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Proposal 1422

Title of the Proposed Research

Developing new outcome measures for SLE by deconvoluting available data sets

Lead Researcher

Peter E. Lipsky, MD

Affiliation

LRI

Funding Source

Funding is being provided by the LRI as well as members of the LRI Lupus Industrial Council (pharmaceutical companies).

Potential Conflicts of Interest

All researchers are working for the LRI Lupus Industrial Council and declare no conflicts of interest.

Data Sharing Agreement Date

11 July 2016

Lay Summary

Systemic lupus erythematosus(SLE) is an autoimmune disease that affects as many as 1.5 million persons in the United States. Despite a great deal of effort, only 1 new drug has been approved for SLE in the past 50 years. One of the difficulties in drug development in SLE is the absence of outcome measures that are responsive to change and also reflect clinical practice.

The goal of the research is to develop new outcome measures that are responsive to change, reflect clinical practice and can facilitate the development of new drugs for SLE. This will be accomplished by re-analyzing data from large completed phase 3 trials and will involve an iterative process incorporating statisticians and a Steering Committee composed of experts in SLE clinical trials and patients.

The specific aims and objectives of the research are to use the data from EMBODY 1 and EMBODY2 as a means to assess whether the information contained in the standard outcome measures can be deconstructed and reassembled into a novel composite endpoint that more effectively separates responders from nonresponders. Current standard outcome measure contain a number of components that can be analyzed separately or in novel combinations. In addition, these components can be coupled to other data collected in the trials, including laboratory evaluations, medication use and patient reported outcomes

A state of the art statistical approach will be employed to deconstruct the end point data in EMBODY 1 into its components and the components reconstructed into new composite end point models that will be assessed statistically for their capacity to distinguish responders from nonresponders in the clinical trial. New possible models will be evaluated by modeling theory and also tested for validity in EMBODY 2 and eventually in other clinical trials. After initial validation, it is anticipated that the results will be presented at a public meeting to discuss their implication and potential utility. Patients will be invited to participate in both the public meeting and as members of a Steering Committee that will evaluate the results as they become available and will be in a position to provide immediate feed back to the statistical team as to their relevance to patients.

Study Data Provided

UCB-SL0009: Study of Epratuzumab Versus Placebo in Subjects With Moderate to Severe General Systemic Lupus Erythematosus (EMBODY 1)
UCB-SL0010: Study of Epratuzumab Versus Placebo in Subjects With Moderate to Severe General Systemic Lupus Erythematosus (SLE) (EMBODY 2)

Statistical Analysis Plan

Publication Citation

The publication citation will be added after the research is published.

Summary Results

Results summary or link will be posted when available.