New treatment investigated for SSc

Systemic sclerosis (SSc) is a rapidly progressing systemic form of scleroderma for which there are currently no approved treatments

SSc is a chronic autoimmune disease characterized by connective tissue fibrosis, involving organs such as the skin, heart, kidneys and lungs. Interstitial lung disease (ILD) is one of the main manifestations of SSc, and the varying degrees of pulmonary inflammation and fibrosis can lead to restrictive lung disease. Progressive SSc with ILD (SSc-ILD) can result in a 10-year mortality rate of about 50%(1) with approximately 30% of SSc deaths attributed to pulmonary fibrosis.(2)

While the DeSScipher(3) global research project include observational trials(4) with the goal to improve our understanding of the SSc condition and patients’ quality of life there is still unmet need for disease and/or organ-specific treatments for systemic sclerosis. The good news is that a Phase II trial has been initiated to investigate CC-4047 (pomalidomide) overall efficacy and safety in patients with SSc-ILD. CC-4047 has demonstrated potent antifibrotic effects in a preclinical skin fibrosis model and the investigators, led by Dr Jörg Distler of the University of Erlangen-Nuremberg, Germany, concluded that these data support clinical studies in SSc.(4)

The trial, entitled “A Phase II, Proof-of-Concept, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of CC-4047 in Subjects with Systemic Sclerosis with Interstitial Lung Disease” [NCT01559129](5) is designed to assess the safety and efficacy of CC-4047 treatment in SSc-ILD patients. Since initiation, there are now 47 active sites across 10 countries: Australia, France, Germany, Italy, Poland, Russia, Spain, Switzerland, the United Kingdom and the United States. The target is to enroll 88 SSc-ILD patients over 1 year at approximately 55 sites.

The progression of this potential therapy into Phase II is very encouraging, particularly in a disease which is so rare and for which there are no treatments available. If you are interested in participating in the trial, or would simply like to find out more, please visit http://www.clinicaltrials.gov/ct2/show/NCT01559129 .

References
(1) Mayes MD et al. Arthritis Rheum 2003;48:2246–2255.
(2) Steen VD et al. Ann Rheum Dis 2007;66:940–944
(3) http://www.eustar.org/index.php?module=ContentExpress&func=display&ceid=110&bid=19&btitle=eustar&meid=70
(4) EUROPA Cordis. http://cordis.europa.eu/search/index.cfm?fuseaction=proj.document&PJ_RCN=13430917.
(5) Weingärtner S et al. Ann Rheum Dis 2012;71:1895–1899.
(6) ClinicalTrials.gov 2012. http://www.clinicaltrials.gov/ct2/show/NCT01559129 (accessed March 2013).