CLINICAL TRIAL

Binimetinib for Neurofibromatoses

Grade III
Waitlist Available · Any Age · All Sexes · Chicago, IL

This study is evaluating whether a drug called binimetinib can shrink tumors in people with neurofibromatosis type 1.

See full description

About the trial for Neurofibromatoses

Eligible Conditions
Neurofibromatosis Type 1 (NF1) · Neurofibromatosis 1 · Neurofibroma, Plexiform · Plexiform Neurofibromas (PN) · Neurofibroma · Neurofibromatoses

Treatment Groups

This trial involves 2 different treatments. Binimetinib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Binimetinib
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Binimetinib
FDA approved

Side Effect Profile for Dose Escalation Phase 2 PF-0341066 and Binimetinib

Dose Escalation Phase 2 PF-0341066 and Binimetinib
Show all side effects
70%
Diarrhoea
55%
Nausea
50%
Fatigue
40%
Rash Acneiform
35%
Blood Creatine Phosphokinase Increased
35%
Rash
30%
Cough
30%
Asthenia
30%
Vomiting
20%
Constipation
20%
Dizziness
15%
Aspartate Aminotransferase Increased
15%
Lung Infection
15%
Alanine Aminotransferase Increased
15%
Hypoalbuminaemia
15%
Myalgia
15%
Periorbital Oedema
15%
Peripheral Sensory Neuropathy
15%
Skin Rash
10%
Sore Throat
10%
Alopecia
10%
Blood Potassium Decreased
10%
Oedema
10%
Productive Cough
10%
Pleural Effusion
10%
Peripheral Oedema
10%
Leg Oedema
10%
Liver Pain
10%
Rash Face
5%
C-Reactive Protein Increased
5%
Genital Itching Nos
5%
Ear Disorder
5%
Neutropenia
5%
Gout
5%
Extrasystoles
5%
Oedema Extremities
5%
Pericardial Effusion
5%
Dyspnea
5%
Ascitis
5%
Brain Metastases
5%
Abdominal Pain
5%
Pain
5%
Pneumonitis
5%
Arthralgia
5%
Blood Sodium Decreased
5%
Ascites
5%
Anaemia
5%
Dry Eye
5%
Chest Pain
5%
Blood Pressure Increased
5%
Bradycardia
5%
Dry Skin
5%
Chills
5%
Dysphonia
5%
Ear infection
5%
Dizziness Postural
5%
Dyspepsia
5%
Dry Mouth
5%
Ejection Fraction Decreased
5%
Hand Swelling
5%
Eye disorder
5%
Flank pain
5%
Gastrooesophageal Reflux
5%
Epigastric Pain
5%
Fever
5%
Flu like symptoms
5%
Heartburn
5%
Hoarseness
5%
Haematoma
5%
Hyperglycaemia
5%
Hypersensitivity
5%
K+ Decreased
5%
Left Ventricular Ejection Fraction Decreased
5%
Mucositis
5%
Maculopapular Rash
5%
Malaise
5%
Musculoskeletal Pain
5%
Neuropathic Pain
5%
Neuropathy Peripheral
5%
Oedema Limbs
5%
Osteoarticular Pain
5%
Oedema Of Lower Extremities
5%
Pain In Hip
5%
Paraesthesia
5%
Platelet Count Decreased
5%
Periorbital Infection
5%
Purpura
5%
Pneumonia
5%
Presyncope
5%
Sodium Decreased
5%
Runny Nose
5%
Seborrhoeic Dermatitis
5%
Retrosternal Chest Pain
5%
Rosacea
5%
Skin Oedema
5%
Upper Respiratory Tract Infection
5%
Swelling Of Legs
5%
Transaminases Increased
5%
Tremor
5%
Vision Abnormal
5%
Postural hypotension
5%
Thromboembolic Event
5%
Skin Infection
5%
Amnesia
5%
Blepharitis
5%
Device Related Infection
5%
Foot Oedema
5%
Pulmonary Embolism
5%
Pustular Rash
5%
Rigors
5%
Swelling Of Hands
5%
Thrombosis
5%
Weight Gain
5%
Wheeze
0%
Neurotoxicity
0%
Qt Prolonged
0%
Body Temperature Increased
