Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study

The Journal of Rheumatology
Vivien M HsuDouglas R Hough

Abstract

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). Twenty-three adult patients diagnosed with SSc were randomized 1:1 POM:placebo (PBO). 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. 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.

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Citations

Aug 17, 2018·Nature Reviews. Rheumatology·Christopher P DentonJohn G Coghlan
Mar 3, 2018·The Journal of Rheumatology·Lesley Ann Saketkoo
Nov 19, 2019·Frontiers in Medicine·Mehdi MirsaeidiMarilyn K Glassberg
Jul 12, 2020·Journal of Cell Science·Pardis PakshirBoris Hinz
Jul 25, 2020·Respiratory Research·Anna-Maria Hoffmann-VoldMichael Kreuter
Nov 28, 2020·Current Rheumatology Reports·Kimberly Showalter, Jessica K Gordon
Jun 3, 2021·Arthritis Research & Therapy·Corrado Campochiaro, Yannick Allanore
Sep 11, 2021·Zeitschrift für Rheumatologie·Katharina BuschulteMichael Kreuter

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