UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Circulating tumor DNA for minimal residual disease in acute myeloid leukemia after allogeneic hematopoietic stem cell transplant: a multi-center prospective study.
Title of the study (Brief title)	ctDNA for MRD with AML after transplant.KSGCT1702(ctDNA)
Region	Japan

Condition
Condition	Acute Myeloid Leukemia, Myelodysplastic Syndromes
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	YES

Objectives
Narrative objectives1	To investigate the impact of circulating tumor DNA on relapse and prognosis in patients with acute myeloid leukemia after allogeneic hematopoietic stem cell transplant
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	To evaluate 1-year cumulative incidence of relapse rate according to circulating tumor DNA status post allogeneic hematopoietic stem cell transplant
Key secondary outcomes	1. To evaluate 1-year overall survival rate according to circulating tumor DNA status post allogeneic hematopoietic stem cell transplant 2. To compare the utility of circulating tumor DNA status and conventional biomarkers 3. To compare the profiles of the somatic mutation, gene expression, and immunological cell status between diagnostic and relapsed samples from bone marrow or peripheral blood

Base
Study type	Observational

Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
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	20 years-old <=
Age-upper limit	65 years-old >=
Gender	Male and Female
Key inclusion criteria	1. Any patient (age >= 20 yo and =< 65 yo) with acute myelogenous leukemia (except for acute promyelocytic leukemia) or myelodysplastic syndromes by WHO2008 criteria 2. Any patient with any disease status or any history/sensitivity of chemotherapy at allogeneic stem cell transplant 3. Patient must have adequate tumor samples at diagnosis or relapse available for analyses (tumor proportion more than 20%) 4. Any patient who have no history of allogeneic stem cell transplant, for whom an myeloablative allogeneic stem cell transplant is warranted 5. Any patient who have signed informed consent available with regard to this study
Key exclusion criteria	1. Any patient who decline to register 2. Any patient who are considered as inappropriate to register by attending physician
Target sample size	70

Research contact person
Name of lead principal investigator	Shinichiro Okamoto
Organization	Kanto Study Group for Cell Therapy
Division name	Chairman
Address	Tokyo
TEL	042-505-4251
Email	ksgctdc@ksgct.net

Public contact
Name of contact person	Kazuaki Yokoyama
Organization	Research Hospital, The Institute of Medical Science, The University of Tokyo
Division name	Department of Hematology/Oncology
Address	4-6-1 Shirokanedai, Minato-ku, Tokyo 108-0071
TEL	03-3443-8111
Homepage URL
Email	ksgctdc@ksgct.net

Sponsor
Institute	Kanto Study Group for Cell Therapy
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)

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	2018 Year 06 Month 15 Day

Progress
Recruitment status	Open public recruiting
Date of protocol fixation	2018 Year 01 Month 18 Day
Anticipated trial start date	2018 Year 06 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information	With the recent advent of molecular techniques, such as droplet digital polymerase chain reaction (ddPCR), tumor-derived fragmented DNA in the plasma or serum, known as circulating tumor DNA (ctDNA), represents one of the most sensitive, non-invasive biomarkers available for use in cancer patients. Successful applications of such so-called liquid biopsy techniques reported to-date include the monitoring of tumor burden and minimal residual disease (MRD), evaluation of tumor clonal heterogeneity; prediction of response to treatment with molecularly targeted drugs; and detection of metastasis in many solid tumor types and hematological malignancies. Several recent studies have indicated that ctDNA quantification can be used as an alternative to conventional MRD detection modalities for hematological malignancies. Of these, Nakamura and Yokoyama et al. performed a retrospective study in 31 patients with AML and MDS undergoing allogeneic hematopoietic stem cell transplant (alloSCT) in their research hospital. They investigated the impact of residual ctDNA status on cumulative incidence of relapse (CIR) and overall survival (OS) rate. The result was as follows: 1) Diagnostic ctDNA and matched tumor had excellent correlations with regard to variant allele frequencies. 2) Residual ctDNA persistence either at 1month or 3 months post alloSCT was associated with higher 3-year cumulative incidence of relapse and inferior OS rates.

Management information
Registered date	2018 Year 06 Month 15 Day
Last modified on	2018 Year 06 Month 15 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000037630