UMIN-CTR Clinical Trial

Basic information
Public title	Multi-center clinical trial for Japanese children with bilateral nephroblastoma(RTBL14)
Acronym	Clinical trial for bilateral nephroblastoma
Scientific Title	Multi-center clinical trial for Japanese children with bilateral nephroblastoma(RTBL14)
Scientific Title:Acronym	Clinical trial for bilateral nephroblastoma
Region	Japan

Condition
Condition	nephroblastoma
Classification by specialty	Pediatrics Urology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	This trial is part of the third generation of clinical trials run by the JWiTS group. Wilms tumor (nephroblastoma) is the most common malignant renal neoplasm in children but the incidence is rare (approximately one by several ten thousands children per year). Therefore, 40-50 cases were registered to JWiTS a year. To identify the new effective regimen, nationwide clinical trials are needed. JWiTS was launched at 1996 and have performed JWiTS 1 and JWiTS 2 protocol studies. In these studies, multimodal therapy using actinomycin D, vincristine, and/or doxorubicin and radiation therapy was effective, and overall survival of the patients was improved to nearly 90 %. About 5-10 % of the Wilms tumors are arise bilaterally, and classified as stage V. In the previous studies, the survival of the bilateral cases was improved to around 90%. However in terms of renal preservation, nephrectomy was performed in two thirds of the patients. These results were not satisfactory, therefore new study protocol to improve the renal preservation is necessary to create. In this protocol, chemotherapy is performed first without tumor biopsy to reduce the tumor size. After chemotherapy, nephron-sparing tumor resection will be performed. To identify the resectability, evaluation of the CT images after chemotherapy will be performed by the specialized JWiTS radiologists. The protocol study was opened in 2014, and national-wide multi-center clinical trial is now undergoing.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase	Phase II

Assessment
Primary outcomes	Rate of the bilateral renal preservation at 1 year after registration
Key secondary outcomes	Overall survival, disease free survival, recurrence rate, disease specific survival Rate of hemilateral/bilateral nephrectomy Rate of renal dysfunction Rate of renal failure Rate of toxicities Evaluation of pathological findings after chemotherapy by JWiTS specified pathologists Response rate to neo-adjuvant chemotherapy evaluated by the specified radiologists.

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Historical
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Chemotherapy regimen DD-4A using actinomycin D (ACD), vincristine (VCR) and are administered for 6 or 12 weeks. ACD iv for 0.045 mg/kg on week 1, 7, 13, 19 and 25. VCR iv for 0.05mg/kg is started at week 2 and continued every week. DOX iv for 1.5mg/kg on Week 4 and 10, and 1.0mg/kg on week 16 and 22. The tumor size is measured by image analysis at week 6 and 12. If nephron sparing surgery is possible, the tumor will be completely resected and post-operative chemotherapy and/or radiotherapy will be performed according to the operational findings and pathological diagnosis.
Interventions/Control_2
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	0 years-old <=
Age-upper limit	18 years-old >
Gender	Male and Female
Key inclusion criteria	1) Radiologicaly confirmed bilateral nephroblastoma. If pathological diagnosis is different, the patient will be excluded after surgery. 2) Age < 18 years 3) No previous chemotherapy, radiotherapy or surgery 4) Written informed consent and national/local ethics committee and regulatory approval 5) Ability to comply with requirements for submission of material for central review (radiology, and pathology)
Key exclusion criteria	Cases with one of the following factors 1)Recurrent disease 2)Active double cancer, synchronous or remission less than 5 years 3)Performance Status (ECOG/Zubrod Score) is 4 (Completely disabled. Cannot carry on any selfcare. Totally confined to bed or chair) 4)Severe organ failure (hematologic, liver, kidney, heart) White blood cells < 2000/mm3 Platelets <100,000/mm3 Serum bilirubin > 2.5 x normal limit Serum ALT > 2.5 x normal limit Serum total bile acid > 2.5 x normal limit Serum creatinine > 2.5 x normal limit 5) Patient unable to undergo chemotherapy due to other disease, or the attending doctor considers inappropriate to entry this study. 6)The patient has a psychological disorder so that unable for entry. 7)Patient unable to tolerate the chemotherapy for any reason
Target sample size	17

Research contact person
Name of lead principal investigator	1st name Middle name Last name Takaharu Oue
Organization	Hyogo College of Medicine
Division name	Pediatric Surgery
Zip code
Address	1-1, Mukogawa-cho, Nishinomiya, Hyogo
TEL	0798-45-6582
Email	ta-oue@tho-med.ac.jp

Public contact
Name of contact person	1st name Middle name Last name Takaharu Oue
Organization	Hyogo College of Medicine
Division name	Pediatric Surgery
Zip code
Address	1-1, Mukogawa-cho, Nishinomiya, Hyogo
TEL	0798-45-6582
Homepage URL	http://jwits.umin.ac.jp/
Email	ta-oue@hyo-med.ac.jp

Sponsor
Institute	Japan Children's Cancer Group(JCCG) (Japan Wilms Tumor Study Group, JWiTS)
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization
Nationality of Funding Organization	Japan

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

IRB Contact (For public release)
Organization
Address
Tel
Email

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	兵庫医科大学病院（兵庫県）他１１７施設

Other administrative information
Date of disclosure of the study information	2016 Year 01 Month 26 Day

Related information
URL releasing protocol	http://jwits.umin.ac.jp/
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	Enrolling by invitation
Date of protocol fixation	2014 Year 09 Month 30 Day
Date of IRB	2014 Year 10 Month 01 Day
Anticipated trial start date	2014 Year 10 Month 01 Day
Last follow-up date	2020 Year 09 Month 30 Day
Date of closure to data entry	2020 Year 12 Month 31 Day
Date trial data considered complete	2020 Year 12 Month 31 Day
Date analysis concluded	2021 Year 12 Month 31 Day

Other
Other related information

Management information
Registered date	2016 Year 01 Month 26 Day
Last modified on	2022 Year 08 Month 01 Day

Link to view the page
URL(English)	https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000023946