Multi-center non-randomized open trial to evaluate the efficacy of azacitidine followed by allo-HSCT for TP53(+) MDS.

JRCT ID: jRCT1051200003

Registered date:20/04/2020

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: myelodysplastic syndromes
• Date of first enrollment: 15/11/2017
• Target sample size: 160
• Study type: Interventional
• Intervention(s): azacitidine treatment followed by hematopoietic stem cell transplantation

Outcome(s)

• Primary Outcome: 2-year OS after allogeneic hematopoietic stem cell transplantation.

Secondary Outcome

Response rate and VAF (variant allele frequency) changes after azacitidine treatment among TP53-mutated patients.
Ratio of the patients who were able to receive HSCT among TP53-mutated patients.
2, 3, 5-year OS afterenrollements among TP53-mutated patients.

Key inclusion & exclusion criteria

• Age minimum: >= 16age old
• Age maximum: < 70age old
• Gender: Both

Include criteria

(1) MDS or MDS/MPN(CMML) according to WHO classification (4th) or MDS (RAEB-t) according to FAB classification.
(2) Age between 16 and 69.
(3) No history of azacitidine treatment OR on azacitidine treatment and fulfills all of the following conditions;
*On azacitidine treatment
*Total interruption period of azacitidine <3 months.
*Efficacy of azacitidine be SD or above.
*Specimen of bone marrow and/or peripheral blood is available for genetic study.
(4) Indication for allogeneic HSCT, and the patient has the will.
(5) ECOG Performance status (PS) 0-2
(6) Sufficient organ function to receive therapy (fulfill all of the below conditions)
*T.Bil<2.0mg/dL
*Cre<2.0mg/dL
*GOT/GPT< 3x ULN
*No serious heart or lung diseases.
(7) Provide written informed consent to join the study on his/her free-will.

Exclude criteria

(1) Previous history of allo-HSCT.
(2) Obviously refractory to azacitidine, or
planning allo-HSCT for disease progression after azacitidine treatment.
(3) Active comorbid malignancy (less than 5 years after cure) except for myeloid malignancies that are assumed to be associated with MDS development.
(4) Have uncontrollable infections.
(5) Have psychiatric disorders requiring major tranquilizers.
(6) During pregnancy or suspected pregnancy.
(7) Positive for either HBs-Ag, HCV-Ab, HIV-Ab.
(8) Assumed inappropriate to register the study by the attending doctors.

Related Information

• Primary Sponsor: Nannya Yasuhito
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development
• Secondary ID(s): UMIN000027789