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The use of KC706 for the Treatment of Pemphigus Vulgaris

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.


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 agluebetween 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 [email protected]


Matt Rose
215-898-0108 or [email protected]

New York University School of Medicine

Principal Investigator: Bruce Strober, M.D. Ph.D.

Clinical Coordinators:

Elizabeth Camacho or [email protected]

Florida Academic Dermatology Centers-Cedars Medical Center

Principal Investigator: Francisco Kerdel M.D.

Clinical Coordinators:

Ray Guevara
305-324-2110 or [email protected]

Harbor UCLA Medical Center

Principal Investigator: Michael Kolodney, MD

Clinical Coordinators:

Rattapon Thuangtong, MD
310-222-1845 or [email protected]

University Hospitals Case Medical Center

Principal Investigator: Neil Korman, M.D.

Clinical Coordinator:

Kelly Jeffords
216-983-0861 or [email protected]

University of Texas-Southwestern Medical Center

Principal Investigator: Amit Pandya, MD

Clinical Coordinators:

Christina Carrigan, R. N.
214-645-8968 or [email protected]

University of North Carolina- Chapel Hill

Principal Investigator: David Rubenstein, M.D. Ph.D.

Clinical Coordinator:

Trevor Presler
919-966-1406 or [email protected]

Geplaatst in Klinische proeven


The P/P Registry has been approved by the Western Institutional Review Board (WIRB) and is actively enrolling participants.