Phase II Double-blind, placebo-controlled, randomized treatment trial with two arms: one
SIROLIMUS arm with 92 patients and one placebo arm with 92 patients. The safety and
therapeutic efficacy of SIROLIMUS will be determined within a dosage range of 1 mg/day to 4
mg/day, which will be titrated to tolerance during an initial 3-month open label period,
relative to placebo in SLE patients over 12 months followed by a 1-month washout. The
proposed study design, known as an enriched enrollment randomized withdrawal (EERW), has
major advantages that (1) only people who tolerate SIROLIMUS are randomized, potentially
reducing the percentage of dropouts in the randomized phase and (2) it allows participants to
use an individualized dosage of study medication, which mimics clinical practice in terms of
how SIROLIMUS would be administered. Healthy subjects receive no drugs and serve as controls
for in vitro studies.
Phase:
Phase 2
Details
Lead Sponsor:
State University of New York - Upstate Medical University