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Double-Blind, Randomized Study of IDEC-C2B8 in Patients with Childhood-onset Complicated Frequently Relapsing or Steroid-dependent Nephrotic Syndrome

Phase 3
Completed
Conditions
Childhood-onset Complicated Nephrotic Syndrome
Registration Number
JPRN-jRCT2091220020
Lead Sponsor
Kobe University Hospital
Brief Summary

We have shown that the relapse-free period increases with rituximab in patients with childhood-onset, complicated FRNS and SDNS. Adverse events were generally mild and the frequency of serious adverse events did not differ significantly between groups. Rituximab is an effective and safe treatment for childhood-onset, complicated FRNS and SDNS.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
60
Inclusion Criteria

1. Diagnosed as idiopathic nephrotic syndrome (INS) according to the ISKDC criteria.
2. Subjects meeting either one of the following criteria:
1) Diagnosed as frequently relapsing or steroid-dependent INS again after immunosuppressant treatment such as cyclosporine A, cyclophosphamide and/or mizoribine for previous frequently relapsing or steroid-dependent NS.
2) Diagnosed as frequently relapsing or steroid-dependent INS during immunosuppressant treatment such as cyclosporine A, cyclophosphamide and/or mizoribine for previous frequently relapsing or steroid-dependent NS.
3) Diagnosed as frequently relapsing or steroid-dependent INS after or during treatment with cyclosporine A alone or in combination with methylprednisolone for previous steroid-resistant NS which was initially diagnosed as NS.
3. First onset of INS is between 1 - 18 years of age, and 2 years of age or older at entry.
4. Subjects with records of nearest preceding 3 relapses
5. Diagnosed as steroid-sensitive relapse
6.CD20 positive B-cells >/= 5/mcL in the peripheral blood
7. Subjects can be hospitalized for treatments
8. Subjects have ability to provide written informed consent form. The written informed consent form of legal representative (i.e., a parent or a legal benefactor) is also required for subjects less than 20 years of age.

Exclusion Criteria

1. History of inflammatory nephritis such as IgA nephritis.
2. History of immunosuppressive treatment for INS other than cyclosporine A, cyclophosphamide, mizoribine, mycophenolate mofetil and chlorambucil
3. History of serious infectious diseases such as pulmonary tuberculosis and deep-seated fungal diseases
4. Positive for HCV antibody, HBs antigen, HBc antibody and HIV antibody
5. Having received a live vaccine within 4 weeks prior to screening
6.Known hypertension which is uncontrollable with conventional anti-hypertensive
7. Deteriorated kidney function, e.g. estimated GFR<60mL/min./1.73m2
8. Deteriorated liver function, e.g., AST or ALT > 2.5 x upper limit of normal value
9. Presence or history of angina pectoris, cardiac failure, myocardial infarction and/or serious arrhythmia grade 4 in Common Terminology Criteria for Adverse Events (ver. 3.0)
10. Presence or history of auto-immune diseases such as Hashimoto disease (struma lymphomatosa), Crohn's disease, ulcerative colitis, rheumatoid arthritis, idiopathic thrombocytopenic purpura, systemic lupus erythematosus, autoimmune hemolytic anemia, scleroderma or vascular purpura
11. Presence or history of cancer
12. History of organ transplantation
13. History of allergic reaction to acetaminophen and/or d-chlorpheniramine maleate
14. Presence of hematological disorder, e.g., WBC < 2,000/mcL, neutrophil < 1,500/mcL, PLT < 50,000/mcL
15. History of receiving any kinds of monoclonal antibody therapy
16. Having received any investigational agent within 6 months prior to enrollment, or participating other clinical studies
17. Pregnant subjects or subjects who do not agree with contraception during the study period (Negative HCG result is required at screening for female subjects after the first menstruation.)
18.Judged inappropriate for this study by the physicians

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
1. Efficacy outcome<br> Relapse-free period<br>2. Safety endpoints <br>The type, severity, duration and frequency of AEs reported during the period from the first administration of study drug (Day 1) to the post-study examination.
Secondary Outcome Measures
NameTimeMethod
1. Efficacy outcomes<br>(1) Time to treatment failure<br>(2) Relapse rate (number of relapse per person-year)<br>(3) Time to frequently relapsing NS<br>(4) Time to steroid-dependent NS<br>(5) Time to transition to steroid resistance<br>(6) Steroid dose after randomisation<br>(7) Changes in steroid dose before and after randomisation<br>2. Other outcomes <br>(1)Serum IDEC-C2B8 level<br>(2)Duration of B-cell depletion<br>(3)Anti-rituximab antibody (human anti-chimeric antibody, HACA) in subject serum
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