UMIN-CTR Clinical Trial

Basic information
Public title	Prospective study of I-131 3-iodobenzylguanidine radiotherapy for high-risk neuroblastoma; in accordance with the Japanese Advanced Medical Care B program for the anticancer drug with high medical needs.
Acronym	Prospective study of I-131 3-iodobenzylguanidine radiotherapy for high-risk neuroblastoma; in accordance with the Japanese Advanced Medical Care B program for the anticancer drug with high medical needs.
Scientific Title	Prospective study of I-131 3-iodobenzylguanidine radiotherapy for high-risk neuroblastoma; in accordance with the Japanese Advanced Medical Care B program for the anticancer drug with high medical needs.
Scientific Title:Acronym	Prospective study of I-131 3-iodobenzylguanidine radiotherapy for high-risk neuroblastoma; in accordance with the Japanese Advanced Medical Care B program for the anticancer drug with high medical needs.
Region	Japan

Condition
Condition	Neuroblastoma
Classification by specialty	Pediatrics
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the safety and efficacy of I-131 3-iodobenzylguanidine (131I-MIBG) radiotherapy when administered prior to high-dose chemotherapy and hematopoietic stem cell transplantation as a consolidation therapy for high-risk neuroblastoma.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Dose limiting toxicity (DLT).
Key secondary outcomes	Type and frequency of adverse event and reaction. Engraftment rate of hematopoietic stem cells. Response rate according to the RECIST criteria. Response rate according to the scintigraphic evaluation of MIBG. Overall survival (OS). Progression-free survival (PFS).

Base
Study type	Interventional

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

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	All patients receive 131I-MIBG at 666 MBq/kg. Subsequently patients receive high-dose chemotherapy and hematopoietic stem cell transplantation. Recommended regimen is MEC regimen consisting of melphalan, etoposide, and carboplatin, or BuMel regimen consisting of busulifan and melphalan. Other regimen is acceptable depending on patient's condition.
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	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1) have a definitive diagnosis of neuroblastoma. 2) have high-risk neuroblastoma. 3) have one or more 123I-MIBG-avid lesion at initial presentation or relapse. 4) have enough cryopreserved autologous peripheral blood stem cells, cord blood, or other stem cell cource. 5) meet the following criteria: Neutrophils >=500/microL Platelets >=20x10E3/microL, and transfusion independent Hb >=7.0g/dL Serum Cr <=0.8mg/dL(<5year), 1.2mg/dL(5-9year), 1.5mg/dL(10-17year) Creatinine clearance >=70mL/min/1.73m2 ALT <= 5 x upper limit of normal for age AST <= 5 x upper limit of normal for age T. Bil <= 3 x upper limit of normal for age NYHA classification class I or below SpO2 >=94% 6) ECOG PS 0 or 1, and KPS >=80% 7) be able to cooperate with the radiation safety isolation 8) written informed consent by patients or guardian
Key exclusion criteria	1) active double cancer 2) diffuse bone marrow involvement on a 123I-MIBG scan 3) Progressive disease 4) HBV (or carrier), HCV, HIV, or other active infections 5) history of fatal arrhythmia or asystole 6) concurrent poorly-controlled symptomatic arrhythmia, thyroid dysfunction, respiratory disorder, pleural effusion, or ascites. 7) concurrent coronary artery disease, usage of amiodarone, severe cardiac valvulopathy, aortic disease, or bleeding tendency. 8) woman during pregnancy or lactation, within the 28 postpartum day, desiring pregnancy within 1 year. 9) concurrent poorly-controlled psychiatric disorder 10) allergy to potassium iodide. 11) difficult to cooperate with the radiation safety isolation. 12) concurrent palliative external radiotherapy to painful lesions. 13) past treatment by the same regimen as this study. 14) be unable to receive at least of 444MBq/kg of MIBG due to exceeding of upper limit of radiation use at the center. As the upper limit of our center is 24,000MBq, the patient over 54kg is excluded. 15) Patients who, in the opinion of the attending physician, may not be able to comply with the requirements of the study.
Target sample size	8

Research contact person
Last name of lead principal investigator	1st name Seigo Middle name Last name Kinuya
Organization	Kanazawa University
Division name	Department of Nuclear Medicine, Institute of Medical, Pharmaceutical and Health Sciences
Zip code	920-8641
Address	13-1 Takara-machi, Kanazawa
TEL	076-265-2333
Email	kinuya@med.kanazawa-u.ac.jp

Public contact
1st name of contact person	1st name Raita Middle name Last name Araki
Organization	Kanazawa University
Division name	Department of Pediatrics, Kanazawa University Hospital
Zip code	920-8641
Address	13-1 Takara-machi, Kanazawa
TEL	076-265-2314
Homepage URL
Email	raraki@staff.kanazawa-u.ac.jp

Sponsor
Institute	Kanazawa University
Institute
Department

Funding Source
Organization	Kanazawa University FUJIFILM RI Pharma Co., Ltd.
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization	Innovative Clinical Research Center, Kanazawa University
Address	13-1 Takara-machi, Kanazawa
Tel	076-265-2090
Email	crc.irb-knz@esct.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	2016 Year 12 Month 12 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	8
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	Terminated
Date of protocol fixation	2017 Year 04 Month 10 Day
Date of IRB	2018 Year 07 Month 26 Day
Anticipated trial start date	2017 Year 07 Month 01 Day
Last follow-up date	2019 Year 10 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2016 Year 11 Month 29 Day
Last modified on	2019 Year 05 Month 27 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000028765