0%
Oedema Legs
0%
Sore Gums
0%
Visual Disturbance
0%
Stoma Site Bleeding
0%
Weakness Left Or Right Side
0%
Abdominal Infection
0%
Dehydration
0%
Cytolysis
0%
Central Serous Retinopathy (Bilateral)
0%
Edema Face
0%
Abdominal Pain Lower
0%
Postoperative Hemorrhage
0%
Tingling In Lower Limbs
0%
Osteonecrosis Of Jaw
0%
Abdominal abscess
0%
Worsening Back Pain
0%
Wound Infection
0%
Anxiety
0%
Back pain
0%
Abdominal Distension
0%
Cardiac Failure
0%
Conjunctival Haemorrhage
0%
Blood Albumin Decreased
0%
Blood Glucose Increased
0%
Blood Lactate Dehydrogenase Increased
0%
Cataract Operation
0%
Chorioretinopathy
0%
Candida Infection
0%
Confusional State
0%
Dysgeusia
0%
Epistaxis
0%
Edema Limbs
0%
Cognitive Disorder Cognitive Disorder
0%
Facial rash
0%
Gamma-Glutamyltransferase Increased
0%
Edema extremities
0%
Edema Lower Limb
0%
Edema
0%
Edema of legs
0%
Electrocardiogram Qtc Interval Prolonged
0%
Generalised Oedema
0%
Folliculitis
0%
Expressive Dysphasia
0%
Eyelid Function Disorder
0%
Exertional Dyspnoea
0%
Fall
0%
Finger cramps
0%
Frequent Bowel Movements
0%
Headache
0%
Gastritis
0%
Hot Flushes
0%
Ggt Increased
0%
Glucose Increased
0%
Haemorrhoids
0%
Haematuria
0%
Hypertension
0%
Hepatic Haemorrhage
0%
Herpes Lesion Intra-Oral
0%
Hypertransaminasaemia
0%
Intestinal Stoma Complication
0%
Itchy Scalp
0%
Localised Numbness
0%
Muscle Ache
0%
Libido Decreased
0%
Lower Respiratory Tract Infection
0%
Macular Rash
0%
Loose Stools
0%
Low Back Pain
0%
Low Mood
0%
Lymphoedema
0%
Muscle Weakness
0%
Nausea And Vomiting
0%
Myalgia Of Lower Extremities
0%
Nasal Congestion
0%
Neck rash
0%
Neck Pain
0%
Neuropathy
0%
Oedema Arms
0%
Oedema Abdomen
0%
Night Sweats
0%
Nose Bleeds
0%
Oesophagitis
0%
Oral Candida
0%
Orofacial Oedema
0%
Paraesthesia Lower Limb
0%
Pain In Thigh
0%
Papular Rash
0%
Pain Jaw
0%
Painful Hips
0%
Parotiditis
0%
Photophobia
0%
Prolapse Of Intestinal Stoma
0%
Paronychia
0%
Pneumothorax
0%
Postural Hypotension
0%
Pruritis
0%
Pleurisy
0%
Postprandial Emesis
0%
Protein Total Decreased
0%
Sinus Bradycardia
0%
Skin Lesion
0%
Rash On Legs & Arms
0%
Renal Impairment
0%
Rib Pain
0%
Right Upper Quadrant Pain
0%
Scar
0%
Skin Peeling
0%
Retinal Detachment
0%
Shoulder Pain
0%
Retinal Hemorrhage
0%
Serous Discharge
0%
Skin Toxicity
0%
Subconjunctival Haemorrhage
0%
Stomach Cramps
0%
Transaminitis
0%
Visual Field Defect
0%
Stomach Pain
0%
Thrombocytopenia
0%
Troponin Increased
0%
Urinary Tract Infection
0%
Vascular Access Complication
0%
Urine Incontinence
0%
Urinary Frequency
0%
Vasovagal Attack
0%
Vertigo
0%
Weight Loss
0%
Shortness Of Breath
0%
Bowel Obstruction
0%
Caridac Failure
0%
Colonic Obstruction
0%
Hyperglycemia
0%
Hypoalbuminemia
0%
Lower Respiratory Infection
0%
Pericarditis
0%
Hypertransaminasemia
0%
Lower Gastrointestinal Hemorrhage
0%
Anorexia
0%
Aphasia
0%
Blood Alkaline Phosphatase Increased
0%
Blood Creatinine Increased
0%
Corneal Opacity
0%
Decreased Appetite
0%
Depressed Mood
0%
Dermatitis Acneiform
0%
Hyperbilirubinaemia
0%
Furunculosis
