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Comparing a MEK inhibitor to chemotherapy for pediatric low-grade glioma

A Randomized and Controlled Phase II Protocol in Non NF1 Pediatric and AYA (Adolescent and Young Adults) Patients Bearing a Newly Diagnosed Low Grade Glioma With Wild Type BRAF Gene Comparing a Daily Oral MEK Inhibitor (Trametinib) Versus Weekly Vinblastine for 18 Months

Phase 2 Interventional University Hospital, Strasbourg, France · NCT05180825

This study is testing whether a new drug called trametinib works better than standard chemotherapy for children and young adults with low-grade brain tumors that have specific genetic features.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment134 (estimated)
Ages1 Month to 25 Years
SexAll
SponsorUniversity Hospital, Strasbourg, France Academic / other
Drugs / interventionsTrametinib
Locations25 sites (Amiens and 24 other locations)
Trial IDNCT05180825 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effectiveness of trametinib, a MEK inhibitor, compared to standard chemotherapy (vinblastine) in treating pediatric low-grade gliomas, specifically targeting patients without congenital NF1 mutations and with wild-type BRAF genes. The study aims to improve tumor response, overall survival, and progression-free survival in this specific subgroup of patients. Participants aged 1 month to 25 years with histologically confirmed tumors will be enrolled, and their tumor characteristics will be carefully assessed to ensure eligibility. The trial is designed to address the limitations of current first-line therapies for these tumors.

Who should consider this trial

Good fit: Ideal candidates include children and young adults aged 1 month to 25 years with specific types of low-grade gliomas that meet the study's eligibility criteria.

Not a fit: Patients with congenital NF1 mutations or those with BRAF mutations may not benefit from this study.

Why it matters

Potential benefit: If successful, this trial could provide a more effective treatment option for children and young adults with low-grade gliomas, potentially improving survival rates and quality of life.

How similar studies have performed: While there have been studies on MEK inhibitors in various cancers, this specific approach for pediatric low-grade gliomas is novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Age: ≥ 1 month to ≤ 25 years
* Signed written informed consent prior to study participation of the legal representatives and the patient if the patient can understand the impact of clinical trial and to give consent. For patients above 18 years, their written informed consent will be obtained.
* Patient may be under guardianship or curatorship (for patient under legal guardianship, authorization is given by the legal representative of the patient under guardianship. For patient under curatorship, consent will be obtained from the adult assisted by his or her legal curator
* Histologically proven grade 1 glioma/mixed glio-neuronal tumors or pleomorphic xanthoastrocytoma (PXA) confirmed by local referee and the centrally pathology reviewing
* Determination of a negative BRAFv600 mutation by immunohistochemistry and/or molecular methods
* Systematic determination 7q34 duplication status or KIAA1549-BRAF fusion
* Midline tumors without proven histone H3 mutations
* Diffuse glioma without IDH1 mutation
* Collection of fresh frozen tumor tissues and/or paraffin-embedded samples for further molecular biomarker testing
* Sus-tentorial, optic pathway, midline and spine locations allowed
* Karnofsky or Lansky ≥ 50%
* Criteria for post-surgical treatment: severe visual or neurological symptoms at diagnosis, clinical deterioration of visual or neurological symptoms or radiological progression. The radiological progression is defined as an increase of solid part of the tumor of more than 25% compared to the pre-baseline MRI-imaging over a time period of at least 3 months or the occurrence of new metastatic lesions.
* Infants below one year of age with chiasmatic and/or hypothalamic tumor will be treated immediately after surgery, independently from neurological and/or visual evolution
* Females of child-bearing potential must be willing to practice highly effective contraception during all treatment and until 6 months after the last dose of study drugs' administration. Additionally, females of child-bearing potential must have a negative serum pregnancy test within 7 days prior to start of study drugs. Boys with reproductive potential must be willing to use condom and consider contraception for partner women of childbearing potential during treatment and until 4 months after the last study drugs' administration.
* Patients must have adequate bone marrow function defined as: absolute neutrophil count (ANC) ≥ 1500/µL; platelets ≥ 100,000/µL and hemoglobin ≥ 9.0 g/dl
* Patients must have adequate liver function within 7 days prior to screening: bilirubin (sum of unconjugated and conjugated) ≤ 1.5 ULN for age, ALT and AST ≤ 2.5 x upper limit of normal, alkaline phosphatase ≤ 4 x upper limit of normal, INR/PTT \< 1.5 x upper limit of normal,
* Patients must have adequate renal function within 7 days prior to screening: serum creatinine \< 1.5 x upper limit of normal for age and a creatinine clearance \> 60 ml/min for 1.73 m2
* Cardiac function defined as a corrected QT (QTcF) interval \< 480 msec, LVEF ≥ lower limit of normal (LLN) by echocardiogram (ECHO)
* Adequate blood pressure control (smaller or equal to the 95th percentile for patient's age, height and gender)
* Patients are willing and able to comply with scheduled visits, treatment plan, laboratory tests and study procedures
* Guardians (in case of patients under 18 years) or patient if above 18 years must be affiliated to or a beneficiary of health insurance system.

Non-inclusion criteria

* Patients presenting a neurofibromatosis type 1 (NF1) congenital disease
* Pure optic nerve glioma, limited to one nerve and without optic chiasma infiltration.
* Completely resected tumors
* Previous treatment except tumor surgery
* Pregnancy and lactation
* Participation in other clinical trials
* Prior non-surgical therapy for this tumor
* Diffuse intrinsic pontine glioma (DIPG), even if histologically diagnosed as WHO grade II
* Subependymal giant astrocytoma (SEGA) in patients with TSC
* Patient having a known diagnosis of human immunodeficiency virus (HIV) infection, hepatitis B or C
* Known hypersensitivity to drugs or excipients
* History of another malignancy
* History of current uncontrolled infection

Where this trial is running

Amiens and 24 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Grade 1 GliomaMixed Glio-neuronal TumorsPleomorphic Xanthoastrocytoma
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.