Clinical Trial: Randomized Trial of IVIg With or Without Cyclophosphamide in Pemphigus

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Phase 2 Randomized Trial of IVIg With or Without Cyclophosphamide in Pemphigus

Brief Summary: The purpose of this study is to compare two standard treatments for pemphigus to determine which more effectively improves the clinical manifestations of the disease and decreases serum level of the autoantibodies which cause the disease.

Detailed Summary:

Pemphigus is a serious and life-threatening autoimmune disease characterized by blisters and erosions that occur on the skin and oral mucosa. It is caused by autoantibodies that attack desmoglein 1 and 3, adhesion molecules that are present on the surface of the cells (keratinocytes) that make up the superficial layer of the skin. As a result these cells stop sticking together, and come apart resulting in the formation of blisters on the skin.

Pemphigus is usually treated with systemic corticosteroids often given together with immunosuppressive drugs such as Cytoxan (cyclophosphamide), Imuran (azathioprine), methotrexate, CellCept (mycophenolate mofetil) and others. However, the prolonged and high doses of systemic steroids and other immunosuppressive agents used to treat the disease are associated with significant toxicity.

A new treatment which is now being used to treat pemphigus patients that are unresponsive, or that have developed complications to conventional treatment is IVIg (intravenous immunoglobulin). IVIg consists of one of the protein fractions present in blood. It is the fraction that contains antibodies and is called immunoglobulin (Ig). It is purified from blood that has been collected from thousands of donors and treated to remove potential infectious agents. It is administered intravenously (IV) over several hours, several days in succession. The cycles are usually repeated every 2 to 4 weeks until the disease is controlled.

IVIg treatment is currently given in either of two ways, either by itself or with an immunosuppressive drug such as cyclophosphamide or azathioprine. It is unknown which of these two procedures is better. This trial is being conducted to determine which treatment is more effective.

The
Sponsor: New York University School of Medicine

Current Primary Outcome:

  • Clinical Outcome: Extent and Severity of Disease [ Time Frame: 6 - 10 weeks after initiation of therapy ]
  • Serum Levels of Pemphigus Antibodies [ Time Frame: 6-10 weeks after initiation of therapy ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Toxicity of Treatment: Measured in Renal Toxicity, Myelosuppression or Hepatic Toxicity [ Time Frame: Throughout course of study ]
  • Ability to be Weaned Off Steroids [ Time Frame: Measured 6 and 10 weeks after initiation of IVIg treatment ]


Original Secondary Outcome:

  • toxicity of treatment: measured in renal toxicity, myelosupression or hepatic toxicity
  • Ability to be Weaned Off Steroids


Information By: New York University School of Medicine

Dates:
Date Received: June 5, 2007
Date Started: April 2007
Date Completion:
Last Updated: January 21, 2016
Last Verified: January 2016