CLINICAL TRIAL

Brigatinib for Ependymoma

High Risk
Metastatic
Stage I
Recruiting · Any Age · All Sexes · Los Angeles, CA

Innovative Trial for Understanding the Impact of Targeted Therapies in NF2

See full description

About the trial for Ependymoma

Eligible Conditions
Neuroma, Acoustic · Neurofibromatoses · Neurilemmoma · Neurofibromatosis 2 · Vestibular Schwannomas · Non-vestibular Schwannoma · Meningioma · Ependymoma · Neurofibroma · Neurofibromatosis Type 2 · Neurofibromatosis 1

Treatment Groups

This trial involves 2 different treatments. Brigatinib 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.
Brigatinib
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
Brigatinib
FDA approved

Side Effect Profile for Randomized Phase: Brigatinib 90 mg QD/180 QD

Randomized Phase: Brigatinib 90 mg QD/180 QD
Show all side effects
57%
Diarrhoea
50%
Blood Creatine Phosphokinase Increased
36%
Cough
32%
Hypertension
32%
Nausea
26%
Back Pain
26%
Aspartate Aminotransferase Increased
23%
Headache
23%
Alanine Aminotransferase Increased
23%
Dyspnoea
23%
Lipase Increased
22%
Vomiting
21%
Pruritus
21%
Fatigue
20%
Arthralgia
19%
Constipation
18%
Rash
17%
Dizziness
15%
Muscle Spasms
15%
Pyrexia
13%
Asthenia
13%
Upper Respiratory Tract Infection
13%
Blood Alkaline Phosphatase Increased
13%
Abdominal Pain
12%
Decreased Appetite
11%
Dyspepsia
11%
Musculoskeletal Pain
10%
Insomnia
10%
Oropharyngeal Pain
10%
Myalgia
10%
Blood Cholesterol Increased
10%
Dermatitis Acneiform
10%
Oedema Peripheral
9%
Paraesthesia
9%
Stomatitis
9%
Musculoskeletal Chest Pain
9%
Productive Cough
9%
Nasopharyngitis
9%
Anaemia
8%
Abdominal Pain Upper
8%
Non-Cardiac Chest Pain
7%
Urinary Tract Infection
7%
Pneumonia
7%
Bradycardia
7%
Epistaxis
7%
Pain In Extremity
7%
Rash Erythematous
6%
Electrocardiogram Qt Prolonged
6%
Dry Mouth
6%
Dry Skin
6%
Eczema
6%
Dysphonia
6%
Blood Creatinine Increased
6%
Rash Maculo-Papular
5%
Respiratory Tract Infection
5%
Vision Blurred
5%
Hypophosphataemia
5%
Malaise
5%
Hypercholesterolaemia
5%
Sinus Bradycardia
5%
Hyperglycaemia
5%
Hypokalaemia
5%
Rhinorrhoea
5%
Blood Lactate Dehydrogenase Increased
4%
Dysgeusia
4%
Depression
4%
Gamma-Glutamyltransferase Increased
4%
Neoplasm Progression
4%
Influenza Like Illness
4%
Peripheral Swelling
2%
Taste Disorder
2%
Hypocalcaemia
2%
Pleural Effusion
2%
Hypotension
2%
Neutrophil Count Decreased
2%
Pulmonary Embolism
2%
Interstitial Lung Disease
2%
Hypoaesthesia
1%
Oesophageal Obstruction
1%
Hypoalbuminaemia
1%
Dysphagia
1%
Appendicitis
1%
Pneumonitis
1%
Acute Myocardial Infarction
1%
Vocal Cord Paralysis
1%
Toxicity To Various Agents
1%
Gout
1%
C-Reactive Protein Increased
1%
Hepatocellular Injury
1%
Hypoglycaemia
1%
Cancer Pain
1%
Muscular Weakness
1%
Gastrooesophageal Reflux Disease
1%
Acute Kidney Injury
1%
Transaminases Abnormal
1%
Femoral Neck Fracture
1%
Hyperkalaemia
1%
Pericardial Effusion
1%
Confusional State
1%
Multiple Organ Dysfunction Syndrome
1%
Ovarian Cancer Stage I
1%
Delirium
1%
Visual Impairment
1%
Gastric Haemorrhage
1%
Seizure
1%
Fall
1%
Ligament Rupture
1%
Photopsia
1%
Malignant Pleural Effusion
1%
Viral Infection
1%
Lower Respiratory Tract Infection
1%
Diffuse Large B-Cell Lymphoma
1%
Gastroenteritis
1%
Lung Adenocarcinoma
1%
Neutropenia
1%
Partial Seizures
1%
Cognitive Disorder
1%
Peripheral Sensory Neuropathy
1%
Invasive Breast Carcinoma
1%
Lung Neoplasm Malignant
1%
Memory Impairment
1%
Cardiac Tamponade
1%
Disseminated Intravascular Coagulation
1%
Syncope
1%
Pulmonary Oedema
1%
Metastases To Meninges
1%
Metastases To Central Nervous System
1%
Balance Disorder
1%
Squamous Cell Carcinoma Of Skin
1%
Cerebrovascular Accident
1%
Encephalopathy
1%
Pneumothorax
1%
Dysarthria
1%
Neutropenic Colitis
1%
Respiratory Distress
1%
Atrial Fibrillation
1%
Pneumonia Aspiration
1%
Cholecystitis
1%
Arrhythmia
1%
Angina Pectoris
1%
Inguinal Hernia
1%
Bile Duct Stone
1%
Cholestasis
1%
Mucosal Inflammation
1%
Sudden Death
0%
Blood Insulin Increased
0%
Aphasia
0%
Tibia Fracture
0%
Amylase Increased
0%
Intraventricular Haemorrhage
0%
Drug-Induced Liver Injury
0%
Palpitations
0%
Respiratory Arrest
0%
Deep Vein Thrombosis
0%
Cardiac Failure
0%
Covid-19
0%
Gastroenteritis Viral
0%
Prostate Cancer
0%
Intestinal Obstruction
0%
Depressed Level Of Consciousness
0%
Atypical Pneumonia
0%
Herpes Zoster
0%
Respiratory Failure
0%
Intracranial Tumour Haemorrhage
0%
Ascites
0%
Brain Herniation
0%
Abdominal Pain Lower
0%
Large Intestine Perforation
0%
Bone Pain
0%
Urinary Retention
0%
Oesophagitis Ulcerative
0%
Rotator Cuff Syndrome
0%
General Physical Health Deterioration
0%
Disorientation
0%
Salmonella Sepsis
0%
Cytomegalovirus Oesophagitis
0%
Cellulitis
0%
Listeriosis
0%
Pleural Infection
0%
Vertigo Positional
0%
Septic Shock
0%
Sinusitis Fungal
0%
Tumour Haemorrhage
0%
Hodgkins Disease
0%
Ischaemic Stroke
0%
Anaemia Macrocytic
0%
Central Nervous System Lesion
0%
Acute Respiratory Failure
0%
Myocardial Infarction
0%
Arthritis
0%
Menorrhagia
0%
Platelet Count Decreased
0%
Spinal Fracture
0%
Haematuria
0%
Hemiparesis
0%
Generalised Tonic-Clonic Seizure
0%
Performance Status Decreased
0%
Pyelonephritis Acute
0%
Anxiety
0%
Haemoptysis
Diarrhoea
57%
Blood Creatine Phosphokinase Increased
50%
Cough
36%
Hypertension
32%
Nausea
32%
Back Pain
26%
Aspartate Aminotransferase Increased
26%
Headache
23%
Alanine Aminotransferase Increased
23%
Dyspnoea
23%
Lipase Increased
23%
Vomiting
22%
Pruritus
21%
Fatigue
21%
Arthralgia
20%
Constipation
19%
Rash
18%
Dizziness
17%
Muscle Spasms
15%
Pyrexia
15%
Asthenia
13%
Upper Respiratory Tract Infection
13%
Blood Alkaline Phosphatase Increased
13%
Abdominal Pain
13%
Decreased Appetite
12%
Dyspepsia
11%
Musculoskeletal Pain
11%
Insomnia
10%
Oropharyngeal Pain
10%
Myalgia
10%
Blood Cholesterol Increased
10%
Dermatitis Acneiform
10%
Oedema Peripheral
10%
Paraesthesia
9%
Stomatitis
9%
Musculoskeletal Chest Pain
9%
Productive Cough
9%
Nasopharyngitis
9%
Anaemia
9%
Abdominal Pain Upper
8%
Non-Cardiac Chest Pain
8%
Urinary Tract Infection
7%
Pneumonia
7%
Bradycardia
7%
Epistaxis
7%
Pain In Extremity
7%
Rash Erythematous
7%
Electrocardiogram Qt Prolonged
6%
Dry Mouth
6%
Dry Skin
6%
Eczema
6%
Dysphonia
6%
Blood Creatinine Increased
6%
Rash Maculo-Papular
6%
Respiratory Tract Infection
5%
Vision Blurred
5%
Hypophosphataemia
5%
Malaise
5%
Hypercholesterolaemia
5%
Sinus Bradycardia
5%
Hyperglycaemia
5%
Hypokalaemia
5%
Rhinorrhoea
5%
Blood Lactate Dehydrogenase Increased
5%
Dysgeusia
4%
Depression
4%
Gamma-Glutamyltransferase Increased
4%
