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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

Must not have

Patients receiving cyclosporine, tacrolimus, or other agents to prevent graft-versus-host disease post bone marrow transplant or organ rejection post-transplant

Patients with a history of hepatic sinusoid obstructive syndrome (veno-occlusive disease) within the prior 3 months

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

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.

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.

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 below

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I am between 1 month and 22 years old.

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My leukemia (JMML) has come back or is not responding to treatment.

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I can do most activities but may need help.

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I can swallow pills or use a feeding tube.

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I have recovered from side effects of my previous cancer treatments.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am taking medication to prevent organ rejection after a transplant.

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I have had a liver condition called sinusoid obstructive syndrome in the last 3 months.

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I have a history or risk of eye vein blockage or swelling.

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My chronic conditions like high blood pressure or diabetes are under control.

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I do not have any infections that are currently uncontrolled.

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I have been diagnosed with Noonan syndrome.

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I am not on any cancer treatments except possibly hydroxyurea.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 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

Objective Response

Secondary study objectives

Complete Response

Duration of Response

Incidence of Adverse Events

+3 more

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

Productive cough

Upper respiratory tract infection

Rash maculo-papular

Mucosal inflammation

Hypercalcaemia

Gastrooesophageal reflux disease

Pleural effusion

Night sweats

Asthenia

Ejection fraction decreased

Electrocardiogram QT prolonged

Gamma-glutamyltransferase increased

Neutrophil count decreased

Neck pain

Rhinorrhoea

Haematochezia

Skin lesion

Acute kidney injury

Pulmonary embolism

Seborrhoeic keratosis

Thrombocytopenia

Atrial fibrillation

Stomatitis

Rhinitis allergic

Tachycardia

Abdominal pain upper

Hyperuricaemia

Leukopenia

Sinusitis

Urinary tract infection

Polyneuropathy

Haematuria

Neutropenia

Ear pain

Abdominal pain

Feeling cold

Non-cardiac chest pain

Pain

Fungal infection

Nasopharyngitis

Blood creatinine increased

Blood urea increased

Neutrophil count increased

Hypomagnesaemia

Myalgia

Neuropathy peripheral

Proteinuria

Nasal congestion

Pneumonitis

Palmar-plantar erythrodysaesthesia syndrome

Pelvic infection

Hypophosphataemia

Sepsis

Tinnitus

Malaise

Rhabdomyolysis

Cataract

Dermatitis acneiform

Erythema nodosum

Femoral neck fracture

Hyperglycaemic hyperosmolar nonketotic syndrome

Aortic thrombosis

Pollakiuria

Flushing

Oesophageal stenosis

Atrioventricular block first degree

Photophobia

Dehydration

Toothache

Oral candidiasis

Urinary retention

Alopecia

Skin mass

Aspiration

Vision blurred

Oedema

Depression

Folliculitis

Staphylococcal infection

Clavicle fracture

Aphasia

Cardiac ventricular thrombosis

Stress cardiomyopathy

Oral pain

Clostridium difficile infection

Diverticulitis

Pneumonia aspiration

Pneumonia necrotising

Wound infection

Hyperkalaemia

Rib fracture

Bladder transitional cell carcinoma

Facial nerve disorder

Hypertension

Paralysis recurrent laryngeal nerve

Syncope

Hallucination

Pulmonary haematoma

Sinus bradycardia

Dry eye

Abdominal distension

Dyspepsia

Gait disturbance

Nodule

Xerosis

Conjunctivitis

Tooth infection

Procedural pain

Blood creatine phosphokinase increased

Platelet count decreased

Weight increased

Flank pain

Muscular weakness

Musculoskeletal chest pain

Pain in extremity

Hypoaesthesia

Paraesthesia

Anxiety

Sleep disorder

Dysphonia

Epistaxis

Upper-airway cough syndrome

Wheezing

Erythema

100%

80%

60%

40%

20%

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

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

Bone Marrow Aspiration and Biopsy

2016

Completed Phase 1

~40

Trametinib

2014

Completed Phase 2

~1630