UMIN-CTR Clinical Trial

Basic information
Public title	Multi-center non-randomized open trial to evaluate the efficacy of azacitidine followed by allo-HSCT for TP53(+) MDS.
Acronym	REPTAT trial
Scientific Title	Multi-center non-randomized open trial to evaluate the efficacy of azacitidine followed by allo-HSCT for TP53(+) MDS.
Scientific Title:Acronym	REPTAT trial
Region	Japan

Condition
Condition	myelodysplastic syndromes
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	YES

Objectives
Narrative objectives1	To evaluated the combined treatment of azacitidine followed by allogeneic HSCT for intractable TP53-positive myelodysplastic syndromes.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	2-year OS after allogeneic hematopoietic stem cell transplantation.
Key secondary outcomes

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 Maneuver
Interventions/Control_1	azacitidine treatment followed by allogeneic HSCT
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) MDS or MDS/MPN(CMML) according to WHO classification (4th) or MDS (RAEB-t) according to FAB classification. (2) Age between 16 and 69. (3) No history of azacitidine treatment OR on azacitidine treatment and fulfills all of the following conditions; *On azacitidine treatment *Total interruption period of azacitidine <3 months. *Efficacy of azacitidine be SD or above. *Specimen of bone marrow and/or peripheral blood is available for genetic study. (4) Indication for allogeneic HSCT, and the patient has the will. (5) ECOG Performance status (PS) 0-2 (6) Sufficient organ function to receive therapy (fulfill all of the below conditions) *T.Bil<2.0mg/dL *Cre<2.0mg/dL *GOT/GPT< 3x ULN *No serious heart or lung diseases. (7) Provide written informed consent to join the study on his/her free-will.
Key exclusion criteria	(1) Previous history of allo-HSCT. (2) Obviously refractory to azacitidine, or planning allo-HSCT for disease progression after azacitidine treatment. (3) Active comorbid malignancy (less than 5 years after cure) except for myeloid malignancies that are assumed to be associated with MDS development. (4) Have uncontrollable infections. (5) Have psychiatric disorders requiring major tranquilizers. (6) During pregnancy or suspected pregnancy. (7) Positive for either HBs-Ag, HCV-Ab, HIV-Ab. (8) Assumed inappropriate to register the study by the attending doctors.
Target sample size	24

Research contact person
Name of lead principal investigator	1st name Yasuhito Middle name Last name Nannya
Organization	Kyoto University
Division name	Dep. of Pathol. & Tumor Biol.
Zip code	6068501
Address	Sakyo-ku
TEL	+81757539285
Email	ynanya-tky@umin.ac.jp

Public contact
Name of contact person	1st name Yasuito Middle name Last name Nannya
Organization	Kyoto University
Division name	Dep. of Pathol. & Tumor Biol.
Zip code	6068501
Address	Yoshida Konoe-cho, Sakyo-ku, Kyoto City
TEL	075-753-9285
Homepage URL
Email	ynanya-tky@umin.ac.jp

Sponsor
Institute	Japan Agency for Medical Research and Development
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization	Kyoto University Graduate School and Faculty of Medicine, Kyoto University Hospital Ethics Committee
Address	Yoshida-Konoe-cho, Sakyo-ku
Tel	075-753-4680
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

Other administrative information
Date of disclosure of the study information	2017 Year 06 Month 20 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	2017 Year 09 Month 15 Day
Date of IRB	2017 Year 09 Month 14 Day
Anticipated trial start date	2017 Year 10 Month 18 Day
Last follow-up date	2025 Year 09 Month 30 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2017 Year 06 Month 16 Day
Last modified on	2022 Year 06 Month 20 Day

Link to view the page
URL(English)	https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000031272