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MEK Inhibitor

Binimetinib for Neurofibromatosis (NF108-BINI Trial)

Phase 2

Waitlist Available

Research Sponsored by University of Alabama at Birmingham

Eligibility Criteria Checklist

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

Must have

Clinical diagnosis of NF1 using the NIH Consensus Conference criteria OR a documented constitutional NF1 mutation

Plexiform neurofibroma(s) that are progressive or causing significant morbidity

Must not have

Impaired cardiovascular function or clinically significant cardiovascular diseases, including: History of acute coronary syndromes (including myocardial infarction, unstable angina, coronary artery bypass grafting, coronary angioplasty, or stenting) < 6 months prior to screening, Symptomatic chronic heart failure, history or current evidence of clinically significant cardiac arrhythmia and/or conduction abnormality < 6 months prior to screening except atrial fibrillation and paroxysmal supraventricular tachycardia, Other concurrent severe and/or uncontrolled medical disease, which could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe infection, severe malnutrition, chronic liver or renal disease, active upper GI tract ulceration, congestive heart failure, etc.), Subjects who have an uncontrolled infection, Known positive serology for HIV (human immunodeficiency virus), active hepatitis B, and/or active hepatitis C infection

Patients who have neuromuscular disorders that are associated with elevated CK (e.g., inflammatory myopathies, muscular dystrophy, amyotrophic lateral sclerosis, spinal muscular atrophy)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 24 months

Awards & highlights

No Placebo-Only Group

Summary

This trial will evaluate if the MEK inhibitor, binimetinib, is an effective treatment for children and adults with neurofibromatosis type 1 and inoperable plexiform neurofibromas.

Who is the study for?

This trial is for children over 1 year old and adults with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas. Participants must have a measurable tumor, be able to swallow pills, have adequate organ function, and not have had recent chemotherapy or major surgery. Pregnant women, those on chronic steroids or immunosuppressants, with certain eye conditions or uncontrolled diseases are excluded.

What is being tested?

The study tests binimetinib, a MEK inhibitor drug, on its ability to shrink tumors by at least 20% after up to 12 treatment cycles. It's an open-label phase II trial meaning everyone gets the drug and both researchers and participants know what's being given.

What are the potential side effects?

Potential side effects of binimetinib may include vision changes due to retinal vein occlusion or increased intraocular pressure; heart issues like hypertension; gastrointestinal problems; liver enzyme elevations; muscle damage indicated by elevated CK levels; allergic reactions similar to other compounds.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with NF1 either through clinical criteria or a genetic test.

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I have a growing nerve tumor causing significant health issues.

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I have a tumor that can be measured by MRI and is at least 3 mL in volume.

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I am 18 years old or older.

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I had surgery for a growing nerve tumor that couldn't be fully removed and can be measured.

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I haven't had chemotherapy that lowers my blood cell counts in the last 3 weeks.

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My blood counts meet the required levels without recent transfusions.

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My kidney function is good based on tests.

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My liver is functioning well according to recent tests.

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My heart is strong, with good pumping ability and normal rhythm.

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I cannot or do not want to have surgery for my plexiform neurofibroma.

Exclusion Criteria

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

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I have a muscle disorder that causes high CK levels, like muscular dystrophy.

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I have a brain or nerve tumor and needed treatment in the last year.

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I have had radiation therapy to my eye area before.

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My high blood pressure is severe and not controlled by medication.

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I do not have serious stomach or bowel problems.

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I have Gilbert's syndrome or am known to carry two copies of the UGT1A1*28 allele.

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I am on long-term steroids or other drugs that weaken my immune system.

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I have previously been treated with a MEK inhibitor.