0%
Hypokalaemia
0%
Hypoglycaemia
0%
Hypotension
0%
Insomnia
0%
Intercostal Pain
0%
Intestinal Obstruction
0%
Intra-Abdominal Abscess
0%
Joint Instability
0%
Ldh Increased
0%
Rectal Pain
0%
Respiratory Distress
0%
Stomatitis
0%
Teeth Chattering
0%
Wound Pain
Diarrhoea
70%
Nausea
55%
Fatigue
50%
Rash Acneiform
40%
Blood Creatine Phosphokinase Increased
35%
Rash
35%
Cough
30%
Asthenia
30%
Vomiting
30%
Constipation
20%
Dizziness
20%
Aspartate Aminotransferase Increased
15%
Lung Infection
15%
Alanine Aminotransferase Increased
15%
Hypoalbuminaemia
15%
Myalgia
15%
Periorbital Oedema
15%
Peripheral Sensory Neuropathy
15%
Skin Rash
15%
Sore Throat
10%
Alopecia
10%
Blood Potassium Decreased
10%
Oedema
10%
Productive Cough
10%
Pleural Effusion
10%
Peripheral Oedema
10%
Leg Oedema
10%
Liver Pain
10%
Rash Face
10%
C-Reactive Protein Increased
5%
Genital Itching Nos
5%
Ear Disorder
5%
Neutropenia
5%
Gout
5%
Extrasystoles
5%
Oedema Extremities
5%
Pericardial Effusion
5%
Dyspnea
5%
Ascitis
5%
Brain Metastases
5%
Abdominal Pain
5%
Pain
5%
Pneumonitis
5%
Arthralgia
5%
Blood Sodium Decreased
5%
Ascites
5%
Anaemia
5%
Dry Eye
5%
Chest Pain
5%
Blood Pressure Increased
5%
Bradycardia
5%
Dry Skin
5%
Chills
5%
Dysphonia
5%
Ear infection
5%
Dizziness Postural
5%
Dyspepsia
5%
Dry Mouth
5%
Ejection Fraction Decreased
5%
Hand Swelling
5%
Eye disorder
5%
Flank pain
5%
Gastrooesophageal Reflux
5%
Epigastric Pain
5%
Fever
5%
Flu like symptoms
5%
Heartburn
5%
Hoarseness
5%
Haematoma
5%
Hyperglycaemia
5%
Hypersensitivity
5%
K+ Decreased
5%
Left Ventricular Ejection Fraction Decreased
5%
Mucositis
5%
Maculopapular Rash
5%
Malaise
5%
Musculoskeletal Pain
5%
Neuropathic Pain
5%
Neuropathy Peripheral
5%
Oedema Limbs
5%
Osteoarticular Pain
5%
Oedema Of Lower Extremities
5%
Pain In Hip
5%
Paraesthesia
5%
Platelet Count Decreased
5%
Periorbital Infection
5%
Purpura
5%
Pneumonia
5%
Presyncope
5%
Sodium Decreased
5%
Runny Nose
5%
Seborrhoeic Dermatitis
5%
Retrosternal Chest Pain
5%
Rosacea
5%
Skin Oedema
5%
Upper Respiratory Tract Infection
5%
Swelling Of Legs
5%
Transaminases Increased
5%
Tremor
5%
Vision Abnormal
5%
Postural hypotension
5%
Thromboembolic Event
5%
Skin Infection
5%
Amnesia
5%
Blepharitis
5%
Device Related Infection
5%
Foot Oedema
5%
Pulmonary Embolism
5%
Pustular Rash
5%
Rigors
5%
Swelling Of Hands
5%
Thrombosis
5%
Weight Gain
5%
Wheeze
5%
Neurotoxicity
0%
Qt Prolonged
0%
Body Temperature Increased
0%
Oedema Legs
0%
Sore Gums
0%
Visual Disturbance
0%
Stoma Site Bleeding
0%
Weakness Left Or Right Side
0%
Abdominal Infection
0%
Dehydration
0%
Cytolysis
0%
Central Serous Retinopathy (Bilateral)
0%
Edema Face
0%
Abdominal Pain Lower
0%
Postoperative Hemorrhage
0%
Tingling In Lower Limbs
0%
Osteonecrosis Of Jaw
0%
Abdominal abscess
0%
Worsening Back Pain
0%
Wound Infection
0%
Anxiety
0%
Back pain
0%
Abdominal Distension
0%
Cardiac Failure
0%
Conjunctival Haemorrhage
0%
Blood Albumin Decreased
0%
Blood Glucose Increased
0%
Blood Lactate Dehydrogenase Increased
0%
