UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Phase I study of tamibarotene for relapse or refractory pediatric solid tumor
Title of the study (Brief title)	TBT-PI
Region	Japan

Condition
Condition	Relapse or refractory pediatric solid tumor
Classification by specialty	Pediatrics
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To determine a recommend dose of tamibarotene and to assess its pharmacokinetics in patients with relapse or refractory pediatric solid tumor.
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Explanatory
Developmental phase	Phase I

Assessment
Primary outcomes	Dose limiting toxicity (DLT)
Key secondary outcomes	1)Adverse events 2)Clinical Benefit Rate(CBR) 3)Pharmacokinetics and dose-response for tamibarotene

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	Tamibarotene is orally administered twice a day in 1-14or1-21 days. 28days per a course,up to 8 courses.
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	3 years-old <=
Age-upper limit	30 years-old >=
Gender	Male and Female
Key inclusion criteria	1)Aged 3 to 30 years old 2)Histologically proven sarcoma, blastoma, germ cell tumors, CNS except malignant lymphoma 3)Relapse or progression following disease-specific standard chemotherapies 4)Site by With evaluable disease 5)PS(Karnofsky or Lansky) >= 50% 6)7 days or more have passed since last use of anti-tumor agents which are continuously-administered and 14 days or more have passed since last use of anti-tumor agents which are not administered continuously 7)No prior irradiation within 12 weeks if radiation fields contain either whole brain and spine, whole abdomen, whole lung, whole body, or more than 50% of pelvis. No prior irradiation within 6 weeks if radiation fields contain either less than 50% of pelvis, or 5 or less vertebras. No prior irradiation within 14 days if radiation fields are limited. 8)No difficulty to take soft capsule or tablet 9)Normal organ function confirmed by laboratory tests within 14 days 10)Written informed consent from patient and/or legal guardian
Key exclusion criteria	1)Synchronous or asynchronous(within 5 years)other cancer except carcinoma in situ or intramucosal carcinoma 2)Active infection requires systemic therapy 3)Any abnormalities in electrocardiogram tested within 28 days, which require intervention 4)Respiratory disorder requiring oxygen supply 5)Woman during pregnancy, or impossible to discontinue breast-feeding from within 1 month prior to enrollment to 2 years after the final dose of study treatment. Woman without intent to use birth control from within 1 month prior to enrollment to 2 years after the final dose of study treatment. Man without intent to use birth control from within 1 month prior to enrollment to 2 years after the final dose of study treatment. 6)Severe psychiatric disorder 7)Patients with history of allergy to tamibalotene 8)Patients with history of treatment with tamibarotene 9)In patients having a brain tumor or metastasis to brain, a symptom with the intracranial hypertension is poor in control 10)Patients otherwise considered ineligible for enrollment in the study by the investigator
Target sample size	21

Research contact person
Name of lead principal investigator	Akihiro Sato
Organization	National Cancer Center Hospital East
Division name	Office of Clinical Research Support
Address	6-5-1, Kashiwanoha, Kashiwa, Chiba
TEL	04-7133-1111
Email	DACTBT@ml.res.ncc.go.jp

Public contact
Name of contact person	Akihiro Sato
Organization	National Cancer Center Hospital East
Division name	Office of Clinical Research Support
Address	6-5-1, Kashiwanoha, Kashiwa, Chiba
TEL	04-7133-1111
Homepage URL
Email	DACTBT@ml.res.ncc.go.jp

Sponsor
Institute	National Cancer Center Hospital East Division of Pediatric Oncology
Institute
Department

Funding Source
Organization	AMED
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization	Japan

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

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	国立がん研究センター中央病院（東京都) 大阪市立総合医療センター（大阪府） 九州大学病院(福岡県） 国立がん研究センター東病院(千葉県)

Other administrative information
Date of disclosure of the study information	2015 Year 04 Month 09 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 03 Month 20 Day
Anticipated trial start date	2015 Year 04 Month 09 Day
Last follow-up date	2017 Year 04 Month 08 Day
Date of closure to data entry	2017 Year 04 Month 21 Day
Date trial data considered complete	2017 Year 10 Month 01 Day
Date analysis concluded	2017 Year 12 Month 01 Day

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2015 Year 04 Month 06 Day
Last modified on	2017 Year 10 Month 13 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019708