Vivien M Hsu1,2, Christopher P Denton3,4, Robyn T Domsic3,4, Daniel E Furst3,4, Maureen Rischmueller3,4, Marina Stanislav3,4, Virginia D Steen3,4, Jörg H W Distler3,4, Shimon Korish3,4, Alyse Cooper3,4, Suktae Choi3,4, Peter H Schafer3,4, Gerald Horan3,4, Douglas R Hough3,4. 1. From the Robert Wood Johnson (RWJ) Medical School Scleroderma Program, New Brunswick; Celgene Corp., Summit, New Jersey; Department of Rheumatology, University of Pittsburgh, Pittsburgh, Pennsylvania; David Geffen School of Medicine at the University of California at Los Angeles (UCLA), Los Angeles, California; Georgetown University Medical Center, Washington, DC, USA; Centre for Rheumatology and Connective Tissue Disease, London, UK; The Queen Elizabeth Hospital and University of Adelaide, Adelaide, Australia; Research Rheumatology Institute n.a. V.A. Nassonova, Moscow, Russia; Department of Internal Medicine 3, University of Erlangen-Nuremberg, Erlangen, Germany. hsuvm@rwjms.rutgers.edu. 2. V.M. Hsu, MD, RWJ Medical School Scleroderma Program; C.P. Denton, MBBS, MRCP, Centre for Rheumatology and Connective Tissue Disease; R.T. Domsic, MD, MPH, Department of Rheumatology, University of Pittsburgh; D.E. Furst, MD, David Geffen School of Medicine at UCLA; M. Rischmueller, MD, FRACP, The Queen Elizabeth Hospital and University of Adelaide; M. Stanislav, MD, PhD, DMSc, Research Rheumatology Institute n.a. V.A. Nassonova; V.D. Steen, MD, Georgetown University Medical Center; J.H. Distler, MD, Department of Internal Medicine 3, University of Erlangen-Nuremberg; S. Korish, MD, Celgene Corp.; A. Cooper, Celgene Corp.; S. Choi, PhD, Celgene Corp.; P.H. Schafer, PhD, Celgene Corp.; G. Horan, PhD, Celgene Corp.; D.R. Hough, MD, Celgene Corp. hsuvm@rwjms.rutgers.edu. 3. From the Robert Wood Johnson (RWJ) Medical School Scleroderma Program, New Brunswick; Celgene Corp., Summit, New Jersey; Department of Rheumatology, University of Pittsburgh, Pittsburgh, Pennsylvania; David Geffen School of Medicine at the University of California at Los Angeles (UCLA), Los Angeles, California; Georgetown University Medical Center, Washington, DC, USA; Centre for Rheumatology and Connective Tissue Disease, London, UK; The Queen Elizabeth Hospital and University of Adelaide, Adelaide, Australia; Research Rheumatology Institute n.a. V.A. Nassonova, Moscow, Russia; Department of Internal Medicine 3, University of Erlangen-Nuremberg, Erlangen, Germany. 4. V.M. Hsu, MD, RWJ Medical School Scleroderma Program; C.P. Denton, MBBS, MRCP, Centre for Rheumatology and Connective Tissue Disease; R.T. Domsic, MD, MPH, Department of Rheumatology, University of Pittsburgh; D.E. Furst, MD, David Geffen School of Medicine at UCLA; M. Rischmueller, MD, FRACP, The Queen Elizabeth Hospital and University of Adelaide; M. Stanislav, MD, PhD, DMSc, Research Rheumatology Institute n.a. V.A. Nassonova; V.D. Steen, MD, Georgetown University Medical Center; J.H. Distler, MD, Department of Internal Medicine 3, University of Erlangen-Nuremberg; S. Korish, MD, Celgene Corp.; A. Cooper, Celgene Corp.; S. Choi, PhD, Celgene Corp.; P.H. Schafer, PhD, Celgene Corp.; G. Horan, PhD, Celgene Corp.; D.R. Hough, MD, Celgene Corp.
Abstract
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.
RCT Entities:
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.
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