EB Research Update, Summer 2016

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.

The Latest in EB Research

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.

The Latest in EB Research

For those facing epidermolysis bullosa, staying informed about the latest clinical research related to the disease can be helpful. A couple times a year, we like to offer a snaphot of clinical trials currently recruiting or in progress that could impact the EB community based on those logged on the U.S. National Institutes of Health (NIH) website.

At present, there are 14 open, international, EB clinical research trials listed as active, with one of the studies not yet recruiting and four of the other international studies listed with unknown recruiting status. Of the remaining nine EB research studies which are open and recruiting, six of them are actively being run within the United States and three are being led in other internationally-based locations.

This summary spotlights key background information as it relates to the six U.S. studies. For the most up-to-date, detailed information about all EB studies open and recruiting here in the U.S. and abroad, please go to the NIH’s Clinical Trials page at this link: 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 Dr. Alfred T. Lane of Stanford University’s School of Medicine, this study launched in December 2010 and was last verified in March 2014. The study represents 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 December 2013, 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. During the trial, EB patients receive a combination of a chemotherapy treatment 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. Dr. Lane at Stanford University is also conducting a screening of subjects with DEB to evaluate the characteristics of these patients, who may develop severely painful blistering and open wounds. Last verified in March 2014, 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.
  • Open Label Extension Study to Evaluate the Safety of SD-101 Cream in Those with EB. Launching in March 2014 and last verified in April 2014, this study led by Scioderm, Inc. is the most recently launched, active EB trial. The purpose of this study is to assess the continued safety of topical use of SD-101 cream in subjects with epidermolysis bullosa. Patients age 6 months or older diagnosed with EB are still being recruited through the following locations:  Stanford University (Palo Alto, CA); Ann & Robert H. Lurie Children’s Hospital of Chicago (Chicago, IL); Washington University School of Medicine (St. Louis, MO); Hackensack University Medical Center (Hackensack, NJ); University of North Carolina School of Medicine (Chapel Hill, NC); Texas Dermatology and Laser Specialists (San Antonio, TX); and Seattle Children’s Hospital (Seattle, WA). For more information about this study, click here to link to ClinicalTrials.gov.
  • Characteristics of Adult Patients with Recessive Dystrophic Epidermolysis Bullosa. In May 2009, Stanford University launched another screening that was last verified in March 2014 and is still currently recruiting participants. This trial featuring RDEB patients screens diagnosed adults to study if there are additional characteristics that support survival into adulthood. 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 continues to recruit for participants, and it was most recently verified in July 2013. The study attempts to measure the effectiveness of the Granulocyte Colony Stimulating Factor treatment in DEB patients over a 7-day trial period. Following this timeframe, the team follows up with participating patients at two intervals: 7 days after and 30 days after discontinuation of the drug. For more information about this study, click here to link to ClinicalTrials.gov.

Dedicate Yourself to Raising EB Awareness

It’s here: National EB Awareness Week. As always, we’re urging all of the members of the EB Resource communities at both Twitter and Facebook to use this week as a wonderful platform to educate others about epidermolysis bullosa. This is a perfect opportunity to share your own story, fill people in on the facts about what EB is and what life is like for those diagnosed and for their loved ones caring for them.

This can also be a great time to host or throw your support behind events to raise awareness and dollars for programs supporting EB families, developing better EB treatments or researching to find a cure. Here are some of the events that we have heard about going on across the country. You can do your part to helping to spread the word about these and any other events you should hear about, and if you would, please send our way any info about other events taking place during EB Awareness Week in your community. Thanks!

 

 

  • EB Resource “RT for EB” Tweet-a-Thon (Online only)
    Starting at 8 a.m. on Friday, October 25, we will be tweeting every hour as part of a 24-hour Twitter marathon designed to educate others about EB and encourage our followers and hopefully THEIR followers to spread the message across the twitterverse. The marathon concludes during the 7 a.m. hour on Saturday, October 26 but don’t forget – EB Awareness Week runs through Thursday, Oct. 31, so don’t let our 24-hour campaign stop you from RT’ing long after we’re done! Keep spreading the word and follow us on Twitter at http://www.twitter.com/ebresource.
     