Cataract Operation
0%
Chorioretinopathy
0%
Candida Infection
0%
Confusional State
0%
Dysgeusia
0%
Epistaxis
0%
Edema Limbs
0%
Cognitive Disorder Cognitive Disorder
0%
Facial rash
0%
Gamma-Glutamyltransferase Increased
0%
Edema extremities
0%
Edema Lower Limb
0%
Edema
0%
Edema of legs
0%
Electrocardiogram Qtc Interval Prolonged
0%
Generalised Oedema
0%
Folliculitis
0%
Expressive Dysphasia
0%
Eyelid Function Disorder
0%
Exertional Dyspnoea
0%
Fall
0%
Finger cramps
0%
Frequent Bowel Movements
0%
Headache
0%
Gastritis
0%
Hot Flushes
0%
Ggt Increased
0%
Glucose Increased
0%
Haemorrhoids
0%
Haematuria
0%
Hypertension
0%
Hepatic Haemorrhage
0%
Herpes Lesion Intra-Oral
0%
Hypertransaminasaemia
0%
Intestinal Stoma Complication
0%
Itchy Scalp
0%
Localised Numbness
0%
Muscle Ache
0%
Libido Decreased
0%
Lower Respiratory Tract Infection
0%
Macular Rash
0%
Loose Stools
0%
Low Back Pain
0%
Low Mood
0%
Lymphoedema
0%
Muscle Weakness
0%
Nausea And Vomiting
0%
Myalgia Of Lower Extremities
0%
Nasal Congestion
0%
Neck rash
0%
Neck Pain
0%
Neuropathy
0%
Oedema Arms
0%
Oedema Abdomen
0%
Night Sweats
0%
Nose Bleeds
0%
Oesophagitis
0%
Oral Candida
0%
Orofacial Oedema
0%
Paraesthesia Lower Limb
0%
Pain In Thigh
0%
Papular Rash
0%
Pain Jaw
0%
Painful Hips
0%
Parotiditis
0%
Photophobia
0%
Prolapse Of Intestinal Stoma
0%
Paronychia
0%
Pneumothorax
0%
Postural Hypotension
0%
Pruritis
0%
Pleurisy
0%
Postprandial Emesis
0%
Protein Total Decreased
0%
Sinus Bradycardia
0%
Skin Lesion
0%
Rash On Legs & Arms
0%
Renal Impairment
0%
Rib Pain
0%
Right Upper Quadrant Pain
0%
Scar
0%
Skin Peeling
0%
Retinal Detachment
0%
Shoulder Pain
0%
Retinal Hemorrhage
0%
Serous Discharge
0%
Skin Toxicity
0%
Subconjunctival Haemorrhage
0%
Stomach Cramps
0%
Transaminitis
0%
Visual Field Defect
0%
Stomach Pain
0%
Thrombocytopenia
0%
Troponin Increased
0%
Urinary Tract Infection
0%
Vascular Access Complication
0%
Urine Incontinence
0%
Urinary Frequency
0%
Vasovagal Attack
0%
Vertigo
0%
Weight Loss
0%
Shortness Of Breath
0%
Bowel Obstruction
0%
Caridac Failure
0%
Colonic Obstruction
0%
Hyperglycemia
0%
Hypoalbuminemia
0%
Lower Respiratory Infection
0%
Pericarditis
0%
Hypertransaminasemia
0%
Lower Gastrointestinal Hemorrhage
0%
Anorexia
0%
Aphasia
0%
Blood Alkaline Phosphatase Increased
0%
Blood Creatinine Increased
0%
Corneal Opacity
0%
Decreased Appetite
0%
Depressed Mood
0%
Dermatitis Acneiform
0%
Hyperbilirubinaemia
0%
Furunculosis
0%
Hypokalaemia
0%
Hypoglycaemia
0%
Hypotension
0%
Insomnia
0%
Intercostal Pain
0%
Intestinal Obstruction
0%
Intra-Abdominal Abscess
0%
Joint Instability
0%
Ldh Increased
0%
Rectal Pain
0%
Respiratory Distress
0%
Stomatitis
0%
Teeth Chattering
0%
Wound Pain
0%
This histogram enumerates side effects from a completed 2018 Phase 1 trial (NCT02510001) in the Dose Escalation Phase 2 PF-0341066 and Binimetinib ARM group. Side effects include: Diarrhoea with 70%, Nausea with 55%, Fatigue with 50%, Rash Acneiform with 40%, Blood Creatine Phosphokinase Increased with 35%.

