UMIN-CTR Clinical Trial

Basic information
Public title	Clinical evaluation of efficacy of dose-modified intravenous busulfan in allogeneic hematopoietic stem cell transplantation for hematological malignancy.
Acronym	Efficacy of dose-modified intravenous busulfan
Scientific Title	Clinical evaluation of efficacy of dose-modified intravenous busulfan in allogeneic hematopoietic stem cell transplantation for hematological malignancy.
Scientific Title:Acronym	Efficacy of dose-modified intravenous busulfan
Region	Japan

Condition
Condition	Hematological malignancy
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	Evaluation of safety and efficacy of dose-modified intravenous busulfan by prospectively analyzing busulfan concentration and subsequent dose adjustment in allogeneic hematopoietic stem cell transplantation for hematological malignancy.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Pragmatic
Developmental phase	Phase II

Assessment
Primary outcomes	The proportion of patients with event-free survival in 100 day after transplantation
Key secondary outcomes	The proportion 0f patients with engraftment in 100 day after transplantation The proportion of patients with grade3 or 4 adverse effects in 100 day after transplantation The proportion of patients with non-relapase mortality in 100 day and 12 month after transplantation The proportion of patients with relapase in 100 day and 12 month after transplantation

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	dose-modified intravenous busulfan
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	16 years-old <=
Age-upper limit	70 years-old >
Gender	Male and Female
Key inclusion criteria	1. Hematological malignancy for AML, MDS, CML and ALL 2. Performance status(PS, ECOG) of 0-2 3. Patients without major organ dysfunction 4. T.Bil less than 2.0 mg/dL 5. AST and ALT less than 3 upper limit of the normal range 6. SCr less than 2.0mg/dl 7. Heart ejection fraction more than 50% 8. SpO2 more than 93% 9. Estimated life expectancy of at least 3 months 10. Patients requird to have an appropriate donor identified before enrollment. (1) HLA-matched related or unrelated donor (2) 1 HLA-mismatched related or unrelated donor (3) Cord blood with nucleated cell counts more than 2 107/kg and mismatched HLA less than 2 11. Ability to provide informed consent.
Key exclusion criteria	1. positive for anti-HIV antibody 2. positive for Hbs antigen 3. active another malignancy 4. uncontrollable psychological disorders 5. uncontrollable active infection 6. positive for donor-specific HLA antibody 7. previous history of hematopoietic stem cell transplantation 8. known hypersensitivity to busulfan 9. pregnant women 10. Patients judged as inappropriate for this study by physicians
Target sample size	40

Research contact person
Name of lead principal investigator	1st name Akifumi Middle name Last name Takaori-Kondo
Organization	Kyoto University Hospital
Division name	Department of Hematology and Oncology
Zip code	606-8507
Address	54 Shogoin Kawaharacho, Sakyo-ku, Kyoto-shi, Kyoto
TEL	075-751-3150
Email	atakaori@kuhp.kyoto-u.ac.jp

Public contact
Name of contact person	1st name Tadakazu Middle name Last name Kondo
Organization	Kyoto University Hospital
Division name	Department of Hematology and Oncology
Zip code	606-8507
Address	54 Shogoin Kawaharacho, Sakyo-ku, Kyoto-shi, Kyoto
TEL	075-366-7550
Homepage URL	http://www.kuhp.kyoto-u.ac.jp/~hemonc/research/trial.html
Email	tadakazu@kuhp.kyoto-u.ac.jp

Sponsor
Institute	Kyoto University Hospital, Department of Hematology and Oncology
Institute
Department

Funding Source
Organization	none
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization	Ethics Committee, Graduate School and Faculty of Medicine, Kyoto University
Address	54 Shogoin Kawaharacho, Sakyo-ku, Kyoto-shi, Kyoto
Tel	075-753-9470
Email	ethcom@kuhp.kyoto-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	京都大学医学部附属病院

Other administrative information
Date of disclosure of the study information	2014 Year 06 Month 15 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	2014 Year 05 Month 09 Day
Date of IRB	2014 Year 06 Month 26 Day
Anticipated trial start date	2014 Year 07 Month 01 Day
Last follow-up date	2019 Year 03 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	2014 Year 05 Month 27 Day
Last modified on	2022 Year 06 Month 02 Day

Link to view the page
URL(English)	https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000016385