A Multicenter, Prospective, Longitudinal Study to Evaluate Gastrointestinal Function and Clinical and Molecular Biomarkers in Subjects With Early and Late Diffuse Cutaneous Systemic Sclerosis, Limited Cutaneous Systemic Sclerosis, and Healthy Volunteers
Trial Snapshot
- Phase
- Not Applicable
- Status
- Withdrawn
- Sponsor
- Biogen
- Primary Endpoint
- Change in University of California at Los Angeles Scleroderma Clinical Trial Consortium GI Tract (UCLA-SCTC GIT) instrument score in participants with SSc
Study Overview
Brief Summary
The primary objective of the study is to assess the change in systemic sclerosis (SSc)-associated gastrointestinal (GI) tract symptoms over a 1-year period in participants with SSc.
Detailed Description
No study treatment will be administered. Study participants will continue to be treated by their respective physicians according to standard clinical practice. Data on concomitant therapy and procedures, administered as part of standard of care, will be collected at baseline and at each study visit.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •Healthy volunteers
- •Must be in good health as determined by the Investigator, based on medical history, physical examination, and vital signs.
- •Participants with diffuse cutaneous systemic sclerosis (dcSSc):
- •Must fulfill the 2013 classification criteria for SSc of the American College of Rheumatology (ACR) and European League Against Rheumatism (EULAR).
- •Absence of the anti-centromere autoantibody.
- •Participants with limited cutaneous systemic sclerosis (lcSSc):
- •Must fulfill the 2013 classification criteria for SSc of the ACR and EULAR.
- •Subjects with lcSSc must have disease duration of less than 5 years.
Exclusion Criteria
- •History of any clinically significant medical condition that can interfere with the conduct of the study, or that in the opinion of the Investigator, would compromise data interpretation.
- •An active severe infection as determined by the Investigator.
- •Female subjects who are pregnant or currently breastfeeding.
- •A known history of malignancy.
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Arms & Interventions
Healthy
Approximately 10 healthy adults will be enrolled into this study
Intervention: Healthy
SSc
Participants with dcSSc and lcSSc
Intervention: SSc
Outcomes
Primary Outcomes
Change in University of California at Los Angeles Scleroderma Clinical Trial Consortium GI Tract (UCLA-SCTC GIT) instrument score in participants with SSc
Time Frame: Baseline to Month 12
This participant-reported assessment is a 34-item instrument designed to measure GI tract involvement in participants with SSc. The UCLA SCTC GIT 2.0 has 7 multi-item scales: Reflux, Distension/Bloating, Diarrhea, Fecal Soilage, Constipation, Emotional Well-being, and Social Functioning and a total GIT score. All scales are scored from 0 \[better health-related quality of life (HRQOL)\] to 3 (worse HRQOL) except the diarrhea and constipation scales (ranges 0-2 and 0-2.5, respectively). The Total GIT Score, is developed by averaging 6 of 7 scales (excluding constipation) with lower scores indicating better HRQOL.
Secondary Outcomes
No secondary outcomes reported