UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000022185
Receipt number R000025558
Scientific Title Analysis on response to darbepoetin alfa in patients with low risk myelodysplastic syndromes
Date of disclosure of the study information 2016/05/09
Last modified on 2023/05/08 09:43:05

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Basic information

Public title

Analysis on response to darbepoetin alfa in patients with low risk myelodysplastic syndromes

Acronym

Myelodysplastic syndrome 01 study in West Japan Hematology Study Group (W-JHS MDS01 study)

Scientific Title

Analysis on response to darbepoetin alfa in patients with low risk myelodysplastic syndromes

Scientific Title:Acronym

Myelodysplastic syndrome 01 study in West Japan Hematology Study Group (W-JHS MDS01 study)

Region

Japan


Condition

Condition

Myelodysplastic syndromes

Classification by specialty

Hematology and clinical oncology Laboratory medicine

Classification by malignancy

Others

Genomic information

YES


Objectives

Narrative objectives1

To determine the existence of gene mutations that predict effectiveness of darbepoietin alfa in treating anemia of low risk (low or Int-1 in IPSS risk categories) MDS, mutation analysis of blood cells is performed, mainly focusing on highly frequent mutations including SF3B1, TET2, SFRS2, ASXL1, DNMT3A, RUNX1, and U2AF1. It is expected that the identification of effect prediction factors brings knowledge about the molecular pathology of ineffective erythropoiesis of patients with myelodysplastic syndromes

Basic objectives2

Efficacy

Basic objectives -Others


Trial characteristics_1


Trial characteristics_2


Developmental phase



Assessment

Primary outcomes

Correlation between highly frequent gene mutations and hematological improvement according to IWG criteria 2006 (HI-E) to darbepoetin alfa until 16 weeks after the initiaion of treatment

Key secondary outcomes

1. Minor response to darbepoetin alfa until 16 weeks after the initiation of treatment in blood transfusion dependent patients.
2. Major response to darbepoetin alfa until 16 weeks after the initiation of treatment in blood transfusion dependent patients.
3. Hematological improvement according to IWG criteria 2003 (HI-E) by darbepoetin alfa until 16 weeks after the initiation of treatment in blood transfusion independent patients.
4. Variety and frequency of gene mutations observed in all subjects.
5. Correlation between decreased cell lineages (erythrocytes, leukocytes, platelets) and types of gene mutations.
6. Analysis on mortality and progression to AML from 16 weeks to 1 year after the initiaion of treatment.
7. Correlation between highly frequent gene mutations and interval to achievement of the first hematological improvement according to IWG criteria 2006 (HI-E) after the initiaion of treatment.


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 peripheral blood of patients is collected before administration of darbepoetin alfa, and DNA is extracted. The presence of gene mutations is then analyzed, mainly on highly frequent gene mutations (e.g. SF3B1, TET2, SFRS2, ASXL1, DNMT3A, RUNX1, U2AF1) on the panel of 104 genes in a previous report (Ogawa S, et al. Leukemia 2014) using next-generation sequencing method.
Darbepoetin alfa at a dose of 240 microgram per body is administered every 7 days for 16 times in total.
Effect of darbepoetin alfa is observed up to 16 weeks and it is statistically analyzed whether the presence of specific gene mutation affects the effectiveness or not.

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


Not applicable

Gender

Male and Female

Key inclusion criteria

1) Patients with definite diagnosis of MDS in diagnostic criteria of refractory anemia (myelodysplastic syndrome) (MHLW, Research and study group on idiopathic hematopoietic disorder, revised in 2010 fiscal year).
2) Patients having anemia associated with MDS, and is aged 16 years or older.
3) Patients categorized in Low or Int-1 risk in IPSS risk categories.
4) Patients who can visit participating institutions in prescribed schedule.
5) Patients providing the written informed consent (in the case of minor subject, taking from both the subject and legal representative).

Key exclusion criteria

1) Patients at risk of thromboembolism with present or past medical history of myocardial infarction, pulmonary infarction and cerebral infarction or similar disorders.
2) Patients with uncontrollable hypertension.
3) Patients with medical history of drug hypersensitivity to darbepoetin alfa or other erythropoietin formulation.
4) Patients with severe (need for hospital care, or judgement by investigators) or uncontrollable complication.
5) Patients inappropriate for study participation due to complication of mental disease or psychiatric symptom.
6) Patients with cognitive disorder.
7) Patients judged by investigators to be inappropriate for study participation.

Target sample size

100


Research contact person

Name of lead principal investigator

1st name Kinuko
Middle name
Last name Mitani

Organization

Dokkyo Medical University School of Medicine

Division name

Department of Hematology and Oncology

Zip code

321-0293

Address

880 Kita-Kobayashi, Mibu-machi, Shimotsuga-gun, Tochigi 321-0293, JAPAN

TEL

0282-86-1111

Email

kinukom-tky@umin.ac.jp


Public contact

Name of contact person

1st name Motoshi
Middle name
Last name Ichikawa

Organization

Dokkyo Medical University School of Medicine

Division name

Department of Hematology and Oncology

Zip code

321-0293

Address

880 Kita-Kobayashi, Mibu-machi, Shimotsuga-gun, Tochigi 321-0293, JAPAN

TEL

0282-86-1111

Homepage URL


Email

motoshi-tky@umin.ac.jp


Sponsor or person

Institute

Cooperative study between the West Japan Hematology Study Group and Clinical Research Support Center Kyushu

Institute

Department

Personal name



Funding Source

Organization

Kyowa Kirin co., Ltd

Organization

Division

Category of Funding Organization

Profit organization

Nationality of Funding Organization



Other related organizations

Co-sponsor


Name of secondary funder(s)



IRB Contact (For public release)

Organization

Clinical Research Network Fukuoka Certified Review Board

Address

3-1-1, Maidashi, Hig ashi-ku, Fukuoka, Fukuoka

Tel

0 9 2 -6 4 3 -7 1 7 1

Email

m a il@ c rnfukuo ka .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

2016 Year 05 Month 09 Day


Related information

URL releasing protocol


Publication of results

Partially published


Result

URL related to results and publications


Number of participants that the trial has enrolled

85

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

2016 Year 04 Month 12 Day

Date of IRB

2016 Year 04 Month 12 Day

Anticipated trial start date

2016 Year 05 Month 09 Day

Last follow-up date

2020 Year 08 Month 31 Day

Date of closure to data entry


Date trial data considered complete


Date analysis concluded



Other

Other related information



Management information

Registered date

2016 Year 05 Month 02 Day

Last modified on

2023 Year 05 Month 08 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000025558


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name