Johns Hopkins Vasculitis Center

The Johns Hopkins University, in collaboration with the International Network for the Study of the Systemic Vasculitides (INSSYS) is conducting the first randomized clinical trial in Wegener’s granulomatosis (WG) in the United States. The trial, coordinated by Johns Hopkins University, is funded by both the National Institutes of Health and the Food and Drug Administration. Enrollment for the trial began in June 2000, and will continue for 30 months.

WG is a potentially devastating disease. The standard treatments (prednisone, cyclophosphamide, methotrexate) are usually effective at getting the disease under control, but have many potentially toxic side–effects. After going into remission, the disease often flares up again. The biggest current challenge in the treatment of WG patients is the lack of an effective, well–tolerated treatment for the maintenance of disease remissions.

To determine if Enbrel (etanercept) enables patients with WG to achieve lasting remissions more quickly and to use less conventional medication than current treatment regimens permit. To answer these questions, we have organized WGET, a clinical trial.

A clinical trial is a type of medical research. Clinical trials are usually done to evaluate a new treatment in patients with a particular disease. Clinical trials are designed to answer specific questions and to find better ways to treat individuals with a disease (such as WG). For ethical reasons, clinical trials can only be carried out when the physicians do not know if a new treatment is effective in a particular disease.

WGET is a randomized trial of the experimental treatment (Enbrel or placebo) combined with conventional treatments for WG. In addition to conventional treatments (the same treatments patients would receive if they were not in this trial), the patients will receive either Enbrel or a placebo.

Enbrel is an injectable inhibitor of “tumor necrosis factor” (TNF), a protein that plays an important role in many forms of inflammation and is believed to play an important role in WG. Enbrel has been tested extensively in the treatment of certain types of arthritis, and is approved by the Food and Drug Administration for use in rheumatoid arthritis and juvenile rheumatoid arthritis. Preliminary studies in WG have indicated that the use of Enbrel is safe in this disease, but its effectiveness is not known.

A placebo is a liquid that looks like Enbrel but has no Enbrel in it. Both Enbrel and the placebo are given by injection under the skin twice a week. Most patients learn to administer the medication themselves quite easily. Others have a friend or family member administer the medication.

In diseases as serious as WG often is, both the patients and the doctors want experimental treatments to work. This desire on the part of patient and doctor may bias the assessment of an experimental treatment’s true effectiveness. The standard way of avoiding such bias is to assign some patients to the experimental treatment, and some to the placebo. This assignment is done randomly, by a computer. Comparison of the results of the patients in the two groups then allows an unbiased assessment of whether or not the experimental treatment really works.

**We emphasize that ALL patients enrolled in WGET receive standard of care treatments, regardless of whether they are in the Enbrel or the placebo group. Thus, all patients are treated just as they would be if not enrolled in the trial, with the exception of the experimental treatment.**

The trial is monitored very closely by a “Data and Safety Monitoring Board” (DSMB), a group of 5 nationally–known physicians and experts in rheumatology or clinical trials. The DSMB oversees the conduct of the trial and receives regular reports about any problems with the treatments, as well as reports of whether or not one treatment group is doing better than the other. If one group appears to be faring much better (or if there are concerns about side–effects in one group), the DSMB may suggest that the trial be stopped.

With the exception of the experimental treatment (Enbrel or placebo), the physicians treating patients in WGET know exactly which standard medications (prednisone, cyclophosphamide, and methotrexate) the patients are on. This is necessary because of the side–effects that these medications sometimes cause.

Patients in WGET are evaluated at 6 and 12 weeks after the first visit, and then every 3 months. This visit schedule is a minimum for patients with active WG. More frequent visits may be indicated by a patient’s clinical condition, and these are scheduled as frequently as necessary to ensure optimal medical care. Between WGET visits, patients may be managed by their own physicians if they desire.

The cost of the experimental treatment is paid for by Immunex Corporation (Seattle, WA). As standard of care medical treatment, other patient care costs are paid through the normal insurance mechanisms. Patients are reimbursed $25 per study visit for costs incurred in participating in this trial (parking fees, transporation, etc.).

If your WG has been active in the past few weeks or is active now, you may be eligible to participate. For further details regarding this study, please contact one of the physicians (listed below) at the participating medical centers.

8 major medical centers in the United States are involved in WGET. The centers and the doctors you should contact are:

• Johns Hopkins University (Baltimore, MD)
  Dr. John Stone
  Dr. David Hellmann
  Dr. Michael Regan
  410-550-6818
• Cleveland Clinic Foundation (Cleveland, OH)
  Dr. Gary Hoffman
  Dr. Len Calabrese
  Dr. Sudhakar Sridharan
  216-445-6996
• Beth Israel Hospital (New York City, NY)
  Dr. Robert Spiera
  212-870-7964
• Boston University Medical Center (Boston, MA)
  Dr. Peter Merkel
  617-414-1221
• Duke University Medical Center (Durham, NC)
  Dr. William St. Clair
  Dr. Nancy Allen
  919-684-5586
• Mayo Clinic (Rochester, MN)
  Dr. Ulrich Specks
  Dr. Steve Ytterberg
  507-538-0785
• University of California, San Francisco (San Francisco, CA)
  Dr. John Davis
  Dr. Ken Fye
  415-502-1886
• University of Michigan (Ann Arbor, MI)
  Dr. Joseph McCune
  Dr. Hilary Haftel
  734-936-5561

All information contained within the Johns Hopkins Vasculitis Center website is intended for educational purposes only. Visitors are encouraged to consult other sources and confirm the information contained within this site. Consumers should never disregard medical advice or delay in seeking it because of something they may have read on this website.