UMIN-CTR Clinical Trial

Basic information
Public title	Mycophenolate mofetil for childhood-onset, complicated, frequently relapsing nephrotic syndrome or steroid-dependent nephrotic syndrome: a multicentre, double-blind, randomised, placebo-controlled trial(JSKDC07)
Acronym	Mycophenolate mofetil for childhood-onset, complicated, frequently relapsing nephrotic syndrome or steroid-dependent nephrotic syndrome: a multicentre, double-blind, randomised, placebo-controlled trial(JSKDC07)
Scientific Title	Mycophenolate mofetil for childhood-onset, complicated, frequently relapsing nephrotic syndrome or steroid-dependent nephrotic syndrome: a multicentre, double-blind, randomised, placebo-controlled trial(JSKDC07)
Scientific Title:Acronym	Mycophenolate mofetil for childhood-onset, complicated, frequently relapsing nephrotic syndrome or steroid-dependent nephrotic syndrome: a multicentre, double-blind, randomised, placebo-controlled trial(JSKDC07)
Region	Japan

Condition
Condition	Childhood-onset, complicated, frequently relapsing nephrotic syndrome or steroid-dependent nephrotic syndrome
Classification by specialty	Nephrology Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the efficacy and safety of mycophenolate mofetil in comparison with placebo as measured by he time to treatment failure in patients with childhood-onset, complicated, frequently relapsing nephrotic syndrome or steroid-dependent nephrotic syndrome.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2
Developmental phase	Phase III

Assessment
Primary outcomes	The time to treatment failure
Key secondary outcomes	Relapse free period, Time to SDNS, Time to FRNS, Time to SRNS, B cell depletion period, Daily steroid dose

Base
Study type	Interventional

Study design
Basic design	Parallel
Randomization	Randomized
Randomization unit	Individual
Blinding	Double blind -all involved are blinded
Control	Placebo
Stratification	NO
Dynamic allocation	YES
Institution consideration	Institution is considered as adjustment factor in dynamic allocation.
Blocking	NO
Concealment	Central registration

Intervention
No. of arms	2
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Rituximab and mycophenolate mofetil
Interventions/Control_2	rituximab and mycophenolate mofetil placebo
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	2 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key 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 assignment. 3.Patients meeting either one of the following criteria: 1) Diagnosed with frequent relapse or steroid dependence and once again diagnosed with frequent relapse or steroid dependence after completion of immunosuppressive drug therapy (cyclosporine, cyclophosphamide, or mizoribine, etc.). 2) Diagnosed with frequent relapse or steroid dependence and once again diagnosed with frequent relapse or steroid dependence during immunosuppressive drug therapy (cyclosporine, cyclophosphamide, or mizoribine, etc.). 3) Diagnosed with steroid resistance following the onset of INS anf diagnosed with frequent relapse or steroid dependence during or after the completion of immunosuppressive drug therapy (cyclosporine alone or combination of cyclosporine and methylprednisolone, etc.). 4.Patients with records of nearest preceding 3 relapses. 5.Patients in whom steroid sensitivity is observed during treatment of relapse immediately prior to assignment. 6.Patients in whom 5/microL or more CD20-positive cells are observed in the peripheral blood. 7.Patients who can be hospitalized overnight on the first day of rituximab administration. 8.Written informed consent.
Key exclusion criteria	1.Patients who have been diagnosed with nephritic-NS, such as IgA nephropathy prior to assignment or in whom secondary NS is suspected. 2.Patients meeting either one of the following infection: 1) Presence or history of severe infections within 6 months prior to assignment. 2) Presence or history of opportunistic infections within 6 months prior to assignment. 3) Presence of active tuberculosis. 4) Patients with a history of tuberculosis or in whom tuberculosis is suspected. 5) Presence or history of active Hepatitis B or Hepatitis C or hepatitis B virus carrier. 6) Presence of HIV infection. 3.Presence or history of angina pectoris, cardiac failure, myocardial infarction, or serious arrhythmia (findings observed under Grade 4 of the CTCAE v4.0-JCOG. 4.Presence or history of auto-immune diseases or vascular purpura. 5.Presence or history of malignant tumor. 6.History of organ transplantation. 7.History of drug allergies to methylprednisolone, acetaminophen, or d-chlorpheniramine maleate. 8.Uncontrollable hypertension. 9.Deteriorated kidney function, e.g. estimated GFR<60 mL/min./1.73m2. 10.Having received a live vaccine within 4 weeks prior to enrollment. 11.Patients showing either one of the following abnormal clinical laboratory value: 1)WBC <3,000/microL. 2)neutrophil <1,500/microL. 3)PLT <50,000/microL. 4)ALT >2.5 x upper limit of normal value. 5)AST >2.5 x upper limit of normal value. 6)Positive for HBs antigen, HBs antibody, HBc antibody and HCV antibody. 7)Positive for HIV antibody. 12.Patients who do not agree with contraception during the study period. 13.Women during pregnancy or breast-feeding. 14.Judged inap.propriate for this study by the physicians.
Target sample size	80

Research contact person
Name of lead principal investigator	1st name Kazumoto Middle name Last name Iijima
Organization	Kobe University Graduate School of medicine
Division name	Division of Child Health and Development
Zip code	650-0017
Address	5-1 Kusunoki-cho 7 chome
TEL	078-382-6093
Email	iijima@med.kobe-u.ac.jp

Public contact
Name of contact person	1st name Mayumi Middle name Last name Sako
Organization	National Center for Child Health and Development
Division name	Division for Clinical Trials
Zip code	157-8535
Address	2-10-1 Ookura, Setagaya-ku, TOKYO
TEL	03-3416-0181
Homepage URL
Email	sako-m@ncchd.go.jp

Sponsor
Institute	Japanese Study Group of Kidney Disease in Children
Institute
Department

Funding Source
Organization	AMED
Organization
Division
Category of Funding Organization	Government offices of other countries
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization	Kobe University Clinical Research Ethical Committee
Address	5-1 Kusunoki-cho 7 chome
Tel	078-382-6669
Email	cerb@med.kobe-u.ac.jp

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2014 Year 06 Month 23 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Main results already published
Date of protocol fixation	2014 Year 04 Month 10 Day
Date of IRB	2014 Year 07 Month 16 Day
Anticipated trial start date	2015 Year 05 Month 21 Day
Last follow-up date	2019 Year 10 Month 16 Day
Date of closure to data entry
Date trial data considered complete	2020 Year 03 Month 31 Day
Date analysis concluded	2020 Year 06 Month 30 Day

Other
Other related information

Management information
Registered date	2014 Year 06 Month 23 Day
Last modified on	2021 Year 12 Month 28 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000016599