Childhood-onset Refractory Nephrotic Syndrome
Conditions
Sponsors
Kobe University Hospital
National Center for Child Medical Health and Development
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Diagnosed as idiopathic nephrotic syndrome (INS) according to the ISKDC criteria 2. The first onset of INS is between 1 - 18 years of age, and 2 years of age or older at entry 3. Subjects meeting either one of the following criteria: 1) having relapsed within 85 days (during Day 1 to Day 85) after receiving "placebo" in the double blind study (a separate study with protocol # RCRNS-01) 2) having diagnosed as frequently relapsing and steroid-dependent INS (FR-SD INS) during the period of Day 86 though Day 365 after receiving the "placebo" in the double blind study (protocol # RCRNS-01) 3) with a history of rituximab treatment for INS in the year of 2007 or before 4. Diagnosed as steroid-sensitive relapse 5. CD20 positive B-cells >/= 5/mcL in the peripheral blood 6. Subjects can be hospitalized for treatments of study drug 7. 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
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 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 IDEC-C2B8 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Investigation of pharmacokinetic profiles | — |
Secondary
| Measure | Time frame |
|---|---|
| 1.Efficacy outcomes (1)Relapse-free duration (2) Relapse rate(number of relapse/subject-year) (3) Rate of relapse subjects (4) Rate of frequently relapsing subjects (5) Rate of steroid-dependent subjects (6) Rate of steroid-resistant subjects (7) Change of steroid quantity administered for prescribed observation period 2.Other outcomes (1)Duration of B-cell depletion (2)Anti-rituximab antibody (human anti-chimeric antibody, HACA) 3. Safety endpoints 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. | — |
Countries
Japan
Contacts
Public ContactManabu Kume
Kobe University Hospital
Outcome results
None listed