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New hope for kids with tough leukemia: targeted drug shows promise

NCT ID NCT03190915

Disease control Sponsor: National Cancer Institute (NCI) Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 21, 2026 · Updated 4 times

Summary

This study tests a drug called trametinib in children with a rare blood cancer (juvenile myelomonocytic leukemia) that has returned or not responded to treatment. The drug works by blocking certain enzymes that help cancer cells grow. The goal is to see if it can shrink or control the cancer, but it is not expected to be a cure.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

10 people

The number who actually took part.

Started

Sep 2018

Expected to finish

Oct 2026

An estimate. End dates often move.

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 month to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 the presence of a clonal abnormality; frontline therapy is defined as one cycle of intravenous chemotherapy that includes any of the following agents: fludarabine, cytarabine, or any anthracycline but specifically excludes oral 6-mercaptopurine; frontline therapy will also include any conditioning regimen as part of a stem cell transplant; patients who transform to AML at any point with more than 20% blasts are not eligible for this trial * Patients must have a Lansky or Karnofsky performance status score of \>= 50, corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0, 1 or 2; use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score * Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study enrollment * Myelosuppressive chemotherapy: patients must have completely recovered from all acute toxic effects of chemotherapy, immunotherapy or radiotherapy prior to study enrollment; at least 14 days must have elapsed since the completion of cytotoxic therapy, with the exception of hydroxyurea * Note: cytoreduction with hydroxyurea can be initiated and continued for up to 24 hours prior to the start of protocol therapy * Hematopoietic growth factors: at least 14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or 7 days for short-acting growth factor; for agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur * Biologic (anti-neoplastic agent): at least 7 days must have elapsed since completion of therapy with a biologic agent; for agents that have known adverse events occurring beyond 7 days after administration, this period prior to enrollment must be extended beyond the time during which adverse events are known to occur * Monoclonal antibodies: * At least 30 days after the completion of any type of immunotherapy, e.g. tumor vaccines * At least 3 half-lives must have elapsed since prior therapy that included a monoclonal antibody * Radiotherapy: * \>= 2 weeks must have elapsed since local palliative external radiation therapy (XRT) (small port) * \>= 6 months must have elapsed if prior craniospinal XRT was received, if \>= 50% of the pelvis was irradiated, or if traumatic brain injury (TBI) was received * \>= 4 weeks must have elapsed if other substantial bone marrow irradiation was given * Stem cell transplant or rescue without TBI: no evidence of active graft versus (vs.) host disease and \>= 3 months must have elapsed since transplant; \>= 4 weeks must have elapsed since any donor lymphocyte infusion * Patients must not be known to be refractory to red blood cell or platelet transfusions * Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 (within 7 days prior to enrollment) or a serum creatinine based on age/gender as follows (within 7 days prior to enrollment): * Age: Maximum serum creatinine (mg/dL) * 1 month to \< 6 months: 0.4 (male) 0.4 (female) * 6 months to \< 1 year: 0.5 (male) 0.5 (female) * 1 to \< 2 years: 0.6 (male) 0.6 (female) * 2 to \< 6 years: 0.8 (male) 0.8 (female) * 6 to \< 10 years: 1 (male) 1 (female) * 10 to \< 13 years: 1.2 (male) 1.2 (female) * 13 to \< 16 years: 1.5 (male) 1.4 (female) * \>= 16 years: 1.7 (male) 1.4 (female) * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment) * Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 3 x ULN (=\< 135 U/L) (within 7 days prior to enrollment) (for the purpose of this study, the ULN for SGPT is 45 U/L) * Serum albumin \>= 2 g/dL (within 7 days prior to enrollment) * Shortening fraction of \>= 27% by echocardiogram OR ejection fraction of \>= 50% by multi-gated acquisition (MUGA) * Corrected QT (by Bazett's formula \[QTcB\]) interval \< 450 msecs * Patients must be able to swallow tablets or liquid; use of a nasogastric or gastrostomy (G) tube is also allowed Exclusion Criteria: * Patients who are pregnant or breast-feeding are not eligible for this study as there is yet no available information regarding human fetal or teratogenic toxicities; negative pregnancy tests must be obtained in girls who are post-menarchal; patients of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for the duration of study therapy; women of childbearing potential should be advised to use effective contraception for 4 months after the last dose of trametinib; trametinib may also potentially be secreted in milk and therefore breastfeeding women are excluded; female patients should not breastfeed during treatment with trametinib, and for 4 months following the last dose; male patients must use a condom during intercourse and agree not to father a child during therapy and for 4 months following discontinuation of trametinib to avoid unnecessary exposure of trametinib to the fetus * Concomitant Medications * Corticosteroids: patients requiring corticosteroids who have not been on a stable or decreasing dose of corticosteroid for the 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, \>= 14 days must have elapsed since last dose of corticosteroid * Note: hydrocortisone used as a pre-medication to prevent transfusion related reactions is not considered a concomitant corticosteroid * Investigational drugs: patients who are currently receiving another investigational drug are not eligible * Anti-cancer agents: patients who are currently receiving other anti-cancer agents are not eligible (except patients receiving hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy) * Anti-graft versus host disease (GVHD) or agents to prevent organ rejection post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial * Cardiac medications: any medications for treatment of left ventricular systolic dysfunction * Patients who have an uncontrolled infection are not eligible * Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible * Patients with a history of hepatic sinusoid obstructive syndrome (veno-occlusive disease) within the prior 3 months are not eligible * Patients with a history of or current evidence/risk of retinal vein occlusion (RVO) or central serous retinopathy (CSR) are not eligible * Patients with a history of RVO or CSR, or predisposing factors to RVO or CSR (e.g., uncontrolled glaucoma or ocular hypertension * Patients with uncontrolled systemic disease(s) such as hypertension or diabetes mellitus are not eligible; blood pressure must be =\< the 95th percentile for age, height, and gender * Patients with a history of allergic reaction attributed to compounds of similar chemical or biologic composition to the MEK inhibitor, trametinib are not eligible * Patients with a clinical diagnosis of Noonan syndrome are not eligible; Note: patients with Casitas B-lineage lymphoma (CBL) syndrome, also known as Noonan-like syndrome, are eligible to enroll