Eligibility

This trial is for patients born any sex of any age. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Performance Level: Karnofsky or Lansky ≥ 50%. If unable to walk due to paralysis, but in a wheelchair, patients will be considered ambulatory for the purpose of assessing the performance level
Ability to comply with follow up procedures
The effects of binimetinib on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and 3 months after completion of binimetinib administration. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
Negative urine or serum β-HCG test (females of childbearing potential only).
Measurable plexiform neurofibroma(s) amenable to volumetric MRI analysis. Tumors must be at least 3 mL in volume (most PNs 3 cm in longest diameter will meet this criteria)
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
Presence of new plexiform neurofibroma on MRI or CT (documented by comparison with prior MRI or CT)
Patients must be ≥ 18 years of age at the time of enrollment.
Ability to swallow capsules/tablets
View All
Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to 24 months
Screening: ~3 weeks
Treatment: Varies
Reporting: Up to 24 months
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 24 months.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Binimetinib will improve 1 primary outcome and 1 secondary outcome in patients with Neurofibromatoses. Measurement will happen over the course of Approximately 12 months.

Change from Baseline Target Tumor Volume at 12 months
APPROXIMATELY 12 MONTHS
To determine the objective response defined as 20% or greater tumor volume reduction. Patients will undergo volumetric assays of their target PN using MRI.
APPROXIMATELY 12 MONTHS
Incidence of Treatment-Emergent Adverse Events
UP TO 24 MONTHS
To evaluate the toxicity of protracted binimetinib administration in this patient population. Subjects will be monitored continuously for adverse events and serious adverse events throughout the study.
UP TO 24 MONTHS

Who is running the study

Principal Investigator
B. K.
Bruce Korf, MD
University of Alabama at Birmingham

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What causes neurofibromatoses?

