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


Chung, Lorinda; Denton, Christopher P; Distler, Oliver; Furst, Daniel E; Khanna, Dinesh; Merkel, Peter A (2012). Clinical trial design in scleroderma: where are we and where do we go next? Clinical and Experimental Rheumatology, 30(2 Suppl 71):S97-S102.

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.

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.

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Additional indexing

Item Type:Journal Article, refereed, further contribution
Communities & Collections:04 Faculty of Medicine > University Hospital Zurich > Rheumatology Clinic and Institute of Physical Medicine
Dewey Decimal Classification:610 Medicine & health
Language:English
Date:2012
Deposited On:14 Feb 2013 16:47
Last Modified:17 Feb 2018 01:00
Publisher:Pacini Editore SpA
ISSN:0392-856X
OA Status:Closed
Official URL:http://www.clinexprheumatol.org/pubmed/find-pii.asp?pii=22691217
PubMed ID:22691217

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