Symptoms and General Pathology

All Conditions

Neoplasms

Rare Diseases

Recurrence

Ependymoma

Disease Attributes

Glioma

Neoplasms, Neuroepithelial

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

Amino Acids

Etoposide

Etoposide phosphate

Erlotinib Hydrochloride

Enzyme Inhibitors

Tyrosine Kinase Inhibitors

Protein Kinase Inhibitors

Tyrosine

Erlotinib was administered orally at a dose of 85 mg/m\^2 per day continuously until either progression, death, patient request or investigator decision to discontinue study drug or intolerable toxicity.

Etoposide 50 mg/m\^2 per day was administered orally for 21 days followed by a 7-day rest period until either progression, death, patient request or investigator decision to discontinue study drug or intolerable toxicity.

erlotinib

etoposide

Medical Monitor

This is a phase 2 study to evaluate the efficacy of single-agent erlotinib versus oral etoposide in patients with recurrent or refractory pediatric ependymoma.

This is a phase 2 study involving a 1:1 randomization of 40 patients with recurrent or refractory pediatric ependymoma who will receive either erlotinib or oral etoposide.

Erlotinib Versus Oral Etoposide in Patients With Recurrent or Refractory Pediatric Ependymoma

A Randomized, Phase 2 Study of Single-agent Erlotinib Versus Oral Etoposide in Patients With Recurrent or Refractory Pediatric Ependymoma

Access to anonymized individual participant level data will not be provided for this trial as it meets one or more of the exceptions described on www.clinicalstudydatarequest.com under "Sponsor Specific Details for Astellas."

Objective response is defined as a best overall response of complete response (CR) or partial response (PR), evaluated using modified International Society of Pediatric Oncology Brain, Tumor Subcommittee for the Reporting of Trials criteria. Response was confirmed at least 28 days after the first assessment where the response criteria were met. Response was assessed by magnetic resonance imaging (MRI) every 8 weeks.

CR:

* Complete disappearance of all enhancing tumor and mass effect
* On a stable or decreasing dose of corticosteroids (or receiving only adrenal replacement doses)
* Stable or improving neurologic examination sustained for ≥ 4 weeks
* If cerebral spinal fluid (CSF) evaluation was positive, it must become negative (confirmed at least 2 times at consecutive samplings).

PR:

* ≥ 50% reduction in tumor size by bi-dimensional measurement
* On a stable or decreasing dose of corticosteroids
* Stable or improving neurologic examination sustained for ≥ 4 weeks.

Duration of response (complete or partial response \[CR/PR\]) was defined as the time from the date of the first documented response (CR/PR) to the first documented progression or death due to underlying cancer. If a participant had not progressed or died, the duration of overall response was censored at the date of last adequate disease assessment. Duration of response was only defined for participants whose best overall response was CR or PR.

Progression was defined as a worsening of neurologic status that could not be explained by other causes, a \> 25% increase in tumor size, the appearance of new lesions or CSF positivity, or increasing doses of corticosteroids required to maintain stable status.

Participants with a best overall response of minor response (MR), defined as:

* ≥ 25% to \< 50% reduction in tumor size by bi-dimensional measurement
* On a stable or decreasing dose of corticosteroids
* Stable or improving neurologic examination sustained for ≥ 4 weeks.

Disease control is a best overall response of CR or PR or MR or Stable disease (SD).

CR:

* Complete disappearance of all enhancing tumor and mass effect
* On a stable or decreasing dose of corticosteroids
* Stable or improving neurologic examination sustained for ≥ 4 weeks
* If CSF evaluation was positive, it must become negative (confirmed at least 2 times consecutively).

PR:

* ≥ 50% reduction in tumor size by bi-dimensional measurement
* On a stable or decreasing dose of corticosteroids
* Stable or improving neurologic examination sustained for ≥ 4 weeks.

MR:

* ≥ 25% to \< 50% reduction in tumor size by bi-dimensional measurement
* On a stable or decreasing dose of corticosteroids
* Stable or improving neurologic examination sustained for ≥ 4 weeks.

SD:

* Neurologic examination is at least stable
* Maintenance corticosteroid dose is not increased
* MRI meets neither the criteria for minor response nor for progressive disease
* Sustained for ≥ 8 weeks.

Progression-free survival was defined as the time from randomization to disease progression based on central nervous system (CNS)-specific evaluation criteria as assessed by the investigator or death due to any cause, whichever occurs first.

Participants did not progress or die before the data cutoff date for analysis were censored at the date of last disease assessment (including both radiologic assessment and neurologic assessment) where non-progression was documented. If a participant received any further anticancer therapy without prior documentation of disease progression, the participant was censored at the date of last disease assessment before starting new anti-cancer treatment. Participants were also censored at the date of last disease assessment with no documented progression if patients discontinued treatment for undocumented progression, toxicity or other reason before the data cutoff date for analysis.

Prolonged stable disease (SD) was defined as SD with a duration of at least 16 weeks. The percentage of participants with prolonged SD was defined as participants who achieved a best overall response of CR or PR or MR or SD, and did not progress within 16 weeks from randomization. Progression was defined as a worsening of neurologic status that could not be explained by other causes, a \> 25% increase in tumor size, the appearance of new lesions or CSF positivity, or increasing doses of corticosteroids required to maintain stable status.

Duration of stable disease (SD; defined as participants with an overall best response of complete, partial or minor response or stable disease) was defined as the time from the date of randomization to the first documented progression or death due to underlying cancer. If a participant had not progressed or died, the duration of SD was censored at the date of last adequate disease assessment. Duration of SD was only defined for participants whose best overall response was CR or PR or MR or SD.

Progression was defined as a worsening of neurologic status that could not be explained by other causes, a \> 25% increase in tumor size, the appearance of new lesions or CSF positivity, or increasing doses of corticosteroids required to maintain stable status.

Overall survival was defined as the time from the date of randomization until the documented date of death. Participants who were still alive by the data cutoff date for analysis were censored on the last day the participant was known to be alive.

An AE was defined as any untoward medical occurrence in a study participant and did not necessarily have a causal relationship with study treatment. Clinically significant vital sign assessments, findings on physical or neurological examination, and laboratory findings associated with signs and/or symptoms requiring withdrawal, dose modification or medical intervention were recorded as AEs.

An AE was considered serious if it resulted in death, a life-threatening situation, inpatient hospitalization or prolongation of an existing hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect in the offspring of a patient who received study drug or other important medical events. The relationship of each AE to study drug was assessed as either related or not related.

Pharmacokinetic parameters were determined from the serial plasma concentration data obtained for erlotinib. Area under the plasma concentration-time curve from time zero to 24 hours (the dosing interval) measured at steady state using sparse sampling.

Pharmacokinetic parameters were determined from the serial plasma concentration data obtained for erlotinib. Maximum observed plasma concentration of erlotinib (Cmax) was measured at steady state on Day 14 using sparse sampling.

Pharmacokinetic parameters were determined from the serial plasma concentration data obtained for erlotinib. Time to the maximum observed plasma concentration of erlotinib (Tmax) was measured at steady state on Day 14 using sparse sampling.

Pharmacokinetic parameters were determined from the serial plasma concentration data obtained for erlotinib. Apparent body clearance (CL/F) of erlotinib was measured at steady state on Day 14 using sparse sampling.

Pharmacokinetic parameters were determined from the serial plasma concentration data obtained for erlotinib. The apparent volume of distribution (Vz/F) of erlotinib was measured at steady state on Day 14 using sparse sampling.

OSI Pharmaceuticals

Erlotinib

Total

Age, Continuous

Sex: Female, Male

Asian - Indian Subcontinent

Asian - Southeast Asia

Black

Other

White

Race/Ethnicity, Customized

Hispanic/Latino

Not Hispanic/Latino

Body Surface Area (BSA)

Anaplastic Ependymoma

Myxopapillary Ependymoma

Tumor Type for Initial Disease Diagnosis

Total Number of Disease Recurrences

Company makes no warranties or representations of any kind as to the posting, expressed or implied, including warranties of merchantability and fitness for a particular purpose, and shall not be liable for any damages.

Senior Medical Director, Medical Oncology

The study was not powered for this comparison due to small sample size.

The Full Analysis Set (FAS) consisted of all randomized participants. Following the intent-to-treat (ITT) principle, participants were analyzed according to the treatment arm they were assigned to at randomization.

Percentage of Participants With an Objective Response

Full Analysis Set participants whose best overall response was CR or PR.

Duration of Response

Percentage of Participants With a Minor Response

Percentage of Participants With Disease Control

Progression Free Survival (PFS)

Percentage of Participants With Prolonged Stable Disease

Full Analysis Set participants whose best overall response was CR, PR, MR or SD.

Duration of Stable Disease

Overall Survival (OS)

Any adverse event

Drug-related adverse event

Adverse event leading to death

Drug-related adverse event leading to death

Serious adverse event

Drug-related serious adverse event

AE leading to discontinuation

Drug-related AE leading to discontinuation

AE leading to dose interruption

Drug-related AE leading to dose interruption

AE leading to dose interruption and reduction

Related AE leading to dose interruption/ reduction

All enrolled participants who received at least 1 dose of study drug (Safety Analysis Set).

Safety Assessed Through Evaluation of Physical Exams, Vital Signs, Clinical Laboratory Tests and Adverse Events (AEs)

The Pharmacokinetics Analysis Set (PKAS) consisted of the participants treated with erlotinib for whom sufficient analyte concentration data were available to facilitate derivation of at least 1 primary pharmacokinetic parameter. Participants may have been excluded from the PKAS for reasons such as missing data or protocol deviation.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Erlotinib Versus Oral Etoposide in Patients With Recurrent or Refractory Pediatric Ependymoma	NCT01032070	Recurrent or Re...	erlotinib etoposide	1 Year - 21 Years	Astellas Pharma Inc
Erlotinib Versus Oral Etoposide in Patients With Recurrent or Refractory Pediatric Ependymoma	NCT01032070	Recurrent or Re...	erlotinib etoposide	1 Year - 21 Years	Astellas Pharma Inc

Spots Global Cancer Trial Database for recurrent or refractory pediatric ependymoma