The purpose of this trial is to (a) determine the ability of KC706 to prevent the appearance of new blisters and heal existing blisters, while maintaining stable doses of corticosteroids and/or immunosuppressants, in patients with pemphigus vulgaris (b) to determine the safety of KC706 and (c) to access the plasma levels of KC706.
This study, sponsored by Kemia, Inc., has been completed.
Background
KC706 is a new class of agent that could offer hope to patients in healing and preventing pemphigus lesions. Patients with PV have antibodies directed against proteins called desmogliens, which serve as a “glue” between the layers of skin. The study will assess the effects of the experimental drug KC706 to interrupt the activity of these antibodies, thereby preventing the skin from forming blisters.
Current treatments have multiple side effects therefore there is a high unmet medical need for treatments with fewer side effects. Dr. Rubinstein’s, from UNC Chapel Hill has developed an animal model of pemphigus vulgaris. When used in this animal model, KC706 prevented the occurrence of blistering.
Enrollment Criteria
Patients must be at least 18 years of age, with active PV skin, scalp or mucosal lesions and a diagnosis of pemphigus vulgaris confirmed by skin lesion biopsy. The patients must be taking and require either stable doses of corticosteroid therapy or immunosuppressive therapy or both. Pregnant or nursing women are not eligible to participate in the trial. All potential participants will have a screening visit which will include a review of their medical history, and a blood draw to determine their eligibility to participate in the study.
Study Conduct
3 months of treatment, assessments of efficacy and tolerability every two weeks, with a four week follow-up.
Contact Information
If you are interested in participating in this trial or referring a patient, please contact:
University of Pennsylvania
Principal Investigator: Victoria Werth, M.D.
Clinical Coordinators:
Joyce Okawa, R. N.
215-898-0168 or joyce.okawa@uphs.upenn.edu
or
Matt Rose
215-898-0108 or Mathew.rose@uphs.upenn.edu
New York University School of Medicine
Principal Investigator: Bruce Strober, M.D. Ph.D.
Clinical Coordinators:
Elizabeth Camacho or dermpharm@popmail.med.nyu.edu
212-263-5244
Florida Academic Dermatology Centers-Cedars Medical Center
Principal Investigator: Francisco Kerdel M.D.
Clinical Coordinators:
Ray Guevara
305-324-2110 or fadcresearch@bellsouth.net
Harbor UCLA Medical Center
Principal Investigator: Michael Kolodney, MD
Clinical Coordinators:
Rattapon Thuangtong, MD
310-222-1845 or rattaponthuangtong@yahoo.com
University Hospitals Case Medical Center
Principal Investigator: Neil Korman, M.D.
Clinical Coordinator:
Kelly Jeffords
216-983-0861 or kelly.jeffords@uhhospitals.org
University of Texas-Southwestern Medical Center
Principal Investigator: Amit Pandya, MD
Clinical Coordinators:
Christina Carrigan, R. N.
214-645-8968 or Christina.Carrigan@UTSouthwestern.edu
University of North Carolina- Chapel Hill
Principal Investigator: David Rubenstein, M.D. Ph.D.
Clinical Coordinator:
Trevor Presler
919-966-1406 or Trevor_presler@med.unc.edu
