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MEK Inhibitor

Trametinib for Histiocytosis

Phase 2
Recruiting
Led By Anish Ray, MD
Research Sponsored by Cook Children's Health Care System
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients with newly diagnosed Langerhans cell histiocytosis (LCH)
Patients with relapsed or refractory LCH
Must not have
Patients diagnosed with Low-Risk True Skin Only or a Single Bone lesion not requiring treatment
Patients with class 3 MAP2K1 mutation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to six years
Awards & highlights
No Placebo-Only Group

Summary

This trial aims to test if trametinib, a specific type of treatment, is safe and works well in treating Langerhans Cell Histiocytosis (LCH) in children

Who is the study for?
This trial is for pediatric patients with newly diagnosed or relapsed Langerhans Cell Histiocytosis (LCH). Participants must have confirmed diagnosis, adequate heart function, and be able to follow study procedures. It's not for those with low-risk LCH, jaundice at diagnosis, trametinib allergy, pregnancy/breastfeeding, substance dependence or certain mutations.
What is being tested?
The trial tests the safety and effectiveness of Trametinib in treating LCH. This Phase II study focuses on children who are newly diagnosed or have not responded to previous treatments. The goal is to see how well this targeted therapy works against LCH.
What are the potential side effects?
Trametinib may cause side effects such as skin rash, diarrhea, fatigue and could potentially affect heart function. Since it targets specific pathways in cells that may be involved in normal body functions as well as cancer growth.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been recently diagnosed with Langerhans cell histiocytosis.
Select...
My LCH has come back or didn't respond to treatment.
Select...
I am taking the liquid form of trametinib for LCH.
Select...
I can care for myself and am up and about more than 50% of my waking hours.
Select...
I understand and can follow the study's procedures.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have a low-risk skin condition or a single bone issue that doesn't need treatment.
Select...
My cancer has a class 3 MAP2K1 mutation.
Select...
I had jaundice when my condition was diagnosed.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to six years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to six years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Overall Survival (OS)
Progression-free Survival (PFS)
Time to Progression (TTP)
Secondary study objectives
Blood HistioTrak Levels in Participants with BRAF V600E Mutations
Response Rate

