Your session is about to expire
← Back to Search
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 belowSelect...
I am between 1 month and 25 years old and starting trametinib treatment.
Select...
I have a specific type of progressing brain tumor or nerve tumor.
Select...
I can do most activities but may need help.
Select...
My organ and bone marrow functions are normal.
Select...
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.
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 moreOther 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 moreSide effects data
From 2021 Phase 2 trial • 206 Patients • NCT0203411058%
Pyrexia
42%
Nausea
35%
Vomiting
33%
Diarrhoea
33%
Fatigue
28%
Gamma-glutamyltransferase increased
28%
Chills
28%
Rash
26%
Aspartate aminotransferase increased
23%
White blood cell count decreased
23%
Cough
23%
Anaemia
23%
Decreased appetite
23%
Headache
21%
Constipation
21%
Blood alkaline phosphatase increased
19%
Dry mouth
19%
Hyperglycaemia
19%
Myalgia
16%
Alanine aminotransferase increased
16%
Asthenia
16%
Abdominal pain upper
16%
Thrombocytopenia
16%
Dyspnoea
14%
Hypertension
14%
Arthralgia
14%
Insomnia
12%
Platelet count decreased
12%
Eczema
12%
Blood creatinine increased
12%
Pruritus
12%
Abdominal pain
12%
Hypomagnesaemia
12%
Hyponatraemia
12%
Erythema
9%
Rash maculo-papular
9%
Oedema peripheral
9%
Hypokalaemia
9%
Dermatitis acneiform
9%
Neutrophil count decreased
9%
Weight increased
9%
Back pain
9%
Dry skin
7%
Blood bilirubin increased
7%
Sepsis
7%
Neutropenia
7%
Mucosal inflammation
7%
Oedema
7%
Cholangitis
7%
Hypoalbuminaemia
7%
Stomatitis
7%
Dyspepsia
7%
Influenza like illness
7%
Herpes zoster
7%
Glycosylated haemoglobin increased
7%
Weight decreased
7%
Musculoskeletal pain
7%
Pain in extremity
7%
Anxiety
7%
Acute kidney injury
7%
Night sweats
5%
Hypophosphataemia
5%
Skin lesion
5%
Erythema nodosum
5%
Dry eye
5%
Toothache
5%
Muscle spasms
5%
Blood creatine phosphokinase increased
5%
Hyperuricaemia
5%
Gastrooesophageal reflux disease
5%
Leukopenia
5%
Folliculitis
5%
Urinary tract infection
5%
Fall
5%
Lymphocyte count decreased
5%
Hypoglycaemia
5%
Joint swelling
5%
Neuropathy peripheral
5%
Epistaxis
5%
Oropharyngeal pain
5%
Acne
5%
Hyperkeratosis
2%
Femoral neck fracture
2%
Productive cough
2%
Hot flush
2%
Hypersensitivity
2%
Upper respiratory tract infection
2%
Pain in jaw
2%
Epilepsy
2%
Peripheral sensory neuropathy
2%
Depression
2%
Hypercalcaemia
2%
Dizziness
2%
Skin mass
2%
Pneumonia
2%
Thrombophlebitis
2%
Tachycardia
2%
Musculoskeletal chest pain
2%
Dysgeusia
2%
Sciatica
2%
Gastroenteritis
2%
Febrile neutropenia
2%
Atrial fibrillation
2%
Oral pain
2%
Rectal haemorrhage
2%
Paronychia
2%
Device related infection
2%
Rhinitis
2%
Sinusitis
2%
Respiratory tract infection
2%
Hyperkalaemia
2%
Transaminases increased
2%
Dehydration
2%
Spinal pain
2%
Haemorrhoids
2%
Cataract
2%
Eye pain
2%
Vision blurred
2%
Visual impairment
2%
Abdominal discomfort
2%
Abdominal distension
2%
Gait disturbance
2%
Non-cardiac chest pain
2%
Xerosis
2%
Conjunctivitis
2%
Nasopharyngitis
2%
Rash pustular
2%
Tooth abscess
2%
Tooth infection
2%
Procedural pain
2%
Blood lactate dehydrogenase increased
2%
Blood uric acid increased
2%
C-reactive protein increased
2%
Muscular weakness
2%
Neck pain
2%
Basal cell carcinoma
2%
Seborrhoeic keratosis
2%
Paraesthesia
2%
Proteinuria
2%
Renal failure
2%
Nasal congestion
2%
Pleural effusion
2%
Nail discolouration
2%
Palmar-plantar erythrodysaesthesia syndrome
2%
Photosensitivity reaction
100%
80%
60%
40%
20%
0%
Study treatment Arm
Biliary Tract Cancer (BTC) (On-Treatment)
Anaplastic Thyroid Cancer (ATC) (Post-treatment Survival Follow-up)
Anaplastic Thyroid Cancer (ATC) (On-Treatment)
Hairy Cell Leukemia (HCL) (Post-treatment Survival Follow-up)
Hairy Cell Leukemia (HCL) (On-Treatment)
Low Grade (WHO G1/G2) Glioma (LGG) (Post-treatment Survival Follow-up)
Low Grade (WHO G1/G2) Glioma (LGG) (On-Treatment)
Multiple Myeloma (MM) (On-Treatment)
High Grade (WHO G3/G4) Glioma (HGG) (Post-treatment Survival Follow-up)
High Grade (WHO G3/G4) Glioma (HGG) (On-Treatment)
Gastrointestinal Stromal Tumor (GIST) (On-Treatment)
Adenocarcinoma of the Small Intestine (ASI) (Post-treatment Survival Follow-up)
Gastrointestinal Stromal Tumor (GIST) (Post-treatment Survival Follow-up)
Adenocarcinoma of the Small Intestine (ASI) (On-Treatment)
Biliary Tract Cancer (BTC) (Post-treatment Survival Follow-up)
Multiple Myeloma (MM) (Post-treatment Survival Follow-up)
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
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
167 Previous Clinical Trials
107,043 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have a specific type of progressing brain tumor or nerve tumor.I am between 1 month and 25 years old and starting trametinib treatment.My condition worsened while on a MEK inhibitor treatment.I had major surgery less than 2 weeks before starting trametinib.I do not have severe illnesses like uncontrolled liver disease, HIV, or hepatitis.I can enroll without tumor tissue if I have NF1 and LGG or PN and no surgery was done.I have tried at least one treatment like chemotherapy or radiation, but not for plexiform neurofibroma.I have recovered from the side effects of my previous cancer treatments.I can do most activities but may need help.My organ and bone marrow functions are normal.I can swallow and keep down pills without significant stomach issues affecting absorption.My tumor has a positive BRAF V600E mutation.My heart's pumping ability is below normal, or I have a long QT interval.I have another cancer, but it's not currently serious or needing treatment.
Research Study Groups:
This trial has the following groups:- Group 1: Progressing/refractory low grade-glioma, KIAA1549-BRAF fusion
- Group 2: Neurofibromatosis Type 1 (NF1) with Plexiform Neurofibroma
- Group 3: Neurofibromatosis Type 1 (NF1) with low-grade glioma
- Group 4: Progressing/Refractory central nervous system (CNS) glioma.
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger