Ofatumumab in children with steroid- and calcineurin-inhibitor-resistant nephrotic syndrome: a double-blind randomized, controlled, superiority trial - Ofatumumab in children with steroid - resistant INS

IRCCS ISTITUTO GIANNINA GASLINI0 个研究点目标入组 50 人开始时间: 2020年11月5日最近更新: 2021年1月5日

适应症

试验速览

暂无简介。

•-Drug resistance: it signifies lack of antiproteinuric effect of a double therapy based on steroid plus CNI or mofetil mycophenolate (MMF).
•Steroid resistance is defined by failure to achieve complete remission after 6 weeks with prednisone 60 mg/m2.
•CNI (cyclosporine/tacrolimus) resistance is defined by failure to achieve complete remission within 6 months after the plasma concentration of cyclosporine (started at dosage of 4 mg/kg/day) or tacrolimus (started at dosage of 0,1 mg/kg/day) reached effective plasma concentrations.
•Mofetil Mycophenolate resistance is defined by failure to achieve complete remission after at least 6 months of treatment with 1200mg/mq/day.
•-Parents’/guardian’s written informed consent, and child’s assent given before any study-related procedure not part of the subject’s normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to his or her future medical care.
•-Age between 2 and 18 years
•-Histological pattern of minimal change disease, mesangial proliferation with IgM deposits or focal segmental glomerulosclerosis
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 10
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•-Positivity to autoimmunity tests (ANA, dsDNA, ANCA).
•-Reduction of C3 levels.
•-eGFR < 30 ml/min/1.73 m2 valuated according to revised Bedside Schwartz Formula for patients between 2 and 17 years and with CKD-EPI Creatinine 2009 Equation for 18 years old patients.
•-Hystological pattern characterized by elements suggestive for congenital disease: diffuse mesangial sclerosis without IgM deposits, cystic-like tubular dilatation, mitochondrial abnormalities evident on electron microscopy, IF suggestive for congenital collagen 4 disease.
•-Histological pattern not suitable with INS in the pediatric age (membranous glomerulonephritis, lupus nephritis, diffuse and/or localized vasculitis, amyloidosis)
•-Homozygous or heterozygous mutations of podocitary genes, commonly involved in the etiology of INS (NPHS1, NPHS2, NPHS3, NPHS6, WT1, COQ2, COQ6, MYO1E, SMARCAL1, LAMB2, SCARB2, CD2AP, TRPC6, ACTN4, INF2, LMX1B, MYH9 )
•-Pregnancy
•-Infections: Previous or actual HBV (with HBeAb positivity) or HCV

发起方

IRCCS ISTITUTO GIANNINA GASLINI