Trial · NCT03190915

Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

NCT03190915, Phase 2, Active not recruiting, National Cancer Institute (NCI); results first posted 2024-07-16

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia. Trials, record NCT03190915. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03190915

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
NCT number: NCT03190915
Status: Active not recruiting
Phases: Phase 2
Enrollment: 10 participants, actual
Lead sponsor: National Cancer Institute (NCI)
Conditions: Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Interventions: Bone Marrow Aspiration and Biopsy, Trametinib
First posted: 2017-06-19
Last updated: 2026-11-03
Results first posted: 2024-07-16

Source ClinicalTrials.gov

Registered outcomes7

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Objective Response	12 cycles (1 cycle = 28 days)	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.
Secondary	Incidence of Adverse Events	Up to cycle 12 (1 cycle = 28 days)	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.
Secondary	Pharmacokinetic (PK) Parameters of Trametinib	Up to cycle 12 (1 cycle = 28 days)	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).
Secondary	Trametinib Concentrations	Up to cycle 12 (1 cycle = 28 days)	Will be measured by mass spectrometry. Will be analyzed descriptively. Values will be summarized with means and standard deviations.
Secondary	Mutant Allele Burden	Up to cycle 12 (1 cycle = 28 days)	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.
Secondary	Complete Response	12 cycles (1 cycle = 28 days)	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.
Secondary	Duration of Response	Up to 5 years	Duration of response (Aim 1.2.5) will be defined as the time from first occurrence of PR or CR until the first occurrence of PD, death, or going off study. Patients who progress will be considered to have had an event, patients who die prior to progressing will be considered to have a competing event, and patients who go off study prior to progressing will be censored at time of last contact. The analysis will be done using the method of Gray.

Source ClinicalTrials.gov

Outcomes7

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Objective Response	12 cycles (1 cycle = 28 days)	All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.	Percentage of patients	NUMBER
SECONDARY	Incidence of Adverse Events	Up to cycle 12 (1 cycle = 28 days)	All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.	Percentage of patients	NUMBER
SECONDARY	Pharmacokinetic (PK) Parameters of Trametinib	Up to cycle 12 (1 cycle = 28 days)	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	Trametinib Concentrations	Up to cycle 12 (1 cycle = 28 days)	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	Mutant Allele Burden	Up to cycle 12 (1 cycle = 28 days)	All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.	Percent Change In Mutant Allele Burden	MEAN
SECONDARY	Complete Response	12 cycles (1 cycle = 28 days)	All patients who met the evaluable for response criteria outlined in section 9.4 of the ADVL1521 protocol.	Percentage of patients	NUMBER
SECONDARY	Duration of Response	Up to 5 years	All patients who achieved an overall best response of Partial Response or Complete Response.	years	MEDIAN

Source ClinicalTrials.gov

Results by group5

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Objective Response	Treatment (Trametinib)	50.0	95% Confidence Interval 18.7 to 81.3	Percentage of patients
Incidence of Adverse Events	Treatment (Trametinib)	30.0	95% Confidence Interval 6.7 to 65.2	Percentage of patients
Mutant Allele Burden	Treatment (Trametinib)	0.0	Standard Deviation 0	Percent Change In Mutant Allele Burden
Complete Response	Treatment (Trametinib)	0.0	95% Confidence Interval 0.0 to 30.8	Percentage of patients
Duration of Response	Treatment (Trametinib)	2.26	Full Range 0.87 to 2.78	years

Source ClinicalTrials.gov

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Treatment (Trametinib)	10
Overall Study	COMPLETED	Treatment (Trametinib)	3
Overall Study	NOT COMPLETED	Treatment (Trametinib)	7

Source ClinicalTrials.gov

Adverse events22

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Anemia	Blood and lymphatic system disorders	Treatment (Trametinib)	1	10
other	Diarrhea	Gastrointestinal disorders	Treatment (Trametinib)	2	10
other	Dry skin	Skin and subcutaneous tissue disorders	Treatment (Trametinib)	1	10
other	Fever	General disorders	Treatment (Trametinib)	2	10
other	Folliculitis	Infections and infestations	Treatment (Trametinib)	1	10
other	Hypertension	Vascular disorders	Treatment (Trametinib)	1	10
other	Hypotension	Vascular disorders	Treatment (Trametinib)	1	10
other	Neutrophil count decreased	Investigations	Treatment (Trametinib)	2	10
other	Paronychia	Infections and infestations	Treatment (Trametinib)	2	10
other	Platelet count decreased	Investigations	Treatment (Trametinib)	3	10

1 to 10 of 22Source ClinicalTrials.gov

See all 22 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Treatment (Trametinib)	2	10
Total, other adverse events	Treatment (Trametinib)	8	10
Total, serious adverse events	Treatment (Trametinib)	4	10

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
33728588	DERIVED	PubMed
38867349	DERIVED	PubMed

Grants2

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

Project	Title	Principal investigators	Organization	Funder	Fiscal years	Source
U10CA180886	COG NCTN Network Group Operations Center	Hawkins, Douglas S.	COG RESEARCH FOUNDATION, LLC	NATIONAL CANCER INSTITUTE	2014 to 2026	NIH RePORTER
U54CA196519	Developmental and Hyperactive Ras Tumor (DHART) SPORE	Clapp, David W; SHANNON, KEVIN M.	INDIANA UNIVERSITY INDIANAPOLIS	NATIONAL CANCER INSTITUTE	2015 to 2026	NIH RePORTER

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03190915

GET https://conductscience.org/api/trials/v1/NCT03190915/events?page=2

API reference For AI assistants