A phase 2, double-blind, placebo-controlled, randomized study of fresolimumab in patients with steroid-resistant primary focal segmental glomerulosclerosis
dc.contributor.author | Vincenti, Flavio | pt_BR |
dc.contributor.author | Fervenza, Fernando Custodio | pt_BR |
dc.contributor.author | Campbell, Kirk Nicholas | pt_BR |
dc.contributor.author | Díaz, Montserrat M. | pt_BR |
dc.contributor.author | Gesualdo, Loreto | pt_BR |
dc.contributor.author | Nelson, Peter | pt_BR |
dc.contributor.author | Praga, Manuel | pt_BR |
dc.contributor.author | Radhakrishnan, Jai | pt_BR |
dc.contributor.author | Sellin, Lorenz K. | pt_BR |
dc.contributor.author | Singh, Ajay | pt_BR |
dc.contributor.author | Thornley-Brown, Denyse P. | pt_BR |
dc.contributor.author | Veronese, Francisco José Veríssimo | pt_BR |
dc.contributor.author | Accomando, Beverly | pt_BR |
dc.contributor.author | Engstrand, Sara | pt_BR |
dc.contributor.author | Ledbetter, Steven R. | pt_BR |
dc.contributor.author | Lin, Julie | pt_BR |
dc.contributor.author | Neylan, John F. | pt_BR |
dc.contributor.author | Tumlin, James A. | pt_BR |
dc.contributor.author | Focal Segmental Glomerulosclerosis Study Group | pt_BR |
dc.date.accessioned | 2020-02-01T04:14:19Z | pt_BR |
dc.date.issued | 2017 | pt_BR |
dc.identifier.issn | 2468-0249 | pt_BR |
dc.identifier.uri | http://hdl.handle.net/10183/205292 | pt_BR |
dc.description.abstract | Introduction: Steroid-resistant focal segmental glomerulosclerosis (SR-FSGS) is a common glomerulopathy associated with nephrotic range proteinuria. Treatment goals are reduction in proteinuria, which can delay end-stage renal disease. Methods: Patients with SR-FSGS were enrolled in a randomized, double-blind placebo-controlled trial of fresolimumab, a monoclonal anti transforming growth factor b antibody, at 1 mg/kg or 4 mg/kg for 112 days, followed double-blind for 252 days (NCT01665391). The primary efficacy endpoint was the percentage of patients achieving partial (50% reduction) or complete (< 300 mg/g Cr) remission of proteinuria. Results: Of 36 enrolled patients, 10, 14, and 12 patients received placebo, fresolimumab 1 mg/kg, and fresolimumab 4 mg/kg, respectively. The baseline estimated glomerular filtration rate (eGFR) and urinary protein/creatinine ratio were 63 ml/min/1.73 m2 and 6190 mg/g, respectively. The study was closed before reaching its target of 88 randomized patients. None of the prespecified efficacy endpoints for proteinuria reduction were achieved; however, at day 112, the mean percent change in urinary protein/creatinine ratio (a secondary efficacy endpoint) was –18.5% (P ¼ 0.008), þ10.5% (P ¼ 0.52), and þ9.0% (P ¼ 0.91) in patients treated with fresolimumab 1 mg/kg, fresolimumab 4 mg/kg, and placebo, respectively. There was a nonsignificant trend toward greater estimated glomerular filtration rate decline in the placebo group compared to either of the fresolimumab-treated arms up to day 252. Discussion: The study was underpowered and did not meet the primary or secondary endpoints. However, fresolimumab was well tolerated and is appropriate for continued evaluation in larger studies with adequate power. | en |
dc.format.mimetype | application/pdf | pt_BR |
dc.language.iso | eng | pt_BR |
dc.relation.ispartof | Kidney international reports. Philadelphia. Vol. 2, no. 5 (Sept. 2017), p. 800-810 | pt_BR |
dc.rights | Open Access | en |
dc.subject | Glomerulosclerose segmentar e focal | pt_BR |
dc.subject | Fresolimumab | en |
dc.subject | Anticorpos monoclonais | pt_BR |
dc.subject | Monoclonal antibody | en |
dc.subject | Proteinuria | en |
dc.subject | Proteinúria | pt_BR |
dc.subject | Ensaio clínico controlado aleatório | pt_BR |
dc.subject | Steroid-resistant primary focal segmental glomerulosclerosis | en |
dc.subject | Ensaio clínico fase II | pt_BR |
dc.subject | Método duplo-cego | pt_BR |
dc.subject | Placebos | pt_BR |
dc.title | A phase 2, double-blind, placebo-controlled, randomized study of fresolimumab in patients with steroid-resistant primary focal segmental glomerulosclerosis | pt_BR |
dc.type | Artigo de periódico | pt_BR |
dc.identifier.nrb | 001107587 | pt_BR |
dc.type.origin | Estrangeiro | pt_BR |
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