and administered at Stanford University in Stanford, California. The purpose of the study is to evaluate the safety of FCX-007, evaluate the C7 expression and 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.
 

·Survey to Identify Burdens and Unmet Needs of Patients with Epidermolysis Bullosa. Launched in April 2017 and last verified in May 2017, this observational study sponsored by Amicus Therapeutics in Cranbury, New Jersey, intends to collect information on key aspects of life with epidermolysis bullosa (EB) including diagnostic journey, treatment, management, daily living challenges, and overall psycho-social, socio-economic, academic and family impact. For more information about this study, click here to link to ClinicalTrials.gov.
 

·Study of Cellutome System for Treatment of Individual Lesions in EB Pts. Led by principal investigator Jakub Tolar, MD, PhD of Masonic Cancer Center at the University of Minnesota in Minneapolis, Minnesota, the target of this study, launched August 2016 and last verified in February 2017, proposes local wound therapy using epidermal skin grafting from the same donor that provided the hematopoietic graft, or from the same EB individual with a mosaic (naturally gene corrected) skin. In both cases permissive immune system and skin chimerism is expected to enable long-term epidermal engraftment and wound healing. For more information about this study, click here to link to ClinicalTrials.gov.
 

· Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients. Sponsored by Jean Yuh Tang of Stanford University in collaboration with the Epidermolysis Bullosa Research Partnership and Menlo Therapeutics, Inc., this interventional study launched in July 2016 and was last verified in August 2016. The study held at Stanford University in Stanford, California aims to target the physiological mechanisms of pruritus (or itch), the most common complaint reported by patients with EB of all subtypes and determine whether daily oral administration of VPD-737 (5 mg) is effective and safe in adolescents and adults with EB. For more information about this study, click here to link to ClinicalTrials.gov.
 

· Using Topical Sirolimus 2% for Patients with Epidermolysis Bullous Simplex (EBS) Study. Sponsored by Stanford University and led by Joyce Teng, MD PhD, this interventional study was launched in May 2016 and last verified in November 2016. The pilot study targets the dominant mutant keratin proteins in the skin to ameliorate the severity of EB simplex. The purpose is to improve the function of EB simplex feet with an application of topical sirolimus, 2%. 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 in Baltimore, Maryland 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.

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