  • ACTion for Jackson 2013 (New York, NY)
    The Jackson Gabriel Silver Foundation (www.jgsf.org) hosts its fourth annual benefit from 6:30 p.m. to 9 p.m. Friday, October 25 at Christie’s, 20 Rockefeller Plaza in NYC. Evening will include cocktails, appetizers and an auction, with Christie’s 19th Century Paintings and Prints on display. Tickets start at $185. Proceeds from the event benefit JGSF’s research fundraising efforts which support promising therapies being developed across the country to treat and cure EB. For more information about the event or to buy tickets, go to www.actionforjackson.com.
     
  • Gourmet Cupboard Fundraiser (Nationwide)
    The Gourmet Cupboard continues its month-long October benefit for DebRA of America during EB Awareness Week and through Thursday, October 31. For every mix purchased from its online store, with mixes ranging from desserts to chili, the company will donate $1.50 each. Visitors must be sure to order through DebRA’s page at www.thegourmetcupboard.com/fundraisers/debraofamerica. Bookmark it and start browsing! After all, holidays are coming…
     
  • Merinda Mae Fundraiser (Hartivlle, OH)
    Stop by the Merinda Mae Children’s Boutique, Hartville MarketPlace at 1289 Edison St. NW, during National EB Awareness Week and help support DebRA of America programs and services. Merinda Mae will donate all profits from Friday, October 25 through Thursday, October 31 to DebRA. For more info, email rhw9976@aol.com.
     
  • EB Awareness Walk-A-Thon (Batesville, MS)
    Sign up to participate in this benefit walk to be held along the walking trail of Batesville Trussell Park on Hwy. 35N and scheduled to start at 9:30 a.m. on Saturday, October 26. All proceeds go toward DebRA of America. For more info, email hfowler@first-heritage.com.

 

  • East Coast Premiere of “What Were We Talking About?” (Spring Lake, NJ)
    Take in an original play by the Wyoming Magnolias, founders of Magnolia Productions, as they put on this stage show in support of the EB community at Spring Lake Community Theatre, 300 Madison Ave. Shows are scheduled Saturday, October 26 and Sunday, October 27, at 7:30 p.m. and 3 p.m. respectively, with all proceeds being donated to DebRA of America. For more info, click here.

 

 

Update on EB Research Trials

A few times a year, we provide an updated list of the current clinical research trials recruiting or in progress that could impact the EB community based on those captured on the U.S. National Institutes of Health (NIH) website. At this time, there are 16 open EB clinical research trials listed as active, up four new trials since we last provided a research update in April. Two studies abroad in France and Switzerland are not yet recruiting, and the status of three other international studies is currently labeled unknown. Of those 11 EB research studies which are open and recruiting, eight of them are actively being run within the United States and three are being led in other internationally-based locations.

 

The following list of trial summaries highlights key information about the U.S. studies that are currently recruiting. For the latest detailed information about all EB studies open and recruiting here in the U.S. and abroad, you can visit the NIH’s Clinical Trials page at the following link: http://clinicaltrials.gov/ct2/results?term=epidermolysis+bullosa&recr=Open. Please keep in mind that since a federal government shutdown became effective October 1, information may not have been updated since that date due to a decrease in staff available to maintain the data. The NIH notes this on its own website in a disclaimer.

 

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 links provided 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 heads up this study first launched in December 2010 and last verified in December 2012. The trial is being conducted in collaboration with the National Institute of Arthritis and Musculoskeletal and Skin Diseases. For this study, researchers create a graft of the participant’s skin that has been genetically engineered in a culture, in an attempt to correct its cellular protein deficiency which has led to 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 was last verified in May 2013 and 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 certain and reduce the skin’s fragility caused by severe forms of EB. For treatment, EB patients are receiving 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 Dr. Lane’s gene transfer study, Stanford University is conducting a screening of subjects with DEB to evaluate the characteristics of these patients, who may develop severely painful blistering and open wounds. This trial was last verified in December 2012 and 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.

 

  • 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 and last verified in September 2013, 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 this population of EB patients. Sites in the U.S. currently recruiting for this study include Phoenix Children’s Hospital, Lucile Packard Children’s Hospital at Stanford University, Rady Children’ Hospital at UCSD in San Diego, Denver Children’s Hospital, Ann and Robert H. Lurie Children’s Hospital in Chicago and Virginia Clinical Research in Norfolk, Va., as well as eight locations across Canada and Europe. 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, which launched in October 2012 and was last verified in February 2013, is also being led by Dr. Lane at Stanford University. The purpose of the study 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. Eligible trial participants must be seven years or older. For more information about this study, click here to link to ClinicalTrials.gov.

