Ofatumumab in Children With Drug Resistant Idiopathic Nephrotic Syndrome
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|ClinicalTrials.gov Identifier: NCT02394106|
Recruitment Status : Unknown
Verified May 2017 by Gian Marco Ghiggeri MD, PhD, Istituto Giannina Gaslini.
Recruitment status was: Recruiting
First Posted : March 20, 2015
Last Update Posted : May 4, 2017
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collection and dipstick readings will be carefully reviewed, compliance assessed and any immunosuppressive therapies withdrawn according to the following schemes: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- calcineurin inhibitors and mofetile mycophenolate will be decreased by 50% and withdrawn after 2 additional weeks In order to minimize the risk of complications of uncontrolled INS a treatment with ACE-inhibitor at 6 mg/m2 will be maintained or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.
|Condition or disease||Intervention/treatment||Phase|
|Nephrotic Syndrome||Drug: Ofatumumab Other: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Ofatumumab in Children With Steroid- and Calcineurin-inhibitor-resistant Nephrotic Syndrome: a Double-blind Randomized, Controlled, Superiority Trial|
|Study Start Date :||July 2015|
|Estimated Primary Completion Date :||September 2017|
|Estimated Study Completion Date :||March 2018|
Ofatumumab 1500 mg/1.73m2 administered once, diluted in 1000 ml of normal saline
Other Name: Arzerra
Placebo Comparator: Placebo
Normal saline, 1000 ml, administered once
Other Name: Normal saline
- Complete or partial disease remission [ Time Frame: 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 (according to KDIGO Clinical Practice Guideline for Glomerulonephritis)
- Complete or partial disease remission [ Time Frame: 12 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 (according to KDIGO Clinical Practice Guideline for Glomerulonephritis)
- Adverse events [ Time Frame: At 1, 3, 6, 9 and 12 months after drug/placebo infusion, during protocol visits ]Measurement of frequency and severity of adverse events due to drug infusion
- Abnormal laboratory values [ Time Frame: At 1, 3, 6, 9 and 12 months after drug/placebo infusion, during protocol visits ]Record of abnormal values in biochemical tests and hematology assessments.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02394106
|Contact: Gianmarco Ghiggeri, MD||+39 010 firstname.lastname@example.org|
|IRCCS Istituto Giannina Gaslini||Recruiting|
|Genoa, Italy/GE, Italy, 16147|
|Contact: Gian Marco Ghiggeri, MD 0039 010 5636 2419 email@example.com|
|Principal Investigator: Gian Marco Ghiggeri, MD|
|Sub-Investigator: Alice Bonanni, MD|
|Principal Investigator:||Gianmarco Ghiggeri, MD||Istituto Giannina Gaslini|