NIPH Clinical Trials Search

Phase II study of allogeneic hematopoietic cell transplantation for children with acute myeloid leukemia in first and second complete remission using fludarabine, cytarabine, melphalan and low-dose total body irradiation as a conditioning regimen (AML-SCT15).

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	acute myeloid leukemia (AML)
Date of first enrollment	2017/07/01
Target sample size	66
Countries of recruitment	Japan,South America
Study type	Interventional
Intervention(s)	FLAMEL regimen: fludarabine (30 mg/m^2 x 1/day for 4 days), cytarabine (2 g/m^2 x 1/day for 4 days), melphalan (60 mg/m^2 x 1/day for 3 days) and low-dose total body irradiation (2 Gy x 1/day for one day) Graft: any of bone marrow, peripheral stem cell and cord blood. Graft-versus-host disease (GVHD) prophylaxis: in essentially tacrolimus or cyclosporine in combination with methotrexate. For evaluation of secondary endo-points, patients received allogeneic transplantation using myeloablative conditioning regimens consisting of busulfan (> 8 mg/kg) or total body irradiation (>= 8 Gy, fractionated) are also enrolled to this study. Graft: any of bone marrow, peripheral stem cell and cord blood. Graft-versus-host disease (GVHD) prophylaxis: in essentially tacrolimus or cyclosporine in combination with methotrexate.

Outcome(s)

Primary Outcome

Probability of overall survival rate at three years after transplantation

Secondary Outcome

1) Efficacy Probability of event-free survival (EFS) rate, Cumulative incidence of relapse and non-relapse mortality (NRM) at 3 years after transplantation Probability of overall survival (OS) rate, EFS rate, Cumulative incidence of relapse and NRM at 10 years after transplantation Profile of cause of death at 3 and 10 years after transplantation Incidence of second transplantation and second malignant neoplasms 2) Short-term safety (up to 100 days after transplantation) Cumulative incidence of engraftment, secondary graft failure (2nd GF) and NRM at 100 days after transplantation Cumulative incidence and severity of acute and chronic graft-versus-host disease (GVHD), sinusoidal obstruction syndrome (SOS) and thrombotic microangiopathy (TMA) Incidence of and reason for donor lymphocyte infusion (DLI) Monitoring of chimerism at 1 and 3 months after FLAMEL regimen Profile of severe infectious disease after KIR ligand-mismatched cord blood transplantation Profile of conditioning regimen-related toxicities (Bearman criteria) up to 4 weeks after transplantation Profile of CTCAE (grade >=3) Cumulative days of fever (>=38C) 3) Long-term safety Cumulative incidence of engraftment, 2nd GF, SOS and TMA up to 1 year after transplantation Incidence of and reason for DLI up to one year after transplantation Cumulative incidence and severity of acute and chronic GVHD Monitoring of height (SD) and body weight (SD) after transplantation Monitoring of height (SD) of patients untreated with growth hormone Monitoring of TSH, free-T4, LH, FSH, testosterone, estradiol and anti-mullerian hormone Monitoring of Tanner stage and menstruation Incidence of growth disturbance, thyroid dysfunction and gonadal dysfunction at 3 and 10 years after transplantation Profile of CTCAE (grade >=3) up to 1 year after transplantation

Key inclusion & exclusion criteria

Age minimum	Not applicable
Age maximum	20years-old
Gender	Male and Female
Include criteria
Exclude criteria	Exclusion criteria 1) Patients who have extramedullary lesions at registry 2) Patients who have prior radiation therapy 3) Patients who have prior hematopoietic cell transplantation 4) Patients who have poorly controlled infectious disease 5) Patients who have psychiatric disorder 6) Patients who have coinciding malignancies 7) Patients who have an episode of hypersensitivity reactions against drugs used in conditioning regimens and/or GVHD prophylaxis 8) Pregnant women or women who may be pregnant 9) Patients who are considered as inappropriate for this trial by attending physicians