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 range
1 Month – 21 Years
Sex
ALL
See this in plain English?
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this trial is no longer recruiting new patients, can you find out whether there are any other open trials testing trametinib or similar MEK inhibitors for JMML that we could consider instead?
2This is a Phase 2 trial, which means researchers are still building evidence on how well trametinib works — what do you know so far about the response rates seen in children with JMML or NF1-related JMML on this study?
3The trial is specifically measuring 'objective response,' meaning how much the leukemia shrinks or goes away — how does that standard compare to what we'd hope to see from a bone marrow transplant or other treatments my child might be eligible for right now?
4Since my child may have NF1-related JMML, which involves a different genetic pathway than other types, does that change how likely they are to respond to a MEK inhibitor like trametinib, and is that something this trial was designed to look at separately?
5Before considering a trial like this, should we first try any standard treatments, or given that this is described as targeting relapsed or refractory JMML, does that mean standard options would need to have already been exhausted?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.