泊马度胺治疗系统性硬化症相关间质性肺病患者的 II 期、多中心、随机、双盲、安慰剂对照、平行分组研究。
Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study.
机构信息
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.
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.
出版信息
J Rheumatol. 2018 Mar;45(3):405-410. doi: 10.3899/jrheum.161040. Epub 2017 Nov 1.
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.
目的
评估泊马度胺（POM）在治疗系统性硬化症（SSc）相关间质性肺病患者 52 周期间对用力肺活量（FVC）、改良 Rodnan 皮肤评分（mRSS）和胃肠道（GI）症状的安全性和疗效。
方法
23 例成人 SSc 患者按 1:1 随机分配接受 POM：安慰剂（PBO）。
结果
与基线相比，第 52 周时预测 FVC%的平均变化分别为-5.2 和-2.8；mRSS 分别为-2.7 和-3.7；以及 UCLA 硬皮病临床试验联盟胃肠道（SCTC GIT 2.0）评分分别为 0.1 和 0.0，POM 和 PBO 组分别为 0.1 和 0.0。在第 52 周时，这 3 个主要终点均未达到统计学意义。
结论
由于招募挑战，提前停止了患者入组。在中期分析中，该研究未达到第 52 周的主要终点。因此，决定终止所有研究阶段。POM 总体耐受性良好，不良事件谱与 POM 在其他疾病中的已知安全性和耐受性谱一致。由于纳入的受试者太少，无法得出有意义的结论，因此研究结果既不是阳性也不是阴性。临床试验编号：NCT01559129。
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