frequently relapsing nephrotic syndrome
Conditions
Interventions
Trade Name: Benlysta
Product Name: belimumab
Pharmaceutical Form: Concentrate for solution for infusion
Sponsors
IRCCS Ospedale PedT..trico Bambino Gesù
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: a. Age =3 years and 90 ml/min/1.73 m2, as calculated by the Schwartz formula) d. Remission of INS at enrolment e. Patients that are on maintenance PDN treatment at a dose higher than 10 mg/m2 on alternate days f. Expected compliance from the patient and his parents to the study protocol g. Signed Informed Consent Form (ICF)/Assent by the subject’s parent(s) or legal representative(s). Children will be informed about the study and will be asked to give their assent as appropriate, depending on age. h. If applicable, female participants must have pregnancy test by beta-HCG dosing and be negative. Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: a. Patients suffering from co-morbidities that are not related to INS, including malignancies b. Patients with the following infections: HIV, hepatitis B and c. Patients with any other chronic infectious condition d. Patients with steroid-resistant INS e. Patients that have achieved remission in more than 21 days during their last relapse (i.e. patients at risk of secondary steroid resistance) f. Patients that have relapsed in the past year while receiving more than 30 mg/m2 of PDN on alternate days (i.e. severe steroid dependence that may require immediate initiation of additional immunosuppressive medication) g. Patients that are have been treated with other immunosuppressive drugs in the last 12 months, particularly with drugs that inhibit B cell function (cyclophosphamide, micophenolate mofetil) h. Patients that have been treated with rituximab i. Patients that have received live vaccines in the 30 days previous to study initiation
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary aim of the study is to test the safety of belimumab 10 mg/kg given i.v. at day 0, 14, 28 and subsequently every 4 weeks for a total of 12 months to patients with frequently relapsing forms of nephrotic syndrome, defined as two or more relapses per 6 months (or 4 or more per 12 month period) following the initial therapy or a relapse therapy (Vivarelli et al 2016). ;Secondary Objective: -to evaluate whether the therapeutic use of belimumab is able to prevent disease relapse despite tapering of prednisone in children with frequently relapsing nephrotic syndrome; -to evaluate the immunomodulatory effect of belimumab on B cell subpopulations in children with frequently relapsing nephrotic syndrome. ;Primary end point(s): The primary end-point is the frequency of allergic reactions and other adverse events. Serious adverse events will be recorded from the time of obtaining informed consent throughout the study period. The time stop for collecting evidence will be every 6 months and at 24 months. Subjects who discontinue the study prematurely will continue to be monitored for safety. ;Timepoint(s) of evaluation of this end point: 6 months and at 24 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - the percentage of patients in remission at 6 and 12 months - the total dose of prednisone/kg of body weight administered in the 12 months of treatment with belimumab.;Timepoint(s) of evaluation of this end point: at 6 and 12 months | — |
Countries
Italy
Contacts
Public ContactMarina Vivarelli
OPBG
Outcome results
None listed