Ofatumumab in children with nephrotic syndrome resistant to common therapy

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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-000569-30-IT

Enrollment

Registered

2020-11-05

Start date

2015-06-16

Completion date

Unknown

Last updated

2025-01-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Steroid- and calcineurin-inhibitor-resistant nephrotic syndrome MedDRA version: 21.0 Level: LLT Classification code 10072914 Term: Steroid-resistant nephrotic syndrome System Organ Class: 100000004857

Interventions

Trade Name: ARZERRA - 100 MG - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONE(VETRO) - 5 ML(20MG/ML) 3 FLACONI Product Name: ofatumumab Pharmaceutical Form: Concentrate for solutio

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: -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any study-related procedure not part of the subject’s normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to his or her future medical care.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

Exclusion criteria

Exclusion criteria: -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 -Neoplasm -Infections: Previous or actual HBV (with HBeAb positivity) or HCV

Design outcomes

Primary

Measure	Time frame
Main Objective: to demonstrate that Ofatumumab can induce stable complete or partial remission without other drugs within 6 months of infusion and for at least 12 months thereafter. We will test whether Ofatumumab is superior to placebo in reducing proteinuria within 6 months and for at least one year in DR-INS patients. ;Secondary Objective: Exclusion of acute and long-term side effects is an important secondary objective;Primary end point(s): Complete or partial disease remission at 6 months from randomization. Complete remission in defined by urinary protein/creatinine ratio (uPCR) <200 mg/g (<20mg/mmol) for 3 consecutive days. Partial remission is defined as proteinuria reduction of 50% or greater from the presenting value and absolute uPCR between 200 and 2000 mg/g. for 3 consecutive days.;Timepoint(s) of evaluation of this end point: 6 months	—

Measure

Time frame

Main Objective: to demonstrate that Ofatumumab can induce stable complete or partial remission without other drugs within 6 months of infusion and for at least 12 months thereafter. We will test whether Ofatumumab is superior to placebo in reducing proteinuria within 6 months and for at least one year in DR-INS patients. ;Secondary Objective: Exclusion of acute and long-term side effects is an important secondary objective;Primary end point(s): Complete or partial disease remission at 6 months from randomization. Complete remission in defined by urinary protein/creatinine ratio (uPCR) <200 mg/g (<20mg/mmol) for 3 consecutive days. Partial remission is defined as proteinuria reduction of 50% or greater from the presenting value and absolute uPCR between 200 and 2000 mg/g. for 3 consecutive days.;Timepoint(s) of evaluation of this end point: 6 months

—

Secondary

Measure	Time frame
Secondary end point(s): complete or partial disease remission at 12 months from randomization; ;Timepoint(s) of evaluation of this end point: 12 months	—

Countries

Italy

Contacts

Public ContactUOSD Epidemiologia, biostatistica e

IRCCS Giannina Gaslini

comitatoetico@ospedale-gaslini.ge.it01056363462

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026