Efficacy of Ofatumumab in children with steroid-dependent nephrotic syndrome a

Efficacy of humanized anti-CD20 antibodies (Ofatumumab) in the treatment of childhood steroid-dependent nephrotic syndrome and development of cell biomarkers predicting outcome. - The OFA3 trial

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-001042-33-IT

Enrollment

160

Registered

2018-10-11

Start date

Unknown

Completion date

Unknown

Last updated

2025-02-03

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

Conditions

steroid dependent nephrotic syndrome MedDRA version: 20.0 Level: HLT Classification code 10018365 Term: Glomerulonephritis and nephrotic syndrome System Organ Class: 100000004857

Interventions

Trade Name: Arzerra Product Name: ofatumumab Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: OFATUMUMAB CAS Number: 679818-59-8 Concentration unit: mg/ml milligram(s)/m

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Age between 3 and 24 years - Prednison dependent steroid syndrome 0.3-1mg/Kg/day and receive prednisone for at least six months before enrolment. Steroid dependence is defined by two consecutive relapse during corticosteroid therapy or within 14 days of ceasing therapy. - 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any study-related procedure not part of the subject’s normal medical care, with the understanding that consent may be withdrawn by the subject any time without prejudice to his or her future medical care. Are the trial subjects under 18? yes Number of subjects for this age range: 120 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 40 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

Design outcomes

Primary

Measure	Time frame
Main Objective: to test whether Ofatumumab is able to achieve and maintain drug-free disease remission in patients with steroid-dependent nephrotic syndrome at 12 months. This outcome will be compared to MMF, the drug recommended by Clinical Practice Guidelines (KDIGO) as benchmark of the treatment. The objective of the proposal is therefore to test whether Ofatumumab is superior to MMF in maintaining oral drug-free disease remission (complete or partial remission) for 12months in patients with SDNS.;Secondary Objective: To reduce the risk of relapse in a longer follow-up of 24 months in the same group of patients. To verify the occurrence of side-effects in the short (30 days), mid (90 days) and long-term periods (2 years). To analyze B and T cell lymphocyte subsets pre and post-therapy with Ofatumumab in order to study reconstitution of immune competence in these patients versus patients treated with MMF and to identify biomarkers of response to Ofatumumab and predictors of disease relapse. ;Primary end point(s): Risk relapse within 12 months without steroid administration;Timepoint(s) of evaluation of this end point: 12 months	—

Measure

Time frame

Main Objective: to test whether Ofatumumab is able to achieve and maintain drug-free disease remission in patients with steroid-dependent nephrotic syndrome at 12 months. This outcome will be compared to MMF, the drug recommended by Clinical Practice Guidelines (KDIGO) as benchmark of the treatment. The objective of the proposal is therefore to test whether Ofatumumab is superior to MMF in maintaining oral drug-free disease remission (complete or partial remission) for 12months in patients with SDNS.;Secondary Objective: To reduce the risk of relapse in a longer follow-up of 24 months in the same group of patients. To verify the occurrence of side-effects in the short (30 days), mid (90 days) and long-term periods (2 years). To analyze B and T cell lymphocyte subsets pre and post-therapy with Ofatumumab in order to study reconstitution of immune competence in these patients versus patients treated with MMF and to identify biomarkers of response to Ofatumumab and predictors of disease relapse. ;Primary end point(s): Risk relapse within 12 months without steroid administration;Timepoint(s) of evaluation of this end point: 12 months

—

Secondary

Measure	Time frame
Secondary end point(s): evaluation of circolant cellular popolations as biomarkers or predictors of treatment with anti CD20;Timepoint(s) of evaluation of this end point: 0, 1, 3, 6, 12, 18, 24 months	—

Countries

Italy

Contacts

Public ContactStefania Gamba

IRCCS Istituto Giannina Gaslini

stefaniagamba@gaslini.org003901056363407

Outcome results

None listed

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