The official title of the study is A Randomized, Double-Blind, Placebo-Controlled, Dose-Response Study of the Safety and Efficacy of Thymosin Beta 4 in the Treatment of Patients with Epidermolysis Bullosa.
RegeneRx Biopharmaceuticals, Inc. (www.regenerx.com) is still recruiting participants for its Phase 2 clinical trial to test the safety and wound healing properties of Thymosin Beta 4 (Tß4) in EB wounds. The study is open to people age two and older with Junctional or Dystrophic EB.
In response to patients who would like to participate at local facilities, RegeneRx has expanded the number of physician investigators throughout the US. Additionally, RegeneRx has a travel expense reimbursement policy in place to facilitate patient participation in the study.
The following physicians are participating in the study:
• Jo-David Fine MD, MPH, Vanderbilt University, Nashville TN
• Anna Bruckner MD, Stanford University, Stanford CA
• Moise Levy MD, Texas Children's Hospital, Houston TX
• Kim Morel MD, Columbia University, New York NY
• Tor Shwayder MD, Henry Ford Medical Center, Detroit MI
• Karen Wiss MD and Amit Garg MD, University of Mass at Hahnemann Campus, Worcester MA
• Elizabeth Alvarez Connelly MD, University of Miami, Miami FL
• Amy Paller MD, Children’s Memorial Hospital, Chicago IL
• Amy Theos MD, University of Alabama Birmingham, Birmingham AB
• Susan Bayliss MD, St. Louis Children’s Hospital, St. Louis MO
• Jon Dyer MD, University of Missouri Columbia, Columbia MO
• David Pariser MD, Virginia Clinical Research, Inc., Norfolk VA
• Mary Gail Mercurio MD, University of Rochester, Rochester NY
Plans are underway to add additional study sites in Pennsylvania and Southern California.
The study medication (Tß4 or Placebo) is supplied as a gel and will be applied once daily to a single lesion (a sore selected by the physician investigator for treatment evaluation). Study medication will be applied daily until the lesion completely heals or for up to fifty-six days, whichever comes first. Visits will be required weekly for the first month of the study and then biweekly.
Tß4 is a naturally occurring substance present in virtually all human cells. It represents a new class of wound healing drug. A key mechanism of action is Tß4's ability to regulate the cell-building protein, actin, a vital component of cell structure. Additionally, it has been reported that Tß4 directly influences the production of laminin-5, a protein,
important to the wound healing process because it is known to promote cell migration and adhesion.
Listed here are some of the criteria patients must meet in order to be eligible to participate in this study:
? Age 2 or older
? Junctional or Dystrophic EB:
? Lesion to be treated must be: located on a limb or the trunk , 14-60 day duration 5cm to 50cm
Clear of infection
? No investigational drug within 30 days
? No immunotherapy or cytotoxic chemotherapy within 60 days
? No systemic or topical steroidal therapy within 30 days (except inhaled steroids)
? No systemic antibiotics within 7 days
? No current or former malignancy, including SCC
? No Diabetes Mellitus
? Not pregnant or breastfeeding
Additional considerations:
? Females of childbearing potential
? Other health issues
? Availability for study visits
For more information about the study and how to participate, you may contact one of the physicians listed above or Madeline Weiner, RN, RegeneRx Biopharmaceuticals Inc., at 919-929-1855 or madeline.weiner@mindspring.com
Stanford University in California is currently looking for patients to participate in a preliminary screening for possible gene transfer trial for recessive dystrophic epidermolysis bullosa (RDEB).
They are looking for patients who meet the following criteria:
Have a clinical diagnosis of RDEB by a local dermatologist
Are 18 years of age or more, and willing to give consent
Estimated to have at least 100 to 200 cm 2 areas of open erosions on the trunk or extremities
Able to undergo adequate anesthesia to allow grafting procedures to take place
Parents alive, do not have EB, and are willing to give consent for genetic testing.
Medically stable to travel to Stanford University Medical Center
No medical illnesses expected to complicate participation.
Currently this study is limited to residents in the USA
If you meet the above criteria, you may be eligible to come to Stanford University and participate in the screening phase of our trial. The screening will involve skin biopsies, blood tests, and genetic testing. We will pay their travel expenses related to this trial.
For more information or any questions regarding this study and/or the eligibility criteria, please contact the Clinical Trial Coordinator, Emily Gorell, at (650) 725-4302 or at egorell@stanford.edu