Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

Draft scope assessment: unclear. Attached because the study's own record describes refractory disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full. Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

Response rates will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed accounting for the two-stage design. A responder is defined as a patient who achieves a best response of PR or CR on the study prior to having an overall response of PD; all others will be considered non-responders. The definitions of response are based on a publication (PMID: 25552679) entitled "Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia". Patients can be categorized as having experienced complete remission, partial remission, stable disease, or progressive disease based on a combination of clinical and molecular variables.

All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.

→

secondary · POSTED

Will be assessed by National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Will report the percentage of patients within each disease stratum who experienced a grade 3 or higher toxicity with attribution of possible, probable, or definite while on protocol therapy or within 30 days of the last dose of therapy.

All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.

→

secondary · POSTED

A descriptive analysis of pharmacokinetic parameters of trametinib will be performed to define systemic exposure, drug clearance, Trametinib concentrations, and other pharmacokinetic parameters. The PK parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit).

Pharmacokinetic samples were collected and run, and the assay results were reviewed. However, two experts who reviewed the data found it to be uninterpretable because it was generated using incorrect standard curves.

→

secondary · POSTED

Will be measured by mass spectrometry. Will be analyzed descriptively. Values will be summarized with means and standard deviations.

→

secondary · POSTED

Will be measured by next-generation sequencing. The percent change in mutant allele burden will be analyzed descriptively. Values will be summarized with means and standard deviations.

All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.

→

secondary · POSTED

Complete Response rates will be calculated as the percent of evaluable patients who had an overall best response of Complete Response, and confidence intervals will be constructed accounting for the two-stage design.

All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.

→

Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

No linked quality-of-life measure is recorded for this setting and study.

Full draft applicability and source-link record

trial

release id: clinical-indications-draft-1
disease state id: ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id: NCT03190915
trial label: Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
applicability: unclear
applicability note: Attached because the study's own record describes refractory disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z
provenance class: registry-condition-search
discovery note: Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.
outcome Count: 6
harms Count: 1
qol Count: 0

outcomes

release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0000
source section id
NCT03190915-om-0000
source outcome index
0
endpoint
Objective Response
endpoint definition
Response rates will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed accounting for the two-stage design. A responder is defined as a patient who achieves a best response of PR or CR on the study prior to having an overall response of PD; all others will be considered non-responders. The definitions of response are based on a publication (PMID: 25552679) entitled "Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia". Patients can be categorized as having experienced complete remission, partial remission, stable disease, or progressive disease based on a combination of clinical and molecular variables.
time origin
12 cycles (1 cycle = 28 days)
analysis role
primary
reporting status
POSTED
data cutoff
Source null
population note
All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0001
source section id
NCT03190915-om-0001
source outcome index
1
endpoint
Incidence of Adverse Events
endpoint definition
Will be assessed by National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Will report the percentage of patients within each disease stratum who experienced a grade 3 or higher toxicity with attribution of possible, probable, or definite while on protocol therapy or within 30 days of the last dose of therapy.
time origin
Up to cycle 12 (1 cycle = 28 days)
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0002
source section id
NCT03190915-om-0002
source outcome index
2
endpoint
Pharmacokinetic (PK) Parameters of Trametinib
endpoint definition
A descriptive analysis of pharmacokinetic parameters of trametinib will be performed to define systemic exposure, drug clearance, Trametinib concentrations, and other pharmacokinetic parameters. The PK parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit).
time origin
Up to cycle 12 (1 cycle = 28 days)
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Pharmacokinetic samples were collected and run, and the assay results were reviewed. However, two experts who reviewed the data found it to be uninterpretable because it was generated using incorrect standard curves.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0003
source section id
NCT03190915-om-0003
source outcome index
3
endpoint
Trametinib Concentrations
endpoint definition
Will be measured by mass spectrometry. Will be analyzed descriptively. Values will be summarized with means and standard deviations.
time origin
Up to cycle 12 (1 cycle = 28 days)
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Pharmacokinetic samples were collected and run, and the assay results were reviewed. However, two experts who reviewed the data found it to be uninterpretable because it was generated using incorrect standard curves.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0004
source section id
NCT03190915-om-0004
source outcome index
4
endpoint
Mutant Allele Burden
endpoint definition
Will be measured by next-generation sequencing. The percent change in mutant allele burden will be analyzed descriptively. Values will be summarized with means and standard deviations.
time origin
Up to cycle 12 (1 cycle = 28 days)
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0005
source section id
NCT03190915-om-0005
source outcome index
5
endpoint
Complete Response
endpoint definition
Complete Response rates will be calculated as the percent of evaluable patients who had an overall best response of Complete Response, and confidence intervals will be constructed accounting for the two-stage design.
time origin
12 cycles (1 cycle = 28 days)
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

arms

release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0000
arm key
OG000
arm label
Treatment (Trametinib)
regimen
Patients receive trametinib PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo a bone marrow aspiration or biopsy at baseline, on day 28 of cycles 1 and 2, at all subsequent odd numbered cycles, and at end of treatment.
analyzed count
10
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03190915-om-0000#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0001
arm key
OG000
arm label
Treatment (Trametinib)
regimen
Patients receive trametinib PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo a bone marrow aspiration or biopsy at baseline, on day 28 of cycles 1 and 2, at all subsequent odd numbered cycles, and at end of treatment.
analyzed count
10
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03190915-om-0001#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0002
arm key
OG000
arm label
Treatment (Trametinib)
regimen
Patients receive trametinib PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo a bone marrow aspiration or biopsy at baseline, on day 28 of cycles 1 and 2, at all subsequent odd numbered cycles, and at end of treatment.
analyzed count
0
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03190915-om-0002#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0003
arm key
OG000
arm label
Treatment (Trametinib)
regimen
Patients receive trametinib PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo a bone marrow aspiration or biopsy at baseline, on day 28 of cycles 1 and 2, at all subsequent odd numbered cycles, and at end of treatment.
analyzed count
0
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03190915-om-0003#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0004
arm key
OG000
arm label
Treatment (Trametinib)
regimen
Patients receive trametinib PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo a bone marrow aspiration or biopsy at baseline, on day 28 of cycles 1 and 2, at all subsequent odd numbered cycles, and at end of treatment.
analyzed count
10
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03190915-om-0004#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
outcome key
om-0005
arm key
OG000
arm label
Treatment (Trametinib)
regimen
Patients receive trametinib PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo a bone marrow aspiration or biopsy at baseline, on day 28 of cycles 1 and 2, at all subsequent odd numbered cycles, and at end of treatment.
analyzed count
10
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03190915-om-0005#groups/OG000

harms

release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia-refractory-33e0633a
nct id
NCT03190915
harm key
EG000
source section id
NCT03190915-ae
event group key
EG000
event group label
Treatment (Trametinib)
serious affected
4
serious at risk
10
other affected
8
other at risk
10
frequency threshold
0
collection period
Adverse Events monitored/assessed up to 30 days after treatment (12 cycles). All-Cause Mortality monitored/assessed up to 5 years from study enrollment.
event definition
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

quality of life

setting source links

Preserved source evidence · Not medical advice