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I am not pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from Baseline Target Tumor Volume at 12 months

Secondary study objectives

Incidence of Treatment-Emergent Adverse Events

Side effects data

From 2022 Phase 3 trial • 702 Patients • NCT02928224

78%

Diarrhoea

68%

Dermatitis acneiform

59%

Nausea

54%

Fatigue

51%

Vomiting

51%

Dry Skin

43%

Pyrexia

43%

Anaemia

41%

Decreased appetite

38%

Abdominal pain

38%

Constipation

35%

Dyspnoea

32%

Vision blurred

30%

Blood creatine increased

30%

Blood creatine phosphokinase increased

24%

Arthralgia

24%

Myalgia

24%

Skin fissures

22%

Back Pain

22%

Dizziness

19%

Malaise

19%

Urinary tract infection

19%

Headache

19%

Aspartate aminotransferase increased

16%

Asthenia

16%

Stomatitis

16%

Oedema peripheral

16%

PPE syndrome

16%

Hypomagnesaemia

16%

Rash maculo-papular

16%

Palmar-planar erythrodysaesthesia

16%

Chills

16%

Paronychia

16%

Rash pustular

16%

Alanine aminotransferase increased

16%

Dysgeusia

16%

Peripheral sensory neuropathy

14%

Cough

14%

Abdominal pain upper

14%

Infusion-related reaction

14%

Ejection fraction decreased

14%

Dry eye

11%

Trichiasis

11%

Pollakiuria

11%

Vitreous floaters

11%

Dyspepsia

11%

Hypoalbuminaemia

11%

Hypertension

11%

Tumour Pain

Rhinitis allergic

Infusion related reaction

Hypokalaemia

Visual impairment

Macular oedema

Hypertrichosis

Iron deficiency

Nasopharyngitis

Weight decreased

Flank pain

Proteinuria

Rash

Pruritus

Pain in extremity

Blood bilirubin increased

Rhinnorrhoea

Hypotension

Restless legs syndrome

Nervous system disorder

Wound

Trichomegaly

Infection

Pleural effusion

Hypocalcaemia

Hypophosphataemia

Rectal haemorrhage

Musculoskeletal pain

Anal haemorrhage

Ascites

Colitis

Abdominal pain lower

Nail disorder

Pruritus generalised

Bone pain

Musculoskeletal chest pain

Chorioretinopathy

Urinary incontinence

Insomnia

Gastroesophageal reflux disease

Abdominal distension

Eczema

Cystitis

Renal failure

Conjunctivitis

Syncope

Dehydration

Dry Mouth

Skin hyperpigmentation

Muscle spasms

Erythema

Retinal detachment

Pulmonary embolism

Dysphonia

Haematuria

Blood creatinine increased

Depression

Palpitations

Melanocytic naevus

Tumour pain

Kidney infection

Large intestinal ulcer

Large intestinal ulcer hemorrhage

Urinary tract infection bacterial

Epistaxis

Hyperkeratosis

Rectal hemorrhage

Streptococcal infection

Alopecia

Upper respiratory tract infection

Skin papilloma

Large intestine perforation

Bacterial sepsis

Cholangitis

Urinary tract obstruction

Confusional state

Device occlusion

Back pain

Rhabdomyolysis

Colon cancer

Sepsis

Acute kidney injury

Large intestine ulcer

Neutropenia

Bacteria sepsis

Hydronephrosis

Neuropathy peripheral

Abdominal abscess

Hyperglycaemia

100%

80%

60%

40%

20%

Study treatment Arm

Combined Safety Lead-in

Phase 3: Triplet Arm

Phase 3: Doublet Arm

Phase 3: Control Arm

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: Open label study of Binimetinib (MEK162)Experimental Treatment1 Intervention

Subjects (≥ 18 years) (Stratum A) will receive a course of binimetinib by mouth twice a day (12 hours apart) of 45 mg/dose. Duration of each course is 4 weeks. After 8 courses, subjects will receive additional courses if MRI results showed at least 15% reduction in volume of the target tumor. Subjects can continue on therapy and will be evaluated at the end of 12 courses. Subjects who have ≥ 20% reduction in volume of the target tumor according to the MRI results can continue therapy up to an additional year (maximum of 24 total courses). Subjects who have not met the tumor reduction at the specified times will be removed from the study therapy. Subjects will be carefully monitored for toxicities associated with binimetinib. Recruitment of subjects 1 - 17 years of age (Stratum B) is currently available. The pediatric maximum tolerated dose (MTD) of binimetinib the pediatric patients (Statum B) was established by a phase 1 study (NCT022).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Binimetinib

2018

Completed Phase 3

~1250

0 / 21Eligibility criteria met