UMIN-CTR Clinical Trial

Basic information
Public title	A prospective non-randomized phase II study to assess the efficacy of unrelated cord blood transplantation for adult unfavorable hematological malignancies (C-SHOT 0601)
Acronym	A phase II study to assess the efficacy of unrelated cord blood transplantation for adult hematological malignancies (C-SHOT 0601)
Scientific Title	A prospective non-randomized phase II study to assess the efficacy of unrelated cord blood transplantation for adult unfavorable hematological malignancies (C-SHOT 0601)
Scientific Title:Acronym	A phase II study to assess the efficacy of unrelated cord blood transplantation for adult hematological malignancies (C-SHOT 0601)
Region	Japan

Condition
Condition	Patients with acute myeloid leukemia/acute lymphocytic leukemia/myelodysplastic syndrome/chronic myelomonocytic leukemia categorized as high risk group
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	YES

Objectives
Narrative objectives1	For patients with relapsed disease or high-risk of relapse, allogeneic hematopoietic transplantation would be indicated. In this study, patients without suitable related or unrelated donor will be transplanted with unrelated cord blood after conditioning including cyclophosphamide, cytosine arabinoside with/without G-CSF and total body irradiation. The purpose of this study is to assess the safety and efficacy of the UCBT.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2	Explanatory
Developmental phase	Phase II

Assessment
Primary outcomes	survival rate of patients with successful engraftment at 180 days post-transplant
Key secondary outcomes	Time to hematopoietic recovery, grade of treatment-associated toxicity by day28, incidence and severity of acute GVHD, incidence and severity of chronic GVHD, treatment-related mortality rate at day180, relapse rate at day180, disease-free survival 2years after transplantation, overall survival 2years 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	G-CSF combined cytosine arabinoside 2g/square meter x4 +cyclophosphamide 60mg/kg x2 +TBI12Gy will be used as conditioning regimen for AML/MDS/CMMoL patients. For ALL patients, cyclophosphamide 60mg/kg x2 +TBI12Gy will be used as conditioning regimen. Subsequently, unrelated cord blood unit will be transplanted under immunosuppressive treatment with short-term methotrexate and tacrolimus.
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	(A) AML 1.Pts in CR1 (a)pts with unfavorable or unknown karyotype (b)FAB-M0/M6/M7 (c)pts with intermediate karyotype and non-CR after first-line induction (d)FAB-M3/M3v failed salvage treatment using As2O3, or ATRA, or other synthetic retinoids. 2.CR2 (except pts with t(15;17) in molecular CR) (B)B-ALL 1.High-risk ALL pts in CR1 where high-risk is defined as; (a)pts with evidence of t(9;22) (b)elevated WBC at presentation: >30000/mm3 and >30 years old (c)pts with evidence of 11q23 or MLL gene rearrangement (d)failed to achieve CR after 4 weeks of induction 2. CR2 or later CR (C)T-ALL 1.High-risk T-ALL pts in CR1 where high-risk is defined as; (a)>30 years old (b)elevated WBC at presentation >50000/mm3 2.CR2 (D)MDS Pts categorized as intermediate-2 or high-risk by IPSS score system (E)CMMoL Pts categorized as intermediate-2 or high-risk by CMML scoring system. Pts with MDS and CMMoL in CR1 or chemo-naive are eligible. Pts with MDS and CMMoL treated only with Azacitidine are also eligible. (1)Age between 16 and 55 years (2)(a)Performance states (ECOG)=0 or 1 (b)Hematopoietic stem cell transplantation-specific comorbidity index (HCT-CI)=0 at registration Pts with transient liver dysfunction are eligible. (3)UCB units are available as follows; (a)HLA-A/B/DR serologically match more than 4/6 (b)TNCC of UCB unit is 2.0 x 10E7/kg recipient weight as of frozen (4)First high dose therapy with hematopoietic cell support including auto transplantation (5)Written informed consent to participate the trial
Key exclusion criteria	(1)Positive for HIV antibody and/or HBs antigen and/or HCV antibody (2)Graft manipulation such as T-cell depletion (3)history of administration of gemtuzumab ozogamicin within 6 month before transplantation (4)Pregnant or during breast feeding (5)Uncontrolled another neoplastic disease (6)Uncontrolled active psychiatric disease (7)Uncontrolled active infection (8)Allergic history to drugs used in the conditioning regimen or GVHD prophylaxis regimen (9)Patients suggested as ineligible by their attending physician
Target sample size	60

Research contact person
Name of lead principal investigator	1st name Koichi Middle name Last name Miyamura
Organization	Japanese Red Cross Nagoya First Hospital
Division name	Department of Hematology
Zip code	453-8511
Address	3-35 Michishita-cho, Nakamura-ku, Nagoya, Aichi, Japan
TEL	052-481-5111
Email	miyamu@nagoya-1st.jrc.or.jp

Public contact
Name of contact person	1st name Tetsuya Middle name Last name Nishida
Organization	Nagoya University Graduate School of Medicine
Division name	Department of Hematology and Oncology
Zip code	466-8560
Address	65 Tsurumai-cho,Showa-ku,Nogoya,Aichi,Japan
TEL	052-744-2145
Homepage URL	http://www.c-shot.or.jp/study/0601/outline/
Email	tnishida@med.nagoya-u.ac.jp

Sponsor
Institute	Nagoya Blood and Marrow Transplantation Group
Institute
Department

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

Other related organizations
Co-sponsor	None
Name of secondary funder(s)	None

IRB Contact (For public release)
Organization	Nagoya University Certified Review Board
Address	65 Tsurumai-cho, Showa-ku, Nagoya, Aichi
Tel	052-744-2947
Email	ethics@med.nagoya-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

Other administrative information
Date of disclosure of the study information	2007 Year 02 Month 07 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	2006 Year 11 Month 26 Day
Date of IRB	2007 Year 01 Month 29 Day
Anticipated trial start date	2007 Year 02 Month 01 Day
Last follow-up date	2012 Year 02 Month 01 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2007 Year 02 Month 07 Day
Last modified on	2021 Year 02 Month 17 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000000710