Side effects data

From 2021 Phase 2 trial • 206 Patients • NCT02034110
47%
Pyrexia
36%
Fatigue
33%
Anaemia
33%
Nausea
33%
Decreased appetite
28%
Rash
22%
Headache
22%
Constipation
22%
Pneumonia
22%
Chills
22%
Dyspnoea
19%
Dizziness
19%
Hypoalbuminaemia
19%
Vomiting
19%
Diarrhoea
19%
Hyponatraemia
17%
Dysphagia
17%
Blood alkaline phosphatase increased
17%
Back pain
14%
Aspartate aminotransferase increased
14%
Hypocalcaemia
14%
Dry mouth
14%
Hyperglycaemia
14%
Arthralgia
14%
Oedema peripheral
14%
White blood cell count decreased
14%
Insomnia
14%
Hypotension
11%
Hypokalaemia
11%
Haemoptysis
11%
Alanine aminotransferase increased
11%
Dry skin
11%
Hypothyroidism
11%
Pruritus
11%
Visual impairment
11%
Weight decreased
11%
Cough
8%
Productive cough
8%
Rash maculo-papular
8%
Upper respiratory tract infection
8%
Mucosal inflammation
8%
Hypercalcaemia
8%
Gastrooesophageal reflux disease
8%
Pleural effusion
8%
Night sweats
8%
Asthenia
8%
Ejection fraction decreased
8%
Electrocardiogram QT prolonged
8%
Gamma-glutamyltransferase increased
8%
Neutrophil count decreased
8%
Neck pain
6%
Rhinorrhoea
6%
Haematochezia
6%
Skin lesion
6%
Acute kidney injury
6%
Seborrhoeic keratosis
6%
Pulmonary embolism
6%
Thrombocytopenia
6%
Atrial fibrillation
6%
Stomatitis
6%
Rhinitis allergic
6%
Tachycardia
6%
Abdominal pain upper
6%
Hyperuricaemia
6%
Leukopenia
6%
Sinusitis
6%
Urinary tract infection
6%
Polyneuropathy
6%
Haematuria
6%
Neutropenia
6%
Ear pain
6%
Abdominal pain
6%
Feeling cold
6%
Non-cardiac chest pain
6%
Pain
6%
Fungal infection
6%
Nasopharyngitis
6%
Blood creatinine increased
6%
Blood urea increased
6%
Neutrophil count increased
6%
Hypomagnesaemia
6%
Myalgia
6%
Neuropathy peripheral
6%
Proteinuria
6%
Nasal congestion
6%
Pneumonitis
6%
Palmar-plantar erythrodysaesthesia syndrome
3%
Pelvic infection
3%
Hypophosphataemia
3%
Sepsis
3%
Tinnitus
3%
Malaise
3%
Cataract
3%
Rhabdomyolysis
3%
Dermatitis acneiform
3%
Erythema nodosum
3%
Femoral neck fracture
3%
Aortic thrombosis
3%
Hyperglycaemic hyperosmolar nonketotic syndrome
3%
Pollakiuria
3%
Flushing
3%
Oesophageal stenosis
3%
Atrioventricular block first degree
3%
Photophobia
3%
Dehydration
3%
Toothache
3%
Oral candidiasis
3%
Urinary retention
3%
Alopecia
3%
Skin mass
3%
Aspiration
3%
Vision blurred
3%
Oedema
3%
Depression
3%
Folliculitis
3%
Staphylococcal infection
3%
Clavicle fracture
3%
Aphasia
3%
Cardiac ventricular thrombosis
3%
Stress cardiomyopathy
3%
Oral pain
3%
Clostridium difficile infection
3%
Diverticulitis
3%
Pneumonia aspiration
3%
Pneumonia necrotising
3%
Wound infection
3%
Hyperkalaemia
3%
Rib fracture
3%
Bladder transitional cell carcinoma
3%
Facial nerve disorder
3%
Hypertension
3%
Paralysis recurrent laryngeal nerve
3%
Syncope
3%
Hallucination
3%
Pulmonary haematoma
3%
Sinus bradycardia
3%
Dry eye
3%
Abdominal distension
3%
Dyspepsia
3%
Gait disturbance
3%
Nodule
3%
Xerosis
3%
Conjunctivitis
3%
Tooth infection
3%
Procedural pain
3%
Blood creatine phosphokinase increased
3%
Platelet count decreased
3%
Weight increased
3%
Flank pain
3%
Muscular weakness
3%
Musculoskeletal chest pain
3%
Pain in extremity
3%
Hypoaesthesia
3%
Paraesthesia
3%
Anxiety
3%
Sleep disorder
3%
Dysphonia
3%
Epistaxis
3%
Upper-airway cough syndrome
3%
Wheezing
3%
Erythema
100%
80%
60%
40%
20%
0%
Study treatment Arm
Anaplastic Thyroid Cancer (ATC) (On-Treatment)
Biliary Tract Cancer (BTC) (On-Treatment)
Gastrointestinal Stromal Tumor (GIST) (On-Treatment)
Low Grade (WHO G1/G2) Glioma (LGG) (On-Treatment)
Anaplastic Thyroid Cancer (ATC) (Post-treatment Survival Follow-up)
Gastrointestinal Stromal Tumor (GIST) (Post-treatment Survival Follow-up)
Low Grade (WHO G1/G2) Glioma (LGG) (Post-treatment Survival Follow-up)
Adenocarcinoma of the Small Intestine (ASI) (Post-treatment Survival Follow-up)
Hairy Cell Leukemia (HCL) (Post-treatment Survival Follow-up)
High Grade (WHO G3/G4) Glioma (HGG) (On-Treatment)
Hairy Cell Leukemia (HCL) (On-Treatment)
Multiple Myeloma (MM) (On-Treatment)
High Grade (WHO G3/G4) Glioma (HGG) (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)

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Active Control
Group I: Prospective TreatmentExperimental Treatment1 Intervention
Trametinib will be administered in 28-day cycles, given once daily or adjusted as per clinical judgment of the treating physician, with a maximum dosage of 2mg daily. Patients will be followed for 4 years after receiving treatment for two years. Patients may continue on the same treatment beyond two years if they and their treating physicians agree to do so in the best interest of the patient.
Group II: Observation OnlyActive Control1 Intervention
Patients who have been receiving trametinib as a treatment for LCH since January 1, 2020 may be included in an observational chart review to track long-term follow-up.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Trametinib
2014
Completed Phase 2
~1630

Find a Location

Who is running the clinical trial?

Cook Children's Health Care SystemLead Sponsor
9 Previous Clinical Trials
5,000,784 Total Patients Enrolled
Anish Ray, MDPrincipal InvestigatorCook Children's Health Care System
~50 spots leftby Jun 2039
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