W-JHS MDS01

JRCT ID: jRCTs071180016

Registered date:13/02/2019

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: myelodysplastic syndrome
• Date of first enrollment: 31/08/2016
• Target sample size: 100
• Study type: Interventional

Intervention(s)

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.

Outcome(s)

• Primary Outcome: 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.

Secondary Outcome

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.

Key inclusion & exclusion criteria

• Age minimum: >= 16age old
• Age maximum: Not applicable
• Gender: Both

Include 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).

Exclude 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.

Related Information

• Primary Sponsor: Mitani Kinuko
• Source(s) of Monetary Support: Kyowa Kirin co., Ltd
• Secondary ID(s): UMIN000022185