Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study
| Autor: | Robyn T. Domsic, Virginia D. Steen, Vivien Hsu, Daniel E. Furst, Peter H. Schafer, Alyse Cooper, Shimon Korish, Gerald Horan, Douglas R. Hough, Suktae Choi, Marina Stanislav, Maureen Rischmueller, Christopher P. Denton, Jörg H W Distler |
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| Rok vydání: | 2017 |
| Předmět: |
0301 basic medicine
Adult Male Vital capacity medicine.medical_specialty Immunology Vital Capacity Placebo 03 medical and health sciences FEV1/FVC ratio 0302 clinical medicine Rheumatology Double-Blind Method Internal medicine Forced Expiratory Volume Immunology and Allergy Medicine Humans Immunologic Factors Lung Skin 030203 arthritis & rheumatology Scleroderma Systemic business.industry Interstitial lung disease Middle Aged medicine.disease Interim analysis Pomalidomide Fibrosis Thalidomide Clinical trial 030104 developmental biology Treatment Outcome Tolerability Early Termination of Clinical Trials Physical therapy Female business Lung Diseases Interstitial medicine.drug Follow-Up Studies |
| Zdroj: | The Journal of rheumatology. 45(3) |
| ISSN: | 0315-162X |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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