Logo

Clinical trial design in scleroderma: where are we and where do we go next?

, , , , ,

Departments of Medicine and Dermatology, Division of Immunology and Rheumatology, Stanford University and Palo Alto VA Health Care System, Palo Alto, USA. shauwei@stanford.edu

CER5590 Submission on line
2012 Vol.30, N°2 ,Suppl.71 - PI 0097, PF 0102
Review

Free to view (click on article PDF icon to read the article)

Rheumatology Article

 

Abstract

Drug development for SSc has been hindered by the relative paucity of validated outcome measures and biomarkers for use in clinical trials. The Scleroderma Clinical Trials Consortium (SCTC) conducted an interactive session at the Scleroderma International Workshop in Cambridge, UK in July 2011 to discuss clinical trial design in SSc. The following issues were discussed: 1) primary outcome for trials of SSc – skin vs. lung vs. composite; 2) ischaemic digital ulcers in SSc – healing vs. repair vs. composite; 3) pulmonary arterial hypertension in SSc; and 4) neglected aspects of SSc – opportunities for study or of lower priority and feasibility. Randomised controlled trials with collection of biospecimens are necessary to assess efficacy of therapeutic agents, validate novel outcome measures, and discover and validate potential biomarkers for each of these areas. Although SSc is a rare, heterogeneous disease, collaborative efforts led by the SCTC and other international networks will ultimately improve the design of clinical trials of promising therapies for SSc.

PMID: 22691217 [PubMed]

Received: 29/03/2012 - Accepted : 15/05/2012 - In Press: 30/05/2012 - Published: 31/05/2012