PT  - JOURNAL ARTICLE
AU  - Vivien M. Hsu
AU  - Christopher P. Denton
AU  - Robyn T. Domsic
AU  - Daniel E. Furst
AU  - Maureen Rischmueller
AU  - Marina Stanislav
AU  - Virginia D. Steen
AU  - Jörg H.W. Distler
AU  - Shimon Korish
AU  - Alyse Cooper
AU  - Suktae Choi
AU  - Peter H. Schafer
AU  - Gerald Horan
AU  - Douglas R. Hough
TI  - Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study
AID  - 10.3899/jrheum.161040
DP  - 2018 Mar 01
TA  - The Journal of Rheumatology
PG  - 405--410
VI  - 45
IP  - 3
4099  - http://www.jrheum.org/content/45/3/405.short
4100  - http://www.jrheum.org/content/45/3/405.full
SO  - J Rheumatol2018 Mar 01; 45
AB  - Objective. To evaluate the safety and efficacy of pomalidomide (POM) on forced vital capacity (FVC), modified Rodnan skin score (mRSS), and gastrointestinal (GI) symptomatology over 52 weeks of treatment in patients with interstitial lung disease due to systemic sclerosis (SSc).Methods. Twenty-three adult patients diagnosed with SSc were randomized 1:1 POM:placebo (PBO).Results. Mean change at Week 52 from baseline in predicted FVC% −5.2 and −2.8; mRSS −2.7 and −3.7; and UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract (SCTC GIT 2.0) score 0.1 and 0.0, with POM and PBO, respectively. Statistical significance was not achieved for any of these 3 primary endpoints at 52 weeks.Conclusion. Because of recruitment challenges, subject enrollment was discontinued early. In an interim analysis, the study did not meet its Week 52 primary endpoints. Therefore, a decision was made to terminate all study phases. POM was generally well tolerated, with an adverse event profile consistent with the known safety and tolerability profile of POM in other diseases. Study results were neither positive nor negative because too few subjects were enrolled to make meaningful conclusions. Clinical Trials number: NCT01559129.