Systemic sclerosis Scleroderma, (SSc) can be associated with a high morbidity and mortality. The complexity and heterogeneity of the disease mandates a composite response index that will capture different organ involvement and patient-reported outcomes. There is a critical need for an SSc-combined response index (SSc-CRI) to facilitate drug development and to allow comparison among clinical trials in SSc, as a similar index did in rheumatoid arthritis. We have assembled a group of international experts who have expertise in clinical trial design and development of response criteria in different arthritides. UCLA Clinical Coordinating Center has successfully coordinated recently concluded NIH-funded SSc clinical trials. We took the first step in the developing an SSc-CRI by conducting a structured Delphi exercise to develop a provisional core set of items for clinical trials. The Delphi exercise identified 11 domains relevant to diffuse SSc clinical trials. Our long-term goals are to improve methodologically sound SSc clinical trials and increase interest in drug development for SSc. This application aims to develop an SSc-CRI to be used as an outcome measure in diffuse SSc clinical trials. Our central hypothesis is that a core set of variables for patients with diffuse SSc can be used to develop a formula for an SSc-CRI with robust test characteristics.
Our Specific Aims for the 3-year proposal are:
Specific Aim 1. Perform a prospective longitudinal observational clinical study in 200 patients with diffuse SSc to define a reliable, valid and responsive set of SSc measures for an SSc-CRI, based on a recently completed Delphi exercise.
Specific Aim 2. Employ a prospective, data-driven, consensus building techniques to develop and quantitatively evaluate candidate definitions for an SSc-CRI for diffuse SSc. The research proposed in this application is significant because it is expected to provide a single SSc-CRI for diffuse SSc, thereby providing an impetus for drug development and improved assessment of efficacy of therapeutic agents in clinical trials. Lay Language: We propose to develop a response criteria for clinical trials in diffuse scleroderma that can facilitate drug development and hopefully, can lead to effective treatments for scleroderma.

Agency
National Institute of Health (NIH)
Institute
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Type
Research Project--Cooperative Agreements (U01)
Project #
5U01AR055057-03
Application #
7678566
Study Section
Special Emphasis Panel (ZAR1-KM-L (J2))
Program Officer
Witter, James
Project Start
2007-08-01
Project End
2012-07-31
Budget Start
2009-08-01
Budget End
2012-07-31
Support Year
3
Fiscal Year
2009
Total Cost
$348,649
Indirect Cost
Name
University of California Los Angeles
Department
Internal Medicine/Medicine
Type
Schools of Medicine
DUNS #
092530369
City
Los Angeles
State
CA
Country
United States
Zip Code
90095
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