UMIN-CTR Clinical Trial

Basic information
Public title	A phase II study of allogeneic hematopoietic stem cell transplantation for patients with myeloid malignancies using intravenous busulfan and cyclophosphamide as conditioning regimen.
Acronym	KSGCT1002/C-SHOT1003
Scientific Title	A phase II study of allogeneic hematopoietic stem cell transplantation for patients with myeloid malignancies using intravenous busulfan and cyclophosphamide as conditioning regimen.
Scientific Title:Acronym	KSGCT1002/C-SHOT1003
Region	Japan

Condition
Condition	CML(chronic myelogenous leukemia) AML(acute myeloid leukemia) MDS(myelodysplastic syndrome)
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To investigate safety and efficacy of allogeneic hematopoietic stem cell transplantation using intravenous busulfan and cyclophosphamide as conditioning regimen for patients with CML, AML and MDS.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2	Pragmatic
Developmental phase	Phase II

Assessment
Primary outcomes	Probability of event-free survival at 1 year after transplantation
Key secondary outcomes	(1) Incidence of Bearman's grade 2 or higher adverse events within 28 days after transplantation (2) Cumulative incidence of engraftment at 100 days after transplantation (3) Cumulative incidence of non-relapse mortality at 100 days after transplantation (4) Cumulative incidence of SOS (sinusoidal obstruction syndrome) at 100 days after transplantation (5) Cumulative incidence of Grade II-IV acute GVHD at 1 year after transplantation (6) Cumulative incidence of chronic GVHD at 1 year after transplantation (7) Probability of overall survival and cumulative incidence of relapse at 1 year after transplantation

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	Conditioning regimen Intravenous busulfan 3.2mg/kg/day x 4days + cyclophosphamide 60mg/kg/day x 2days
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	55 years-old >
Gender	Male and Female
Key inclusion criteria	(1) Patients between the age of 16 and 55 at informed consent (2) Patients diagnosed as CML, AML, or MDS who are eligible for allogeneic hematopoietic stem cell transplantation. No limitation of disease status at transplantation. Patients who need chemotherapy for disease control except imatinib, dasatinib and nilotinib for CML are excluded. (3) Patients who have the fixed schedule about the harvest of bone marrow or peripheral blood stem cells from related or unrelated donors who meet the following conditions: 1) HLA-A, B, DRB1 are genetically identical 2)HLA-A and B are genetically identical and 1 locus of HLA-DRB1 is genetically mismatched (4) Patients whose performance status are 0 or 1 by ECOG criteria (5) Patients who meet all of the following conditions: 1) SPO2>=94% by room air 2) sCr=<2.0mg/dL 3) T-bil=<1.5mg/dL 4) AST, ALT and r-GTP=<3 x upper limit of normal 5) Absence of abnormal findings of ECG which require therapeutic interventions 6) Ejection fraction of left ventricle>=50% by UCG (6) Patients who give written informed consent following sufficient explanation. Patients who are under 20 years old are required to give informed consent from both persons with parental authority and patients themselves
Key exclusion criteria	(1) Patients with uncontrolled diabetes mellitus in spite of regular use of insulin (2) Patients with uncontrolled hypertension in spite of use of antihypertensive drugs (3) Patients who have active infection (4) Patients who are positive for TPHA, HBV surface antigen, or anti-HCV antibody. (5) Patients who are positive for anti-HTLV-I or anti-HIV antibody (6) Patients who are not evaluated to be able to survive more than 100 days after transplantation (7) Patients who have coinciding active malignancies (8) Patients who are pregnant or in the lactation period (9) Patients who have psychiatric disorder (10) Patients who have prior hematopoietic stem cell transplantation (11) Patients who are considered as inappropriate to register by attending physicians
Target sample size	55

Research contact person
Name of lead principal investigator	1st name Middle name Last name Shinichiro Okamoto, Miyamura Kouichi
Organization	Kanto Study Group for Cell Therapy(KSGCT) Nagoya Blood and Marrow Transplantation (NBMT) Group
Division name	Chairman
Zip code
Address	Tokyo, Aichi
TEL	03-6225-2040
Email	ksgctdc@ksgct.net

Public contact
Name of contact person	1st name Middle name Last name Takuya Yamashita, Sawa Masashi
Organization	KSGCT, NBMTG
Division name	Study Coordinators
Zip code
Address	Tokyo
TEL	03-6225-2040
Homepage URL
Email	tayamash@ncc.go.jp

Sponsor
Institute	Kanto Study Group for Cell Therapy Nagoya Blood and Marrow Transplantation (NBMT) Group
Institute
Department

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

Other related organizations
Co-sponsor	Center for Supporting Hematology-Oncology Trial(C-SHOT)
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	2010 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	2010 Year 05 Month 19 Day
Date of IRB
Anticipated trial start date	2010 Year 06 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2010 Year 05 Month 21 Day
Last modified on	2015 Year 08 Month 29 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000004409

Research Plan
Registered date	File name

Research case data specifications
Registered date	File name

Research case data
Registered date	File name

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