Neoplasm Progression
4%
Influenza Like Illness
4%
Peripheral Swelling
4%
Taste Disorder
2%
Hypocalcaemia
2%
Pleural Effusion
2%
Hypotension
2%
Neutrophil Count Decreased
2%
Pulmonary Embolism
2%
Interstitial Lung Disease
2%
Hypoaesthesia
2%
Oesophageal Obstruction
1%
Hypoalbuminaemia
1%
Dysphagia
1%
Appendicitis
1%
Pneumonitis
1%
Acute Myocardial Infarction
1%
Vocal Cord Paralysis
1%
Toxicity To Various Agents
1%
Gout
1%
C-Reactive Protein Increased
1%
Hepatocellular Injury
1%
Hypoglycaemia
1%
Cancer Pain
1%
Muscular Weakness
1%
Gastrooesophageal Reflux Disease
1%
Acute Kidney Injury
1%
Transaminases Abnormal
1%
Femoral Neck Fracture
1%
Hyperkalaemia
1%
Pericardial Effusion
1%
Confusional State
1%
Multiple Organ Dysfunction Syndrome
1%
Ovarian Cancer Stage I
1%
Delirium
1%
Visual Impairment
1%
Gastric Haemorrhage
1%
Seizure
1%
Fall
1%
Ligament Rupture
1%
Photopsia
1%
Malignant Pleural Effusion
1%
Viral Infection
1%
Lower Respiratory Tract Infection
1%
Diffuse Large B-Cell Lymphoma
1%
Gastroenteritis
1%
Lung Adenocarcinoma
1%
Neutropenia
1%
Partial Seizures
1%
Cognitive Disorder
1%
Peripheral Sensory Neuropathy
1%
Invasive Breast Carcinoma
1%
Lung Neoplasm Malignant
1%
Memory Impairment
1%
Cardiac Tamponade
1%
Disseminated Intravascular Coagulation
1%
Syncope
1%
Pulmonary Oedema
1%
Metastases To Meninges
1%
Metastases To Central Nervous System
1%
Balance Disorder
1%
Squamous Cell Carcinoma Of Skin
1%
Cerebrovascular Accident
1%
Encephalopathy
1%
Pneumothorax
1%
Dysarthria
1%
Neutropenic Colitis
1%
Respiratory Distress
1%
Atrial Fibrillation
1%
Pneumonia Aspiration
1%
Cholecystitis
1%
Arrhythmia
1%
Angina Pectoris
1%
Inguinal Hernia
1%
Bile Duct Stone
1%
Cholestasis
1%
Mucosal Inflammation
1%
Sudden Death
1%
Blood Insulin Increased
0%
Aphasia
0%
Tibia Fracture
0%
Amylase Increased
0%
Intraventricular Haemorrhage
0%
Drug-Induced Liver Injury
0%
Palpitations
0%
Respiratory Arrest
0%
Deep Vein Thrombosis
0%
Cardiac Failure
0%
Covid-19
0%
Gastroenteritis Viral
0%
Prostate Cancer
0%
Intestinal Obstruction
0%
Depressed Level Of Consciousness
0%
Atypical Pneumonia
0%
Herpes Zoster
0%
Respiratory Failure
0%
Intracranial Tumour Haemorrhage
0%
Ascites
0%
Brain Herniation
0%
Abdominal Pain Lower
0%
Large Intestine Perforation
0%
Bone Pain
0%
Urinary Retention
0%
Oesophagitis Ulcerative
0%
Rotator Cuff Syndrome
0%
General Physical Health Deterioration
0%
Disorientation
0%
Salmonella Sepsis
0%
Cytomegalovirus Oesophagitis
0%
Cellulitis
0%
Listeriosis
0%
Pleural Infection
0%
Vertigo Positional
0%
Septic Shock
0%
Sinusitis Fungal
0%
Tumour Haemorrhage
0%
Hodgkins Disease
0%
Ischaemic Stroke
0%
Anaemia Macrocytic
0%
Central Nervous System Lesion
0%
Acute Respiratory Failure
0%
Myocardial Infarction
0%
Arthritis
0%
Menorrhagia
0%
Platelet Count Decreased
0%
Spinal Fracture
0%
Haematuria
0%
Hemiparesis
0%
Generalised Tonic-Clonic Seizure
0%
Performance Status Decreased
0%
Pyelonephritis Acute
0%
Anxiety
0%
Haemoptysis
0%
This histogram enumerates side effects from a completed 2021 Phase 3 trial (NCT02737501) in the Randomized Phase: Brigatinib 90 mg QD/180 QD ARM group. Side effects include: Diarrhoea with 57%, Blood Creatine Phosphokinase Increased with 50%, Cough with 36%, Hypertension with 32%, Nausea with 32%.

