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MEK Inhibitor
Trametinib for Juvenile Myelomonocytic Leukemia
Phase 2
Waitlist Available
Led By Elliot Stieglitz
Research Sponsored by National Cancer Institute (NCI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2-year
Awards & highlights
Study Summary
This trial looks at how well trametinib works in treating patients with JML who have relapsed or who don't respond to treatment. Trametinib may stop tumor cell growth by blocking enzymes needed for cell growth.
Who is the study for?
This trial is for young patients under 22 years with juvenile myelomonocytic leukemia (JMML) that's relapsed or not responding to treatment. They must have recovered from previous treatments, be able to swallow pills, and meet specific health criteria like heart function and normal blood levels.Check my eligibility
What is being tested?
The study tests trametinib effectiveness in treating JMML that has returned or is refractory. Trametinib aims to halt tumor growth by inhibiting enzymes necessary for cell proliferation. The process includes bone marrow procedures alongside medication administration.See study design
What are the potential side effects?
Trametinib may cause side effects such as skin rash, diarrhea, fatigue, nausea and swelling of the face or limbs due to its action on cellular enzymes. Heart rhythm abnormalities could also occur given the QT interval requirement.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 1 month and 22 years old.
Select...
My leukemia (JMML) has come back or is not responding to treatment.
Select...
I can do most activities but may need help.
Select...
I can swallow pills or use a feeding tube.
Select...
I have recovered from side effects of my previous cancer treatments.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 2-year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2-year
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Objective response
Secondary outcome measures
Complete Response
Duration of Response
Incidence of adverse events
+3 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%
Oedema peripheral
9%
Hypokalaemia
9%
Dermatitis acneiform
9%
Rash maculo-papular
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%
Hot flush
2%
Femoral neck fracture
2%
Hypersensitivity
2%
Upper respiratory tract infection
2%
Productive cough
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)
Hairy Cell Leukemia (HCL) (On-Treatment)
Hairy Cell Leukemia (HCL) (Post-treatment Survival Follow-up)
Anaplastic Thyroid Cancer (ATC) (On-Treatment)
Low Grade (WHO G1/G2) Glioma (LGG) (On-Treatment)
High Grade (WHO G3/G4) Glioma (HGG) (On-Treatment)
Anaplastic Thyroid Cancer (ATC) (Post-treatment Survival Follow-up)
Low Grade (WHO G1/G2) Glioma (LGG) (Post-treatment Survival Follow-up)
High Grade (WHO G3/G4) Glioma (HGG) (Post-treatment Survival Follow-up)
Adenocarcinoma of the Small Intestine (ASI) (Post-treatment Survival Follow-up)
Multiple Myeloma (MM) (On-Treatment)
Gastrointestinal Stromal Tumor (GIST) (Post-treatment Survival Follow-up)
Gastrointestinal Stromal Tumor (GIST) (On-Treatment)
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
1Treatment groups
Experimental Treatment
Group I: Treatment (trametinib)Experimental Treatment2 Interventions
Patients receive trametinib PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo a bone marrow aspiration or biopsy at baseline, on day 28 of cycles 1 and 2, at all subsequent odd numbered cycles, and at end of treatment.
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?
National Cancer Institute (NCI)Lead Sponsor
13,654 Previous Clinical Trials
40,933,143 Total Patients Enrolled
Children's Oncology GroupNETWORK
453 Previous Clinical Trials
237,690 Total Patients Enrolled
Elliot StieglitzPrincipal InvestigatorChildren's Oncology Group
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am between 1 month and 22 years old.I am taking medication to prevent organ rejection after a transplant.I have had a liver condition called sinusoid obstructive syndrome in the last 3 months.I have a history or risk of eye vein blockage or swelling.My leukemia (JMML) has come back or is not responding to treatment.I can do most activities but may need help.My chronic conditions like high blood pressure or diabetes are under control.I can swallow pills or use a feeding tube.I do not have any infections that are currently uncontrolled.I have been diagnosed with Noonan syndrome.I have recovered from side effects of my previous cancer treatments.I am not on any cancer treatments except possibly hydroxyurea.
Research Study Groups:
This trial has the following groups:- Group 1: Treatment (trametinib)
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.
Can people over the age of 20 participate in this research?
"This clinical trial is only for paediatric patients that fall in the 1 month to 21 year age range. Out of the 4,885 active clinical trials, 494 are for patients under 18 and 1291 are for senior citizens."
Answered by AI
Could you tell me if this research is currently ongoing and recruiting participants?
"That is correct. The trial, which began recruiting on October 6th 2017, as indicated by clinicaltrials.gov, is still looking for 24 more patients to be admitted at 54 different sites."
Answered by AI
When was the last time Trametinib received a thumbs up from the FDA?
"Trametinib's rating is a 2. Although there is some evidence affirming its safety, no studies have looked into this medication's effectiveness."
Answered by AI
Are there other examples in the medical literature of Trametinib's efficacy?
"Trametinib was first used in 2012 during a clinical trial at University of Chicago Medical Center. 155 such studies have completed, with 96 more ongoing. Many of the active trials are based in Austin, Texas."
Answered by AI
What is the total number of test subjects in this clinical trial?
"A total of 24 individuals are required for this clinical trial, all of which must meet the pre-specified inclusion criteria. The Dell Children's Medical Center of Central Texas in Austin and Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center in Houston are two locations where patients can take part in this study."
Answered by AI
Where are the different sites for this trial taking place?
"In total, there are 57 sites where patients can enroll in this trial, including Dell Children's Medical Center of Central Texas in Austin, Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center in Houston, and Nemours Children's Hospital in Orlando."
Answered by AI
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