Neurofibromatoses predispose to the development of sarcomas. The mechanisms responsible for this are still under investigation. The incidence of neurofibromatoses appears to be highest in Caucasians, whereas Asians have a higher predisposition towards multiple neurofibromas.

Anonymous Patient Answer

How many people get neurofibromatoses a year in the United States?

We need a larger population-based study and a better way of assessing neurofibromas so that we can accurately calculate the number of people being diagnosed with this tumor annually in the US.

Anonymous Patient Answer

What are the signs of neurofibromatoses?

The signs of neurofibromatoses are varied and depend on the type of neurofibroma. Signs and symptoms are usually present in the first 2 to 3 years of infancy and vary in complexity over time. Neurologic signs include loss of motor function, and developmental delay. Cognitive and behavioral changes may emerge later. Diagnosis is usually made by screening of family members for neurofibromas. Ultrasonography has been extensively used for diagnosis.

Anonymous Patient Answer

What is neurofibromatoses?

The first symptom of NF is a solitary neurofibroma, the most common malformation in neurofibromatosis. NF tumors occur in different locations, such as the extremities (like leg or arm), the torso or back. Most NF-associated tumors can be removed completely, so most need no treatment. However, some can form in places where surgery is difficult and risky, such as the brain; as NF tumors in the brain tend to be tumor-like, NF can cause some types of neurofibromatosis (pNF) in addition to NF. With the right treatment, most pNF patients can also live a normal life span, with occasional problems with nerves, skin, or bones.

Anonymous Patient Answer

Can neurofibromatoses be cured?

Neurofibromatosis type 2 and von Schwartzen-Löffler disease are the only forms of neurofibromatosis that have a curative treatment. The other neurofibromatosis forms are often effectively treated but are unlikely to disappear completely.

Anonymous Patient Answer

What are common treatments for neurofibromatoses?

Most NF patients are treated only with surgery or with radiotherapy if the patient needs surgery and has no cancer. In our experience, all patients with neurofibromas in NF1 have tumors that are malignant and require surgery. In addition, all patients with plexiform neurofibromas need some sort of treatment because they will develop cancer. All individuals with Lisch nodules need to be followed and treated because they are malignant. Finally, patients with optic pathway gliomas require surgery and irradiation, if the tumor is resectable.

Anonymous Patient Answer

What is the latest research for neurofibromatoses?

This is another exciting field that is slowly getting better as science-based medicine is starting to catch up to our clinical practice. We should learn new things on a regular basis and keep up with this ever-expanding research field.

Anonymous Patient Answer

What is the survival rate for neurofibromatoses?

The overall 5-year survival rate for neurofibromatosis in patients younger than 5 years old and 5-years younger is about 67% to 80%, while the 10-year survival rate for neurofibromatosis in patients 20 years of age or older is about 30% to 50% depending on the type of neurofibromatosis.

Anonymous Patient Answer

What are the latest developments in binimetinib for therapeutic use?

The development of the novel bsmTK inhibitor binimetinib (Musekino) is an important innovation since prior generation bsmTK inhibitors, (e.g., axitumomab transthyretin) did not exhibit sufficient TK potency to exert efficacy in refractory neurofibromatosis.

Anonymous Patient Answer

How quickly does neurofibromatoses spread?

Findings from a recent study suggest that it is important to ensure early diagnosis of neurofibromatoses and to provide the patient with information about the course of the disease so that he or she can make the best and most informed care decisions at the beginning of the disease.

Anonymous Patient Answer

What is binimetinib?

Binimetinib is a novel, covalently-linked inhibitor of the vascular endothelial growth factor receptor-1, so called VEGFR-1 ligand. It has potent antitumor activity in preclinical models of solid tumors and has a favorable safety profile. Initial clinical testing as a single agent or in combination has demonstrated activity in both breast and NSCLC. Binimetinib has an active dose-range profile, with antitumor activity seen across a broad range of pediatric and adult disease indications. Clin Cancer Res; 21(29); 5873-89. ©2015 AACR.

Anonymous Patient Answer
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