Clinical trial design in scleroderma: where are we and where do we go next?
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.
Department of Medicine and Dermatology, Stanford University, Palo Alto, USA.
Lorinda
L
Chung
Christopher P
CP
Denton
Oliver
O
Distler
Daniel E
DE
Furst
Dinesh
D
Khanna
Peter A
PA
Merkel
D015415:Biomarkers / Q000032:analysis; D002986:Clinical Trials as Topic / Q000379:methods*; D023381:Endpoint Determination; D006801:Humans; D011237:Predictive Value of Tests; D015203:Reproducibility of Results; D012107:Research Design; D012595:Scleroderma, Systemic / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D016896:Treatment Outcome
D015415:Biomarkers