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Mitogen-activated Protein Kinase (MAPK) Inhibitor

Trametinib for Pediatric Brain Tumor

Phase 2

Waitlist Available

Led By Sébastien Perreault, MD

Research Sponsored by St. Justine's Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients must be aged ≥ 1 month (corrected age) to ≤ 25 years when starting trametinib

Participants must belong to one of the specified groups: NF1 with progressing/refractory LGG, NF1 with PN, progressing/refractory LGG with KIAA 1549-BRAF fusion, progressing/refractory glioma with activation of the MAPK/ERK pathway who do not meet criteria for other study groups

Timeline

Screening 3 weeks

Treatment Varies

Follow Up within 14 days prior to treatment start for investigations of tumor tissue. at screening, week 13, week 25, week 37, week 49, week 61, at the end of treatment day 504 and every 6 months up to 3 years for ctdna evaluation.

Awards & highlights

Study Summary

This trial will study the response rate of pediatric brain tumors to oral administration of the drug trametinib. A total of 150 patients will be recruited, and the study will also explore the molecular mechanisms behind tumor development, progression and resistance to treatment.

Who is the study for?

This trial is for children and young adults aged 1 month to 25 years with specific brain tumors or neurofibromatosis who have not responded to at least one prior treatment. Eligible participants must be able to take oral medication, agree to use contraception if applicable, and commit to study requirements like MRI scans.Check my eligibility

What is being tested?

The trial tests Trametinib's effectiveness in treating pediatric patients with glioma or plexiform neurofibroma that involves the MAPK/ERK pathway. It's an open-label phase 2 study where patients receive daily doses of Trametinib over cycles lasting 28 days each.See study design

What are the potential side effects?

Trametinib may cause side effects such as skin rash, diarrhea, fatigue, nausea and vomiting, abdominal pain, hypertension, bleeding events, heart problems (like reduced ejection fraction), vision changes, muscle aches or weakness.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 1 month and 25 years old and starting trametinib treatment.

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I have a specific type of progressing brain tumor or nerve tumor.

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I can do most activities but may need help.

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My organ and bone marrow functions are normal.

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I can swallow and keep down pills without significant stomach issues affecting absorption.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ within 14 days prior to treatment start for investigations of tumor tissue. at screening, week 13, week 25, week 37, week 49, week 61, at the end of treatment day 504 and every 6 months up to 3 years for ctdna evaluation.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~within 14 days prior to treatment start for investigations of tumor tissue. at screening, week 13, week 25, week 37, week 49, week 61, at the end of treatment day 504 and every 6 months up to 3 years for ctdna evaluation.

This trial's timeline: 3 weeks for screening, Varies for treatment, and within 14 days prior to treatment start for investigations of tumor tissue. at screening, week 13, week 25, week 37, week 49, week 61, at the end of treatment day 504 and every 6 months up to 3 years for ctdna evaluation. for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Objective Response Rate

Secondary outcome measures

Determination of the Serum Level of Trametinib.

Evaluation of the Quality of Life During Treatment.

Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability).

+3 more

Other outcome measures

Comparison of responses with RECIST 1.1 and volumetric measurement for plexiform neurofibroma

Investigation and correlation of biological features to tumor response.

Neurocognitive assessment of NF1 patients between 1 and 42 months using the Bayley Scales of Infant and Toddlers Development, Third Edition (Bailey-III).

+4 more

Trial Design

4Treatment groups

Experimental Treatment

Group I: Progressing/refractory low grade-glioma, KIAA1549-BRAF fusionExperimental Treatment1 Intervention

Patients presenting with a progressing/refractory low-grade glioma with a KIAA1549-BRAF fusion.

Group II: Progressing/Refractory central nervous system (CNS) glioma.Experimental Treatment1 Intervention

Patients presenting with a progressing/refractory central nervous system glioma with an activation of the MAPK/ERK pathway who do not meet criteria for inclusion in other study groups.

Group III: Neurofibromatosis Type 1 (NF1) with low-grade gliomaExperimental Treatment1 Intervention

Patients presenting with Neurofibromatosis Type 1 (NF1) and a progressing/refractory low-grade glioma.

