Research

Scleroderma Treatment Outcome Study

Development of Clinical Disease Outcome Measures with Biological Substudies for Localized Scleroderma (LOCUS-2)

Protocol Description

The purpose of this multi-center study is to standardize and refine clinical assessments of patients with juvenile localized scleroderma so that sensitive scoring tools can be developed to facilitate comparison between patients at different time points, and between patients. Patient and parent quality of life and function will also be evaluated. There will be optional laboratory sub-studies to: 1) evaluate T-cell associated cytokine profiles during disease activity and damage, and 2) evaluate RNA expression to identify novel immunogenetic profiles specific to different types of localized scleroderma. The study does not include therapeutic treatment.

Eligibility Criteria

Subject to certain exclusion criteria, this study is open to individuals of both genders under the age of 25 who have been diagnosed with localized scleroderma and who were under the age of 16 when the first symptoms attributable to scleroderma were identified.
Males: Under age 25
Females: Under age 25

Requirements
Participants will complete quality of life questionnaires, and their physicians will also complete study forms. Those participating in optional substudies will provide blood samples at each of three visits, which will be done in 1 to 6 month intervals.
Visits: 3
Duration: Up to 18 months

Status: Open for Enrollment

Source(s) of Support
National Institute of Health, K23 Award
The Nancy Taylor Foundation for Chronic Diseases