NCT02394106

Ofatumumab in Children With Drug Resistant Idiopathic Nephrotic Syndrome

Ofatumumab in Children With Steroid- and Calcineurin-inhibitor-resistant Nephrotic Syndrome: a Double-blind Randomized, Controlled, Superiority Trial

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Istituto Giannina Gaslini · Academic / Other
• Sex: All
• Age: 2 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

Double-blind, two-parallel-arm, placebo-controlled randomized clinical trial testing the superiority of Ofatumumab versus placebo in the treatment of children with DR-INS. Participants will be stratified according to eGFR at enrollment. Eligible participants will enter a 3-months run-in period, during which instructions on urine collection and dipstick readings will be carefully reviewed, compliance assessed and any immunosuppressive therapies withdrawn according to the following schemes: * prednisone will be tapered off by 0.3 mg/kg per week until complete withdrawal; * calcineurin inhibitors and mofetile mycophenolate will be decreased by 50% and withdrawn after 2 additional weeks In order to minimize the risk of complications of uncontrolled INS a treatment with ACE-inhibitor at 6 mg/m2 will be maintained or started in all patients. After run-in period, children will be randomized to the intervention arm (Ofatumumab) or comparator arm (placebo). Randomization will be stratified by eGFR at randomization: ≥90 and \<90 ml/min/1.73 m2. All patients will be followed up to 12 months and they will leave the study at time of relapse. Relapse will be defined as uPCR ≥2000 mg/g (≥200 mg/mmol) or ≥ 3+ protein on urine dipstick for 3 consecutive days.

Conditions

• Nephrotic Syndrome

Interventions

Type	Name	Description
DRUG	Ofatumumab	Ofatumumab 1500 mg/1.73m2 administered once, diluted in 1000 ml of normal saline
OTHER	Placebo	Normal saline, 1000 ml, administered once

Timeline

• Start date: 2015-07-01
• Primary completion: 2019-06-01
• Completion: 2019-06-01
• First posted: 2015-03-20
• Last updated: 2020-07-29

Locations

1 site across 1 country: Italy