Phase II study of allogeneic hematopoietic stem cell transplantation using a preparative regimens consisting of iv Bu + Flu + L-PAM for patients with juvenile myelomonocytic leukemia (JMML)

Phase II study of allogeneic hematopoietic stem cell transplantation using a preparative regimens consisting of iv Bu + Flu + L-PAM for patients with juvenile myelomonocytic leukemia (JMML) - JMML-11

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000005936

Enrollment

Registered

2011-07-07

Start date

2011-07-01

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Juvenile myelomonocytic leukemia (JMML)

Interventions

The effect of allogeneic hematopoietic stem cell transplantation assessing event-free survival 1 year after transplantation.

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Diagnosis of JMML, that was confirmed by the pathological central review conducted by the MDS Committee of the Japanese Society of Pediatric Hematology. Diagnosis between 6 months old and 14 years old. No previous treatment including unaccepted chemotherapy, radiotherapy and allogeneic hematopoietic stem cell transplantation. Adequate end organ function. ECOG performance status score should be 0-2. Total bilirubin less than 2mg/dl, creatinin below the upper normal limit defined according to the age, creatinin clearance (or cystatin) within normal range. Normal EKG. Ejection fraction of left ventricle more than 50%. Saturation of oxygen more than 95%. Legal guardians must sign an informed consent and have been told of its possible benefits and toxic side effects. An adequate donor is available.

Exclusion criteria

Exclusion criteria: Diagnosis of Noonan syndrome, disorders with germline RAS mutations and ALPS-like diseases with somatic RAS mutations. Intracranial hemorrhage. Heart failure requiring treatment. Chronic renal failure. Active uncontrolled infections. Uncontrolled diabetes mellitus. Liver cirrhosis. Psychiatric problems.

Design outcomes

Primary

Measure	Time frame
Event-free survival 1 year after stem cell transplantation	—

Secondary

Measure	Time frame
Safety of the regimen. The adverse events and the efficacy of GVHD (acute/chronic) Overall survival 3 years after stem cell transplantation Event-free survival 3 years after stem cell transplantation Non-relapse mortality within 100 days after stem cell transplantation The rate of late effects after stem cell transplantation The effect of individualized iv Bu dose modification on the engraftment rate and adverse event rate The effect of chimerism assays on engraftment rate and adverse event rate within 100 days after stem cell transplantation To elucidate the efficacy of MRD assessment using DNA mutation status of PTPN11 or RAS on relapse rate, eFS at 1 year and 3 years after stem cell transplantation. Engraftment rate 100 days after stem cell transplantation	—

Measure

Time frame

Safety of the regimen. The adverse events and the efficacy of GVHD (acute/chronic) Overall survival 3 years after stem cell transplantation Event-free survival 3 years after stem cell transplantation Non-relapse mortality within 100 days after stem cell transplantation The rate of late effects after stem cell transplantation The effect of individualized iv Bu dose modification on the engraftment rate and adverse event rate The effect of chimerism assays on engraftment rate and adverse event rate within 100 days after stem cell transplantation To elucidate the efficacy of MRD assessment using DNA mutation status of PTPN11 or RAS on relapse rate, eFS at 1 year and 3 years after stem cell transplantation. Engraftment rate 100 days after stem cell transplantation

—

Countries

Japan

Contacts

Public ContactAkiko Saito

NPO-OSCR Data Center

officejp@nnh.hosp.go.jp052-951-1111

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026