steroid and calcineurin inhibitor-dependent idiopatic nephrotic syndrome MedDRA version: 21.1 Level: PT Classification code 10029164 Term: Nephrotic syndrome System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Interventions
Trade Name: ARZERRA - 100 MG - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONE(VETRO) - 5 ML(20MG/ML) 3 FLACONI
Product Name: ofatumumab
Product Code: ofatumumab
Pharmaceutical Form
Sponsors
IRCCS ISTITUTO GIANNINA GASLINI
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -To be in complete disease remission -Drug dependence: remission has to be maintained with both steroids and CNI steroid dependence is defined by two consecutive relapses during corticosteroid therapy or within 14 days of ceasing therapy. CNI (cyclosporine/tacrolimus) dependence is defined by presence of relapse at discontinuation. -Ability to provide consent and assent: 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any study-related procedure not part of the subject’s normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to his or her future medical care.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 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, nDNA, ANCA) -Reduction of C3 levels. -eGFR<90/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. -Pregnancy -Neoplasm -Infections: previous or actual HBV (with HBeAb positivity) or HCV infection -CD20 B lymphocytes count <2,5% -Treatment with Rituximab or cyclophosphamide in the last 6 months -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 )
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: test whether Ofatumumab is superior to Rituximab in maintaining oral drug-free disease remission (complete or partial remission) at 12 months ;Secondary Objective: test whether Ofatumumab is superior to Rituximab in reduce the risk of relapse in a longer follow-up of 24 months in children with steroid and CNI-dependent INS;Primary end point(s): The primary endpoints will be risk of relapse at 12 months without steroid or calcineurin-inhibitors. ;Timepoint(s) of evaluation of this end point: 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoint will be the amount of steroids required to maintain complete disease remission at 6 and 24 months after Ofatumumab or Rituximab pulse.; evaluation of circulating cell populations as biomarkers and predictors of response to treatment with anti-CD20.;Timepoint(s) of evaluation of this end point: 6 months - 24 months ; 6-24 months | — |
Countries
Italy
Contacts
Public ContactUOSD Epidemiologia, biostatistica e
IRCCS G,Gaslini
Outcome results
None listed