Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03190915|
Recruitment Status : Recruiting
First Posted : June 19, 2017
Last Update Posted : May 28, 2021
|Condition or disease||Intervention/treatment||Phase|
|Juvenile Myelomonocytic Leukemia Neurofibromatosis Type 1||Drug: Trametinib||Phase 2|
I. To determine the objective response rate to trametinib in children with recurrent or refractory juvenile myelomonocytic leukemia (JMML).
I. To further define and describe the toxicities of single agent trametinib in children with recurrent or refractory JMML.
II. To further characterize the pharmacokinetics of trametinib in children with recurrent or refractory JMML.
III. To prospectively evaluate mutant allele burden as a marker of disease activity in JMML.
IV. To measure the rate of complete responses in children with recurrent or refractory JMML.
V. To measure the duration of response among responders.
I. To describe the distribution of JMML diagnostic criteria in children with recurrent or refractory JMML.
Patients receive trametinib orally (PO) once daily (QD) on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up annually for up to 5 years.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2 Study of the MEK Inhibitor Trametinib (NSC# 763093) in Children With Relapsed or Refractory Juvenile Myelomonocytic Leukemia|
|Actual Study Start Date :||October 6, 2017|
|Estimated Primary Completion Date :||March 31, 2024|
|Estimated Study Completion Date :||March 31, 2024|
Experimental: Treatment (trametinib)
Patients receive trametinib PO QD on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.
- Objective response [ Time Frame: 4 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.
- Incidence of adverse events [ Time Frame: Up to cycle 12 (1 cycle = 28 days) ]Will be assessed by National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Toxicity tables will be constructed to summarize the observed incidence by type of toxicity and grade.
- Pharmacokinetic (PK) parameters of trametinib [ Time Frame: 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, 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).
- Mutant allele burden [ Time Frame: Up to cycle 12 (1 cycle = 28 days) ]Will be measured by mass spectrometry. Will be analyzed descriptively. Values will be summarized with means, standard deviations, and 95% confidence intervals.
- Trametinib concentrations [ Time Frame: Up to cycle 12 (1 cycle = 28 days) ]Will be measured by mass spectrometry. Will be analyzed descriptively. Values will be summarized with means, standard deviations, and 95% confidence intervals.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03190915
|Principal Investigator:||Elliot Stieglitz||Children's Oncology Group|