Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia (NCT03190915) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
United States10 participantsStarted 2018-09-09
Plain-language summary
This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment (refractory). Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
Who can participate
Age range1 Month – 21 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Patients must be \>= 1 month and \< 22 years of age at the time of study entry
* Patients must have had histologic verification of juvenile myelomonocytic leukemia (JMML) at original diagnosis and currently have relapsed or refractory disease; the diagnosis is made based on the following criteria
* JMML category 1 (all of the following): the diagnostic criteria must include all features in category 1 and EITHER (i) one of the features in category 2 OR (ii) two features from category 3 to make the diagnosis
* Splenomegaly
* \> 1000 (1 x 10\^9/uL) circulating monocytes
* \< 20% blasts in the bone marrow or peripheral blood
* Absence of the t(9;22) or BCR/ABL fusion gene
* JMML category 2 (at least one of the following if at least two category 3 criteria are not present):
* Somatic mutation in RAS or PTPN11
* Clinical diagnosis of NF1 or NF1 gene mutation
* Homozygous mutation in CBL
* Monosomy 7
* JMML category 3 (at least two of the following if no category 2 criteria are met):
* Circulating myeloid precursors
* White blood cell count, \> 10 000 (10 x 10\^9/ uL)
* Increased hemoglobin F for age
* Clonal cytogenetic abnormality
* GM-CSF hypersensitivity
* Patients with refractory or relapsed JMML must have had at least one cycle of intensive frontline therapy or at least 2 cycles of a deoxyribonucleic acid (DNA) demethylating agent with persistence of disease, defined by clinical symptoms or th…