TriangleNCT03190915 · STUDY IN CONTEXT
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. 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
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.
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.
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).
Will be measured by mass spectrometry. Will be analyzed descriptively. Values will be summarized with means and standard deviations.
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.
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.
Reported safety
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
- nct id
- NCT03190915
- trial label
- Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
- applicability
- narrower
- applicability note
- Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded.
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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