UMIN-CTR Clinical Trial

Basic information
Public title	The prospective, multicenter Stop Tyrosine Kinase Inhibitor trial of pediatric chronic myeloid leukemia with sustained complete molecular response (STKI-14)
Acronym	The prospective, multicenter Stop TKI trial of pediatric CML (STKI-14)
Scientific Title	The prospective, multicenter Stop Tyrosine Kinase Inhibitor trial of pediatric chronic myeloid leukemia with sustained complete molecular response (STKI-14)
Scientific Title:Acronym	The prospective, multicenter Stop TKI trial of pediatric CML (STKI-14)
Region	Japan

Condition
Condition	Chronic Myeloid Leukemia (CML)
Classification by specialty	Hematology and clinical oncology Pediatrics
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	We study the efficacy of tyrosine kinase inhibitor (TKI) discontinuation for the patients less than age of 20 at diagnosis of CML in chronic or accelerated phase who has been maintaining complete molecular remission for two years or more under the treatment of TKI.
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Treatment-free remission rate at 12 months
Key secondary outcomes	*Cumulative incidence of MMR loss at 12 months after stopping TKI *Cumulative incidence of CMR loss at 12 months after stopping TKI *Exploration of the TFR-related factors *Cumulative incidence of MMR after restarting TKI *Cumulative incidence of CMR after restarting TKI *Growth rate at TKI discontinuation, during MMR maintenance, and after restarting TKI *QOL at TKI discontinuation, during MMR maintenance, and after restarting TKI *Adverse events at TKI discontinuation, during MMR maintenance, and after restarting TKI

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	After sustained CMR for 2 years or more under the treatment of tyrosine kinase inhibitor (TKI) for 3 years or more, TKI treatment is discontinued. In case of MMR loss, the same TKI as before discontinuation is restarted at the same dose.
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	1 years-old <=
Age-upper limit	20 years-old >
Gender	Male and Female
Key inclusion criteria	1) Patients with BCR-ABL fusion transcript-positive CML 2) Chronic or accelerated phase at diagnosis 3) Age under 20 years old at diagnosis 4) Regsitered to the JPLSG CHM-14 study 5) The total period of treatment with TKI for 3 years or more 6) CMR maintained for 2 years or more under the treatment of TKI 7) Less than 0.01% of BCR-ABL/ABL mRNA (international scale) within 4 weeks before the registration 9) Sufficient hepatic and renal function satisfying the laboratory data listed below ; a)T-Bili: within 3x of age adjusted upper-limit of normal range. b)Creatinine: within 3x of age adjusted upper-limit of normal range. 10) Written informed consent obtained from patient or guardians
Key exclusion criteria	1) History of blast crisis 2) CNS hemorrage more than grade 3 of CACAE v4.0 3) Uncontrolled infection, including active tuberculosis and positive of HIV antibody. 4) Pregnancy or high possibility of pregnancy and giving suck woman. 5) History of congenital or acquired immunodeficiency. 6) QTfc, corrected by Friderics formula as QTfc = QT/RR*1/3, is more than 0.45 seconds. 7) Any inappropriate status judged by physician.
Target sample size	25

Research contact person
Last name of lead principal investigator	1st name Middle name Last name Hiroyuki Shimada
Organization	Keio University School of Medicine
Division name	Department of Pediatrics
Zip code
Address	35 Shinanomachi, Shinjuku-ku, Tokyo, JAPAN
TEL	03-5363-3816
Email	hshimada@a5.keio.jp

Public contact
1st name of contact person	1st name Middle name Last name Hiroyuki Shimada
Organization	Keio University School of Medicine
Division name	Department of Pediatrics
Zip code
Address	35 Shinanomachi, Shinjuku-ku, Tokyo, JAPAN
TEL	03-5363-3816
Homepage URL
Email	hshimada@a5.keio.jp

Sponsor
Institute	Japanese Pediatric Leukemia/Lymphoma Study Group (JPLSG)
Institute
Department

Funding Source
Organization	Ministry of Health, Labour and Welfare
Organization
Division
Category of Funding Organization	Japanese Governmental office
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

Other administrative information
Date of disclosure of the study information	2015 Year 06 Month 01 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	Completed
Date of protocol fixation	2015 Year 04 Month 27 Day
Date of IRB	2015 Year 05 Month 21 Day
Anticipated trial start date	2015 Year 06 Month 01 Day
Last follow-up date	2018 Year 12 Month 26 Day
Date of closure to data entry	2019 Year 02 Month 28 Day
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2015 Year 05 Month 21 Day
Last modified on	2019 Year 04 Month 11 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000020394