Phase II clinical trials aiming to establish standard therapy by chemotherapy for transient myelodysplastic syndrome (TAM)

JRCT ID: jRCTs041190063

Registered date:28/08/2019

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Transient abnormal myelopoiesis
• Date of first enrollment: 30/09/2019
• Target sample size: 250
• Study type: Interventional

Intervention(s)

Those who meet the following criteria will be administered a cytarabine therapy.
1) White blood cell count is 100,000 /micro L or more
2) Self-urine at the start of treatment (1 ml/kg/h) or more is obtained

One course of cytarabine therapy is performed.
Cytarabine 1 hour i.v. 1mg/kg/dose for 7 days.

Outcome(s)

• Primary Outcome: 1 year overall survival of patients who received low dose cytarabine therapy.

Secondary Outcome

1 year overall survival rate of total cohort.
2 year/4 year overall survival rate of total cohort and low dose cytarabine treatment group
Positive rates of minimal residual disease (MRD) at 1 month /3 months /6 months /12 months /18 months /24 months in overall and low dose cytarabine treatment group
2 years /4 years cumulative incidence of leukemia in total cohort and low dose cytarabine treatment group
Cumulative incidence of leukemia in MRD (GATA1-MRD/FCM-MRD) positive patients and negative patients in total cohort and low dose cytarabine treatment group
Adverse events of low-dose cytarabine treatment group
Early death (within 9 months of birth) and cumulative incidence of leukemia among GATA1 gene mutation types in total cohort and low dose cytarabine treatment group

Key inclusion & exclusion criteria

• Age minimum: >=
• Age maximum: <= 2month old
• Gender: Both

Include criteria

Those that meet all the following criteria shall be eligible.
(1) Blast cells appeared in peripheral blood less than 30 days of age, and diagnosed as TAM.
(2) Registration is within 30 days from the diagnosis date.
(3) Written consent was obtained from the substitute.

• Exclude criteria: Not particularly provided.

Related Information

• Primary Sponsor: Muramatsu Hideki
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development