Clinical trial NCT01716442: Rituximab Trial for Pediatric Nephrotic Syndrome
Official title: A Multi-center Randomized Controlled Trial of Rituximab for Refractory Nephrotic Syndrome in Children
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Steroid Resistant Nephrotic Syndrome
Steroid Dependent Nephrotic Syndrome
Keywords: rituximab
nephrotic
refractory
Lead sponsor: Seoul National University Childrens Hospital
Sponsor class: OTHER
First posted: 2012-10-29
Enrollment: 88
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Anti-CD20 agent has been proposed as a rescue therapy for refractory nephrotic syndrome(NS) on the basis of favorable clinical observations. Yet the long-term effect on maintaining remission or the likelihood of becoming rituximab-dependent is unclear and the information on the safety profile of rituximab is limited. This trial was designed to investigate the safety and efficacy of Rituximab in children with refractory NS.


Eligibility criteria: Inclusion Criteria:

(A)steroid/calcineurin inhibitor resistant nephrotic syndrome

1. steroid resistance: no remission after 4 weeks of daily oral corticosteroid (60mg/m2/day)
2. calcineurin inhibitor resistance: no remission after 3 months of therapeutic dose administration of cyclosporine and/or tacrolimus
3. no remission defined by persistent proteinuria of nephrotic range for the last 3 months
4. post-transplant patients were included in the study

(B)steroid-dependent nephrotic syndrome

1. Steroid-dependent patients who had been on various known medications (such as corticosteroids, cyclophosphamide, chlorambucil, calcineurin inhibitors, levamisole ..) continuously for more than 2 years
2. definition of dependency: more than two consecutive relapse events in 2 weeks after discontinuation of steroid or calcineurin inhibitor

   * no improvement in relapsing frequency with calcineurin inhibitor use
   * unable to continue with calcineurin inhibitor due to side effects
   * unable to continue with calcineurin inhibitor due to prolonged use (over 2 years)
   * other conditions in which the clinician considers difficult to control disease with steroids or calcineurin inhibitors only.

Exclusion Criteria:

* previous rituximab use
* secondary nephrotic syndrome
* estimated GFR \<60mL/min/1.73m2 or under 50% of age-matched standard GFR
* chronic or acute active infection (e.g. hepatitis B,C, herpes, varicella zoster)
* prior live vaccine inoculation within 1 month (from the study enrollment)
* cardiovascular diseases, pulmonary or pleural diseases
* uncontrolled hypertension
* leukocytopenia (absolute neutrophil count \<1500/mm3) or thrombocytopenia (\<75000/mm3)
* pregnancy

Primary outcomes:
measure: The remission rate of steroid-resistant nephrotic syndrome patients after rituximab treatment
timeFrame: within 6 months from the initiation of treatment
measure: The rate of maintaining remission in steroid-dependent nephrotic syndrome patients
timeFrame: within 6 months from the initiation of treatment versus placebo control
Secondary outcomes:
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Seoul National University Children's Hospital
Study officials:
affiliation: Seoul National University Children's Hospital
name: Hee Gyung Kang A. Kang, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: