Trial Outcomes & Findings for Bevacizumab and Lapatinib in Children With Recurrent or Refractory Ependymoma (NCT NCT00883688)

Last Updated: 2020-08-26

Results Overview

Objective response rate defined as number of participants out of total participants with complete response plus partial response (CR+PR) sustained for at least four weeks. Complete Response (CR) - Complete disappearance on magnetic resonance imaging (MRI) of all enhancing tumor and mass effect, on a stable or decreasing dose of corticosteroids (or receiving only adrenal replacement doses), accompanied by a stable or improving neurologic examination and must be sustained for at least 4 weeks. If cerebrospinal fluid (CSF) for presence of disease was positive it must become negative. Partial Response (PR) - Greater than or equal to 50% reduction in tumor size by bi-dimensional measurement on a stable or decreasing dose of corticosteroids, accompanied by stable or improving neurological examination and must be sustained for at least 4 weeks.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

24 participants

Primary outcome timeframe

4 weeks following treatment, repeat assessments up to one year.

Results posted on

2020-08-26

Participant Flow

Recruitment Period: July 23, 2009 to April 3, 2013. All recruitment done in medical clinics.

Participant milestones

Participant milestones
Measure	Bevacizumab + Lapatinib Bevacizumab 10 mg/kg intravenous over 90 minutes for first injection (30-60 minutes for subsequent doses) every 2 weeks while on study (2 times during each 4-week "study cycle"). Lapatinib 700 mg/m\^2/dose given orally 2 times each day.
Overall Study STARTED	24
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Bevacizumab + Lapatinib Bevacizumab 10 mg/kg intravenous over 90 minutes for first injection (30-60 minutes for subsequent doses) every 2 weeks while on study (2 times during each 4-week "study cycle"). Lapatinib 700 mg/m\^2/dose given orally 2 times each day.
Overall Study Ineligible	2

Baseline Characteristics

Bevacizumab and Lapatinib in Children With Recurrent or Refractory Ependymoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bevacizumab + Lapatinib n=24 Participants Bevacizumab 10 mg/kg intravenous over 90 minutes for first injection (30-60 minutes for subsequent doses) every 2 weeks while on study (2 times during each 4-week "study cycle"). Lapatinib 700 mg/m\^2/dose given orally 2 times each day.
Age, Categorical <=18 years	24 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	17 Participants n=5 Participants
Region of Enrollment United States	24 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 weeks following treatment, repeat assessments up to one year.

Population: Of the total, 22 participants were eligible and only 20 evaluable for response.

Outcome measures

Outcome measures
Measure	Bevacizumab + Lapatinib n=20 Participants Bevacizumab 10 mg/kg intravenous over 90 minutes for first injection (30-60 minutes for subsequent doses) every 2 weeks while on study (2 times during each 4-week "study cycle"). Lapatinib 700 mg/m\^2/dose given orally 2 times each day.
Objective Response Rate	0 Percentage of Participants

Adverse Events

Bevacizumab + Lapatinib

Serious events: 11 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Bevacizumab + Lapatinib n=24 participants at risk Bevacizumab 10 mg/kg intravenous over 90 minutes for first injection (30-60 minutes for subsequent doses) every 2 weeks while on study (2 times during each 4-week "study cycle"). Lapatinib 700 mg/m\^2/dose given orally 2 times each day.
Investigations Increase serum glutamic pyruvic transaminase (ALT, SGPT)	12.5% 3/24 • Number of events 4 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Apnea	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Aspiration	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Increase serum glutamic oxaloacetic transaminase (AST, SGOT)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Increase creatine phosphokinase (CPK )	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Skin and subcutaneous tissue disorders Pustular Rash/Scalp	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Dyspnea	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Blood and lymphatic system disorders Febrile neutropenia	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations High gamma-Glutamyl transpeptidase (GGT)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Hemorrhage, Central Nervous System	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Hemorrhage: Respiratory tract NOS	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Hypoxia	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils : Abdomen NOS	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Cardiac disorders Left ventricular systolic dysfunction	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Cranial Neuropathy CN IX Motor	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Hepatobiliary disorders Gastrointestional Obstruction: Gallbladder	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Abdomen Pain	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Extremity Pain, Limb	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Muscle Pain	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Pancreatitis	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Somnolence/depressed level of consciousness	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Speech impairment	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).

