RT Journal Article
SR Electronic
T1 Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study
JF The Journal of Rheumatology
JO J Rheumatol
FD The Journal of Rheumatology
SP 405
OP 410
DO 10.3899/jrheum.161040
VO 45
IS 3
A1 Vivien M. Hsu
A1 Christopher P. Denton
A1 Robyn T. Domsic
A1 Daniel E. Furst
A1 Maureen Rischmueller
A1 Marina Stanislav
A1 Virginia D. Steen
A1 Jörg H.W. Distler
A1 Shimon Korish
A1 Alyse Cooper
A1 Suktae Choi
A1 Peter H. Schafer
A1 Gerald Horan
A1 Douglas R. Hough
YR 2018
UL http://www.jrheum.org/content/45/3/405.abstract
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.