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Conditions

The condition(s) this trial relates to.

juvenile myelomonocytic leukemia neurofibromatosis type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Juvenile myelomonocytic leukemia Neurofibromatosis type 1

Contacts and locations

Locations

  • Alfred I duPont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202-3591, United States

  • Arnold Palmer Hospital for Children

    Orlando, Florida, 32806, United States

  • BI-LO Charities Children's Cancer Center

    Greenville, South Carolina, 29605, United States

  • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

    Houston, Texas, 77030, United States

  • C S Mott Children's Hospital

    Ann Arbor, Michigan, 48109, United States

  • Carolinas Medical Center/Levine Cancer Institute

    Charlotte, North Carolina, 28203, United States

  • Children's Healthcare of Atlanta - Arthur M Blank Hospital

    Atlanta, Georgia, 30329, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Alabama

    Birmingham, Alabama, 35233, United States

  • Children's Hospital of Orange County

    Orange, California, 92868, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224, United States

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Children's Hospitals and Clinics of Minnesota - Minneapolis

    Minneapolis, Minnesota, 55404, United States

  • Children's Mercy Hospitals and Clinics

    Kansas City, Missouri, 64108, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Connecticut Children's Medical Center

    Hartford, Connecticut, 06106, United States

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Dayton Children's Hospital

    Dayton, Ohio, 45404, United States

  • Dell Children's Medical Center of Central Texas

    Austin, Texas, 78723, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Kaiser Permanente Downey Medical Center

    Downey, California, 90242, United States

  • Kaiser Permanente-Oakland

    Oakland, California, 94611, United States

  • Loma Linda University Medical Center

    Loma Linda, California, 92354, United States

  • Lucile Packard Children's Hospital Stanford University

    Palo Alto, California, 94304, United States

  • Lurie Children's Hospital-Chicago

    Chicago, Illinois, 60611, United States

  • MedStar Georgetown University Hospital

    Washington D.C., District of Columbia, 20007, United States

  • Methodist Children's Hospital of South Texas

    San Antonio, Texas, 78229, United States

  • Mount Sinai Hospital

    New York, New York, 10029, United States

  • NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

    New York, New York, 10032, United States

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Nemours Children's Clinic-Jacksonville

    Jacksonville, Florida, 32207, United States

  • Nemours Children's Hospital

    Orlando, Florida, 32827, United States

  • Nicklaus Children's Hospital

    Miami, Florida, 33155, United States

  • OSF Children's Hospital of Illinois

    Peoria, Illinois, 61637, United States

  • Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • Phoenix Childrens Hospital

    Phoenix, Arizona, 85016, United States

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113, United States

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202, United States

  • Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center

    Denver, Colorado, 80218, United States

  • Saint Joseph's Hospital/Children's Hospital-Tampa

    Tampa, Florida, 33607, United States

  • Saint Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • The Children's Hospital at TriStar Centennial

    Nashville, Tennessee, 37203, United States

  • The Steven and Alexandra Cohen Children's Medical Center of New York

    New Hyde Park, New York, 11040, United States

  • UCSF Medical Center-Mission Bay

    San Francisco, California, 94158, United States

  • UT Southwestern/Simmons Cancer Center-Dallas

    Dallas, Texas, 75390, United States

  • University of Iowa/Holden Comprehensive Cancer Center

    Iowa City, Iowa, 52242, United States

  • University of Kentucky/Markey Cancer Center

    Lexington, Kentucky, 40536, United States

  • University of Miami Miller School of Medicine-Sylvester Cancer Center

    Miami, Florida, 33136, United States

  • University of Minnesota/Masonic Cancer Center

    Minneapolis, Minnesota, 55455, United States

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104, United States

  • Vanderbilt University/Ingram Cancer Center

    Nashville, Tennessee, 37232, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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