Other adverse events

Other adverse events
Measure	Bevacizumab + Lapatinib n=24 participants at risk Bevacizumab 10 mg/kg intravenous over 90 minutes for first injection (30-60 minutes for subsequent doses) every 2 weeks while on study (2 times during each 4-week "study cycle"). Lapatinib 700 mg/m\^2/dose given orally 2 times each day.
Metabolism and nutrition disorders Albumin, serum-low (hypoalbuminemia)	12.5% 3/24 • Number of events 9 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Alkaline phosphatase	8.3% 2/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Immune system disorders Allergy - Bumpy tongue	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations ALT, SGPT (serum glutamic pyruvic transaminase)	29.2% 7/24 • Number of events 16 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Anorexia	29.2% 7/24 • Number of events 9 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Apnea	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Aspiration	4.2% 1/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations AST, SGOT(serum glutamic oxaloacetic transaminase)	29.2% 7/24 • Number of events 14 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Ataxia (incoordination)	50.0% 12/24 • Number of events 14 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Ear and labyrinth disorders Hearing loss	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Bicarbonate, serum-low	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Bilirubin (hyperbilirubinemia)	16.7% 4/24 • Number of events 5 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Bronchospasm, wheezing	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Calcium, serum-high (hypercalcemia)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Calcium, serum-low (hypocalcemia)	12.5% 3/24 • Number of events 13 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Coagulation: Activated partial thromboplastin time (aPTT or APTT)	4.2% 1/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Cognitive disturbance	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Colitis	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Constipation	25.0% 6/24 • Number of events 9 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
General disorders Constitutional Symptoms	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Cough	8.3% 2/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations CPK (creatine phosphokinase) increase	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Creatinine Increases	4.2% 1/24 • Number of events 4 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Endocrine disorders Cushingoid appearance (e.g., moon face, buffalo hump, centripetal obesity, cutaneous striae)	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Dehydration	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Skin and subcutaneous tissue disorders Sores & pustules	20.8% 5/24 • Number of events 5 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Diarrhea	75.0% 18/24 • Number of events 44 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Dizziness	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Dry mouth/salivary gland (xerostomia)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Skin and subcutaneous tissue disorders Dry skin	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Dysphagia (difficulty swallowing)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
General disorders Edema: limb	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
General disorders Extremity-lower (gait/walking)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
General disorders Fatigue (asthenia, lethargy, malaise)	58.3% 14/24 • Number of events 17 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Blood and lymphatic system disorders Febrile neutropenia	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
General disorders Fever	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Flatulence	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Gastrointestinal - Other (Blood in stool, gas)	4.2% 1/24 • Number of events 4 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations GGT (gamma-Glutamyl transpeptidase)	4.2% 1/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Glucose, serum-high (hyperglycemia)	4.2% 1/24 • Number of events 4 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Glucose, serum-low (hypoglycemia)	4.2% 1/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Skin and subcutaneous tissue disorders Hair loss/alopecia (scalp or body)	16.7% 4/24 • Number of events 4 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Ear and labyrinth disorders Hearing: patients with/without baseline audiogram and enrolled in a monitoring program	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Heartburn/dyspepsia	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Hemoglobin Change	12.5% 3/24 • Number of events 7 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Hemorrhage, CNS	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Hemorrhage, pulmonary/upper respiratory : Nose	16.7% 4/24 • Number of events 9 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Hemorrhage, pulmonary/upper respiratory : Respiratory tract NOS	4.2% 1/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Vascular disorders Hypertension	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Vascular disorders Hypotension	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Hypoxia	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Renal and urinary disorders Incontinence, urinary	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Infections and infestations Infection - Other (Specify, Upper Respiratory)	8.3% 2/24 • Number of events 4 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Infections and infestations Infection with normal ANC, Grade 1/2 neutrophils	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils : Abdomen NOS	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Infections and infestations Infection normal ANC or Grade 1 or 2 neutrophils: Foreign body	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils : Trachea	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils : Urinary tract NOS	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils : Vagina	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Infections and infestations Infection with unknown ANC : Lung (pneumonia)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Infections and infestations Infection with unknown ANC : Urinary tract NOS	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations INR (International Normalized Ratio of prothrombin time)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Psychiatric disorders Insomnia	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Laryngeal nerve dysfunction	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Cardiac disorders Left ventricular systolic dysfunction	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Lumbar spine-range of motion	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Vascular disorders Lymphatics - Other (Specify, swollen hands)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Vascular disorders Lymphopenia	12.5% 3/24 • Number of events 6 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Magnesium, serum-high (hypermagnesemia)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Magnesium, serum-low (hypomagnesemia)	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Metabolic/Laboratory - Other (Specify, phosphorus increase)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Psychiatric disorders Mood alteration : Anxiety	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Psychiatric disorders Mood alteration : Depression	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Mucositis/stomatitis (clinical exam) : Oral cavity	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Mucositis/stomatitis (functional/symptomatic) : Oral cavity	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Muscle weakness, generalized or specific area (not due to neuropathy) : Extremity-lower	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Muscle weakness, generalized or specific area (not due to neuropathy) : Left-sided	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Muscle weakness, generalized or specific area (not due to neuropathy) : Right-sided	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Weakness, limp	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Nausea	29.2% 7/24 • Number of events 9 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Central Nervous System (CNS) affectation	20.8% 5/24 • Number of events 5 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Cranial Neuropathy: CN IX Motor-pharynx	16.7% 4/24 • Number of events 4 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Cranial Neuropathy: CN VI Lateral deviation of eye	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Cranial Neuropathy: CN VII Motor-face	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Cranial Neuropathy: CN X Motor-palate	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Cranial Neuropathy: CN XII Motor-tongue	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Motor Neuropathy	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Sensory Neuropathy	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Nystagmus	25.0% 6/24 • Number of events 7 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Obesity	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Hepatobiliary disorders Obstruction, GI : Gallbladder	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Eye disorders Ptosis, dry eyes	12.5% 3/24 • Number of events 4 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Eye disorders Ophthalmoplegia/diplopia (double vision)	16.7% 4/24 • Number of events 4 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Ear and labyrinth disorders Otitis, external ear (non-infectious)	4.2% 1/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Knee Pain	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Pain - Neck	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Pain - Thigh	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Pain : Abdomen NOS	29.2% 7/24 • Number of events 7 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Pain : Back	8.3% 2/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Ear and labyrinth disorders Pain : External ear	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Pain : Extremity-limb	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Pain : Head/headache	29.2% 7/24 • Number of events 12 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Musculoskeletal and connective tissue disorders Pain : Muscle	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Pain : Oral cavity	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Pain : Stomach	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Pain : Throat/pharynx/larynx	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Pancreatitis	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Phosphate, serum-low (hypophosphatemia)	25.0% 6/24 • Number of events 8 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Blood and lymphatic system disorders Platelets low	4.2% 1/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Potassium, serum-high (hyperkalemia)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Potassium, serum-low (hypokalemia)	20.8% 5/24 • Number of events 9 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Prolonged intubation after pulmonary resection (>24 hrs after surgery)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Renal and urinary disorders Proteinuria	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Skin and subcutaneous tissue disorders Pruritus/itching	8.3% 2/24 • Number of events 5 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Partial Thromboplastin Time (PTT) outside normal	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Respiratory, thoracic and mediastinal disorders Respiratory arrest/choking, congestion, tachypnea	16.7% 4/24 • Number of events 4 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Skin and subcutaneous tissue disorders Rash/desquamation	50.0% 12/24 • Number of events 23 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Skin and subcutaneous tissue disorders Rash: acne/acneiform	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Renal and urinary disorders Renal failure	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Renal and urinary disorders Renal/Genitourinary - Other (urination issues)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Seizure	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Sodium, serum-high (hypernatremia)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Sodium, serum-low (hyponatremia)	8.3% 2/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Somnolence/depressed level of consciousness	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Speech impairment (e.g., dysphasia or aphasia)	12.5% 3/24 • Number of events 3 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Skin and subcutaneous tissue disorders Striae	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Endocrine disorders Thyroid function, low (hypothyroidism)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Ear and labyrinth disorders Tinnitus	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Nervous system disorders Tremor	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Metabolism and nutrition disorders Triglyceride, serum-high (hypertriglyceridemia)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Renal and urinary disorders Urinary retention (including neurogenic bladder)	8.3% 2/24 • Number of events 2 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Cardiac disorders Valvular heart disease	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Eye disorders Vision-blurred vision	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Immune system disorders Voice changes/dysarthria (e.g., hoarseness, loss or alteration in voice, laryngitis)	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Gastrointestinal disorders Vomiting	33.3% 8/24 • Number of events 10 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).
Investigations Weight loss	4.2% 1/24 • Number of events 1 • Adverse event collection through 8 week treatment and may continue for up to 26 courses (2 years).

Additional Information

MD Anderson Multicenter coordinating for Collaborative Ependymoma Research Network Study (CERN)

The University of Texas (UT) MD Anderson Cancer Center

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place