Group IV: Neurofibromatosis Type 1 (NF1) with Plexiform NeurofibromaExperimental Treatment1 Intervention

Patients presenting with Neurofibromatosis Type 1 (NF1) and a plexiform neurofibroma

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Trametinib

2014

Completed Phase 2

~1550

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Montreal Children's Hospital of the MUHCOTHER

29 Previous Clinical Trials

114,497 Total Patients Enrolled

St. Justine's HospitalLead Sponsor

196 Previous Clinical Trials

78,915 Total Patients Enrolled

CHU de Quebec-Universite LavalOTHER

168 Previous Clinical Trials

107,083 Total Patients Enrolled

Media Library

Trametinib (Mitogen-activated Protein Kinase (MAPK) Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT03363217 — Phase 2

$Plexiform Neurofibroma Research Study Groups: Progressing/refractory low grade-glioma, KIAA1549-BRAF fusion, Neurofibromatosis Type 1 (NF1) with Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1) with low-grade glioma, Progressing/Refractory central nervous system (CNS) glioma.$

Plexiform Neurofibroma Clinical Trial 2023: Trametinib Highlights & Side Effects. Trial Name: NCT03363217 — Phase 2

Trametinib (Mitogen-activated Protein Kinase (MAPK) Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03363217 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this research include young people in the target age group?

"Individuals that meet the age criteria of being over 1 month old but younger than 25 years are eligible for this trial."

Answered by AI

What are some of the known side effects of Trametinib?

"Trametinib's safety falls in the middle of our 1-to-3 scale at a 2. This is because, while there is some data suggesting that it is safe, there is no evidence that it is effective."

Answered by AI

Are there other instances of Trametinib being studied in a clinical setting?

"As of now, there are 96 ongoing clinical trials for Trametinib with 6 being in Phase 3. The largest amount of these trials are taking place in Saint Louis, Missouri; however, there are 5799 total locations running trials for Trametinib."

Answered by AI

How many different hospitals are conducting this study?

"Presently, there are 7 active clinical trial sites. They are situated in cities like Calgary, Quebec City and Halifax. It is best to select the closest location to you to limit travel time if you decide to participate."

Answered by AI

Are investigators actively working to find more participants for this clinical trial?

"According to the latest information on clinicaltrials.gov, this trial is not recruiting patients at this time. The study was first posted on 8/16/2018 and updated as recently as 10/25/2022; however, there are over 500 other trials that are actively looking for participants."

Answered by AI

How many people are included in this clinical trial at most?

"As of October 25th, 2022, this particular clinical trial is no longer recruiting patients. It was initially uploaded to the website on August 16th, 2018. For anyone still seeking medical trials to participate in, there are 413 studies for glioma and 96 for Trametinib that are currently enrolling patients."

Answered by AI

Who does this trial seek as participants?

"This clinical trial is recruiting 114 people with glioma who are between 1 month and 25 years old. The following criteria must be met: Assent from minor participants should be obtained per local laws and regulations and should be documented in accordance with local requirements, 8. Prior therapy Participants must have failed at least one line of treatment including chemotherapy and/or radiation therapy except for plexiform neurofibroma (since there is no recognized standard treatment for his tumor)., Age Patient must be aged ≥ 1 month (corrected age) to ≤ 25 years when starting trametinib., Study group Participants must belong to one of"

Answered by AI

Recent research and studies

BMC CancerJournal

A Phase 2 Study of Trametinib for Patients with Pediatric Glioma or Plexiform Neurofibroma with Refractory Tumor and Activation of the MAPK/ERK Pathway: TRAM-01

Perreault, Sébastien, Valérie Larouche, Uri Tabori, Cynthia Hawkin, Sarah Lippé, Benjamin Ellezam, Jean-Claude Décarie, et al.. 2019. “A Phase 2 Study of Trametinib for Patients with Pediatric Glioma or Plexiform Neurofibroma with Refractory Tumor and Activation of the MAPK/ERK Pathway: TRAM-01”. BMC Cancer. Springer Science and Business Media LLC. doi:10.1186/s12885-019-6442-2.

clinicaltrials.govStudy

Trametinib for Pediatric Neuro-oncology Patients With Refractory Tumor and Activation of the MAPK/ERK Pathway.

Dr Perreault 2018. "Trametinib for Pediatric Neuro-oncology Patients With Refractory Tumor and Activation of the MAPK/ERK Pathway.". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03363217.

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