Pilot Study of Raptiva to Treat Sjogren's Syndrome

Study Summary

This study will examine the effect of the drug Raptiva (efalizumab) in patients with Sjögren's syndrome (SS), an autoimmune disease affecting the glands producing saliva \& tears. The cause of SS is not known, but inflammation plays an important role. Raptiva is approved by the Food and Drug Administration to treat psoriasis, an inflammatory skin disease. Patients 18 years of age \& older with SS may be eligible for this study. Candidates are screened with a history \& physical examination, chest x-ray, and oral \& eye examinations. Participants are randomly assigned to receive either Raptiva or placebo (an inactive substance that looks like Raptiva) for the first 3 months of the study. For the next 3 months, all participants receive Raptiva. Both Raptiva \& placebo are injected under the skin once a week. Evaluation during treatment \& for 2 months after treatment as follows: Full comprehensive evaluations (beginning of the study, at weeks 13 \& 25 and 2 months after treatment ends): * Physical examination \& blood draw. * Saliva collection done in two ways: 1) suctions cups connected to collection tubes are placed over the salivary gland ducts in the mouth and under the tongue; and 2) a sour-tasting liquid is applied to the top \& sides of the tongue at 30-second intervals to stimulate saliva production. * Eye exam for tear gland function. * Questionnaires about mouth \& eye dryness, energy level and overall well-being. * Lip biopsy (screening \& week 13 visits only). A few minor salivary glands are removed for examination under a microscope. The lower lip is numbed, a small cut is made on the inside of the lip, and several glands are removed. The cut is closed with a few stitches that are removed after 5 to 7 days. * Magnetic resonance imaging of the parotid glands (salivary glands near the ear) at weeks 1, 13 and 25. The patient lies on a stretcher that is moved into the scanner (a metal cylinder containing a strong magnetic field). The head is held in place

Primary Outcome

Patient will be considered a responder if (s)he demonstrates improvement in 2 / 3 disease activity measures without worsening of the third one. 1. Salivary flow: 0.45 ml / 15 min improvement in unstimulated whole salivary flow from baseline value obtained at the study entry. 2. Salivary gland biopsy: at least 2 points improvement in the focus score on MSG biopsy 3. Tear flow: at least 30% improvement in ophthalmic Oxford grading scheme or normalization of the scale as defined by score of 0

Interventions

• DRUG Raptiva