 

  • Characteristics of Adult Patients with Recessive Dystrophic Epidermolysis Bullosa. Stanford University launched another screening in May 2009 that continues and was last verified in December 2012. This study of RDEB patients screens diagnosed adults to assess if there are additional characteristics that support survival into adulthood. 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 was launched in February 2012 and verified in July 2013. The study attempts to measure the effectiveness of the Granulocyte Colony Stimulating Factor treatment in DEB patients over 7-day trial period. The team would then follow up with participating patients at two intervals: 7 days after and 30 days after 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 University. Launched in October 2011 and last verified in December 2012,this trial plans to use Revertant Mosaicism to test a hypothesis. Revertant Mosaicism means that a patient has two different genetically different cell populations due to spontaneous mutations. In this study, the patient will be treated using his or her own normal, non-fragile skin patches from cells the research team has cultured from unaffected areas of the EB patients’ skin using grafts to treat wounded areas on the same patient. For more information about this study, click here to link to ClinicalTrials.gov.

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.

Latest EB Clinical Trials

The U.S. National Institutes of Health (NIH) currently reflects eleven EB clinical trials that are listed as “recruiting.” These include seven active EB research studies in the U.S. and four clinical trials abroad. We have compiled brief summaries highlighting specific aspects of the U.S. studies below. For complete information about all eleven of the EB studies recruiting internationally, visit the NIH’s Clinical Trials page by clicking here.

  • Allogeneic Hematopoietic Stem Cell Transplant for Epidermolysis Bullosa. Led by the team at the Masonic Cancer Center at the University of Minnesota, this trial is testing the safety and effectiveness of stem cell infusion for treatments of RDEB. Results based on animal models have indicated that stem cells may be able to home in on the skin and repair biochemical and structural abnormalities associated with RDEB and its collagen 7 deficiency. For complete information about this study, click here to link to ClinicalTrials.gov.
  • Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa. Dr. Alfred T. Lane of Stanford University’s School of Medicine is the primary investigator for this study, which first launched in December 2010. The trial, being conducted in collaboration with the National Institute of Arthritis and Musculoskeletal and Skin Diseases, involves creating a graft of the participant’s skin to attempt to correct the cellular protein deficiency that causes RDEB in a culture. Once this is completed, doctors then transplant the “corrected” cells back onto the patient’s skin. For complete information about this study, click here to link to ClinicalTrials.gov.
  • Stem Cell Transplant for Epidermolysis Bullosa. This study, entering Phase 2 of research by the Masonic Cancer Center at the University of Minnesota, is attempting to test its main hypothesis that the infusion of bone marrow or umbilical cord blood from a healthy donor can correct the cellular protein deficiency of several severe forms of EB and that this infusion may reduce skin fragility in participants. The study is also testing a secondary hypothesis that mesenchymal stem cells from a healthy donor will enhance the safety and effectiveness of the allogeneic hematopoietic stem cell transplant to serve as a renewable cell source to treat specific areas of blistering. For complete information about this study, click here to link to ClinicalTrials.gov.
  • Characteristics of Patients with Dystrophic Epidermolysis Bullosa. Stanford University is also conducting a screening of subjects with DEB to evaluate the characteristics of the patients, who usually develop severely painful blistering and open wounds, as well as their cells as a means to support the development of future therapies. For complete information about this study, click here to link to ClinicalTrials.gov.
  • Characteristics of Adult Patients with Recessive Dystrophic Epidermolysis Bullosa. Similarly, Stanford University is conducting a screening to evaluate characteristics of patients diagnosed with RDEB who survive to adulthood. For complete information about this study, click here to link to ClinicalTrials.gov.
  • Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa. This Columbia University-sponsored study continues to seek qualified patients at three locations, including The Children’s Hospital in Aurora, CO, Children’s Memorial Hospital in Chicago, IL, and Morgan Stanley Children’s Hospital of NYP in New York City. The study is trying to determine the event-free survival and overall survival for RDEB patients after undergoing Reduced Intensity Conditioning by a combination of busulfan/flurdarabine/alemtuzumad (BFA) and Allogeneic Stem Cell Transplantation (ALLOSCT). For complete information about this study, click here to link to ClinicalTrials.gov.
  • Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes. One of the most recently launched clinical trials that began in October of last year, this study led by Dr. Lane and the Stanford University team is listed as open to EB patients 18 and older (simplex, junctional or dystrophic). The trial will attempt to culture cells from EB patients’ non-fragile skin patches and graft them on to wounded areas for the same patient. For complete information about this study, click here to link to ClinicalTrials.gov.