Eligibility

This trial is for patients born any sex of any age. There are 6 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
The person has a bilateral vestibular schwannoma, which is a tumor on the 8th cranial nerve, OR the person has a first-degree relative with NF2 and EITHER an unilateral eighth nerve mass OR two of the following: neurofibroma, meningioma, glioma, schwannoma, juvenile posterior subcapsular lenticular opacity. show original
A first-degree relative with NF2 and Proof of either an unilateral eighth nerve mass or two of the following: neurofibroma, meningioma, glioma, or schwannoma, juvenile posterior subcapsular lenticular opacity, or unilateral vestibular schwannoma show original
The patient is not able to have surgery due to either the patient's refusal or the high risk for surgical complications show original
The text is saying that an increase in size of a tumor by at least 2mm is required to be considered a target lesion. show original
Ependymoma lesions can be measured in a linear way. show original
The person must have a established relationship with a primary care physician and provide contact information. show original
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
Similar Trials

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 2 years
Screening: ~3 weeks
Treatment: Varies
Reporting: 2 years
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 2 years.
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 Brigatinib will improve 1 primary outcome and 1 secondary outcome in patients with Ependymoma. Measurement will happen over the course of 2 years.

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability
2 YEARS
Number of Participants with Treatment Emergent Adverse Events as Assessed CTCAE v5.0
2 YEARS
Radiographic response rate (for each drug substudy)
2 YEARS
Radiographic response rates in target tumors according to tumor-associated criteria: VS, non-VS, and meningiomas: Dombi criteria (2013) Ependymomas: RECIST 1.12
2 YEARS

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 is ependymoma?

