UMIN-CTR Clinical Trial

Public title

Efficacy and safety of a 5-day regimen of azacitidine in patients with low-risk myelodysplastic syndrome

Acronym

Efficacy and safety of a 5-day regimen of azacitidine in patients with low-risk myelodysplastic syndrome

Scientific Title

Efficacy and safety of a 5-day regimen of azacitidine in patients with low-risk myelodysplastic syndrome

Scientific Title:Acronym

Efficacy and safety of a 5-day regimen of azacitidine in patients with low-risk myelodysplastic syndrome

Region

Japan

Condition

low-risk myelodysplastic syndrome

Classification by specialty

Hematology and clinical oncology

Classification by malignancy

Malignancy

Genomic information

NO

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

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

Study type

Interventional

Basic design

Single arm

Randomization

Non-randomized

Randomization unit

Blinding

Open -no one is blinded

Control

Uncontrolled

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

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

Name of lead principal investigator

1st name
Middle name
Last name	Mitsuhiro Matsuda, MD, PhD

Organization

PL General Hospital

Division name

Department of Hematology

Zip code

Address

2204 Shindo, Tondabayashi, Osaka 584-8585

TEL

0721-23-7805

Email

Name of contact person

1st name
Middle name
Last name	Mitsuhiro Matsuda, MD, PhD

Organization

PL General Hospital

Division name

Department of Hematology

Zip code

Address

2204 Shindo, Tondabayashi, Osaka 584-8585

TEL

0721-23-7805

Homepage URL

Email

matsuda@plhospital.or.jp

Institute

Department of Hematology, Kinki University School of Medicine

Institute

Department

Personal name

Organization

none

Organization

Division

Category of Funding Organization

Self funding

Nationality of Funding Organization

Co-sponsor

Name of secondary funder(s)

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

Date of disclosure of the study information

2011

Year

05

Month

30

Day

URL releasing protocol

Publication of results

Published

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

Recruitment status

Completed

Date of protocol fixation

2011

Year

05

Month

01

Day

Date of IRB

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

Other related information

Registered date

2011

Year

05

Month

27

Day

Last modified on

2018

Year

07

Month

27

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000006693