UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Efficacy and safety of a 5-day regimen of azacitidine in patients with low-risk myelodysplastic syndrome
Title of the study (Brief title)	Efficacy and safety of a 5-day regimen of azacitidine in patients with low-risk myelodysplastic syndrome
Region	Japan

Condition
Condition	low-risk myelodysplastic syndrome
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	The aime of this study is to analyze the efficacy and safety of 5-day regimen of azacitidine in patients with low-risk MDS (RA, RARS) and to evaluate the changes of various clinical markers.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2	Pragmatic
Developmental phase	Phase II

Assessment
Primary outcomes	hematological improvement
Key secondary outcomes	1. hematological remission 2. transfusion dependency 3. hematological improvement in each chromosomal karyotype 4. progression stage (WT1) 5. continuity of therapy 6. adverse events

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	The dosage is 75mg/m2 of azacitidine by subcutaneous injection or intravenous infusion for 10 minutes once daily for 7 days every four weeks.
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	Not applicable
Gender	Male and Female
Key inclusion criteria	1.Patients with low risk MDS (RA, RARS) according to FAB classification and is considered by investigator to be adequate for this study, meeting at least one of three following criteria. i) a packed red blood cell transfusion history in 3 months (12 weeks) prior to the enrollment ii) platelet count less than 50,000/mm3 or clinically meaningful bleeding symptoms iii) neutrophil count less than 1,000/mm3 and increased susceptibility to infection (requiring use of antibiotics) 2. Patients with a life expectancy of at least 3 months at the time of enrollment 3. Patients with no prior administration of azacitidine-based treatment (Chemotherapy, HSCT, Lenalidomide, Imuunosuppressive therapy, Anabolic steroids therapy, Iron-chelation therapy, Vitamin D and Vitamin K are allowed) 4. Age: 20 years and over 5. Patients who is not planned for allo-HSCT. 6. Patients with ECOG performance status of 0-2 7. Patients with clinically adequate hepatic, renal and heart function defined as follows i) Serum total bilirubin less than 2.0 mg/dl ii) Serum creatinine less than 2.0 mg/dl iii) PaO2 more than 60 Torr or SaO2 more than 93% while breathing room air. iv) no severe ECG abnormalities. 8. Patients who can be hospitalized during at least 1st cycle. 9. Patients who has given a written informed consent for this study (including contraception).
Key exclusion criteria	1. Patients with simultaneous multiple cancers. 2. Patients with history of hyper- sensitivity to mannitol 3. Patients with poorly-controlled infection requiring intensive care with antibiotics and antifungals. 4. Patients with poorly-controlled diabetes. 5. Patients with severe psychiatric disorder. 6. Pregnant or lactating females. 7. Patients with positive serology for HBs antigen, HCV antibody or HIV antibody. 8. Patients who is deemed as ineligible for this study by investigator.
Target sample size	50

Research contact person
Name of lead principal investigator	Mitsuhiro Matsuda, MD, PhD
Organization	PL General Hospital
Division name	Department of Hematology
Address	2204 Shindo, Tondabayashi, Osaka 584-8585
TEL	0721-23-7805
Email

Public contact
Name of contact person	Mitsuhiro Matsuda, MD, PhD
Organization	PL General Hospital
Division name	Department of Hematology
Address	2204 Shindo, Tondabayashi, Osaka 584-8585
TEL	0721-23-7805
Homepage URL
Email	matsuda@plhospital.or.jp

Sponsor
Institute	Department of Hematology, Kinki University School of Medicine
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	2011 Year 05 Month 30 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2011 Year 05 Month 01 Day
Anticipated trial start date	2011 Year 05 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete	2016 Year 05 Month 31 Day
Date analysis concluded	2018 Year 04 Month 07 Day

Related information
URL releasing protocol
Publication of results	Published
URL releasing results
Results
Other related information

Management information
Registered date	2011 Year 05 Month 27 Day
Last modified on	2018 Year 07 Month 27 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000006693