This very rare and challenging benign cortical brain tumour typically involves a single site in the posterior fossa of the brain and, if surgically removed, carries a very poor prognosis with over 50% of children dying in the year after diagnosis. In this situation, chemotherapy followed by radiation is often recommended.

Anonymous Patient Answer

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

Many cases of ependymomas are occurring in children and teens, especially in the first two decades of life. This information would help the surgeon and the child and adolescent patient and their parents discuss treatment options. Because fewer than 10% of all cases of ependymomas are diagnosed before 15 years of age, ependymoma awareness campaigns need to extend beyond children of all ages to younger teens.

Anonymous Patient Answer

What are the chances of developing ependymoma?

There does not currently seem to be any significant difference between the age of onset of ependymomas and their tumor location. However, the occurrence of these ependymomas in adults raises the possibility of this tumor's having a genetic cause - which is not well understood.

Anonymous Patient Answer

What causes ependymoma?

The usual causes of ependymoma include genetic disorders, prenatal exposure to tobacco smoke and prenatal and early-childhood environmental factors. The high rate of recurrence observed previously in this study suggests that more aggressive surgical excision may be advisable in patients with this condition.

Anonymous Patient Answer

What are common treatments for ependymoma?

There is a high incidence of relapse after total surgical resection. The use of radiotherapy or chemotherapy must be carefully considered, depending on disease risk and patient comorbidities as well as disease aggressiveness. The role of high-dose radiation therapy, in particular radiation therapy with proton beams, should be explored further.

Anonymous Patient Answer

Can ependymoma be cured?

Patients who have not undergone initial resection of ependymoma have a very high risk of recurrence or of the development of second tumors, and their prognoses are generally poor.

Anonymous Patient Answer

What are the signs of ependymoma?

When people are asked if they have experienced any symptoms, some say yes. Pain or stiffness in either or both legs is another symptom of ependymoma. Children more often have tumour in one or more regions of the body than adults. Increased intracranial pressure and ocular signs, such as decreased vision and eye pain, are symptoms of tumour in the optic nerve.

Anonymous Patient Answer

Does ependymoma run in families?

Familial ependymomas occur rarely, with familial cases representing 1.0% of this group. Of the 17 familial cases studied in this series, 14 tumors occurred in 1 family member. Familial cases are most likely caused by somatic mutations or by germ line mutations. Further study is needed to determine the underlying causes of familial cases of ependymomas.

Anonymous Patient Answer

How does brigatinib work?

Brigatinib exert anti-proliferative effects in vitro and in vivo by inhibiting PI3K/AKT, MAPK/ERK, and STAT3 signaling and by blocking the production of angiogenic proteins from ECs.

Anonymous Patient Answer

What is the primary cause of ependymoma?

In patients with a surgically cured ependymoma, postoperative radiotherapy and prolonged follow up should be recommended if the relapse occurs, especially with tumor grade IV.

Anonymous Patient Answer

Have there been other clinical trials involving brigatinib?

brigatinib was the first drug approved by regulatory agencies for adults with relapsed or refractory Hodgkin's lymphoma or relapsing indolent lymphoma. However, clinical trials involving brigatinib that included post-hoc analysis of survival data are no longer being published in peer-reviewed journals. Although the efficacy and safety of brigatinib has been demonstrated in patients with relapsed or refractory Hodgkin's lymphoma and indolent lymphoma, patients who have received brigatinib have discontinued treatment citing safety concerns, exacerbation of CLL, and possible worsening of myelofibrosis. [Find more clinical trials at clinicalTrials.gov (e.g.

Anonymous Patient Answer

What does brigatinib usually treat?

Brigatinib has shown significant single agent activity in phase I trials in patients whose tumors carried EGFR exon 19 deletions. It has demonstrated clear dose-dependent efficacy at this dose in patients with tumors carrying T790M mutations. Results from a recent clinical trial support continued investigations of brigatinib in combination with other agents.

Anonymous Patient Answer
See if you qualify for this trial
Get access to this novel treatment for Ependymoma by sharing your contact details with the study coordinator.