NCT01032070

Brief Summary

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.

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2010

Geographic Reach
3 countries

28 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 10, 2009

Completed
5 days until next milestone

First Posted

Study publicly available on registry

December 15, 2009

Completed
10 months until next milestone

Study Start

First participant enrolled

September 27, 2010

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 26, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 26, 2012

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

January 8, 2014

Completed
Last Updated

December 12, 2024

Status Verified

November 1, 2024

Enrollment Period

2.2 years

First QC Date

December 10, 2009

Results QC Date

November 20, 2013

Last Update Submit

November 18, 2024

Conditions

Keywords

pediatricetoposideerlotinibphase 2ependymoma

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants With an Objective Response

    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.

    From randomization until the end of treatment. The median time on treatment was 52 days for erlotinib and 58 days for etoposide.

Secondary Outcomes (13)

  • Duration of Response

    From randomization until the end of treatment. The median time on treatment was 52 days for erlotinib and 58 days for etoposide.

  • Percentage of Participants With a Minor Response

    From randomization until the end of treatment. The median time on treatment was 52 days for erlotinib and 58 days for etoposide.

  • Percentage of Participants With Disease Control

    From randomization until the end of treatment. The median time on treatment was 52 days for erlotinib and 58 days for etoposide.

  • Progression Free Survival (PFS)

    From randomization until the end of treatment. The median time on treatment was 52 days for erlotinib and 58 days for etoposide.

  • Percentage of Participants With Prolonged Stable Disease

    From randomization until the end of treatment. The median time on treatment was 52 days for erlotinib and 58 days for etoposide.

  • +8 more secondary outcomes

Study Arms (2)

Erlotinib

EXPERIMENTAL

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.

Drug: erlotinib

Etoposide

ACTIVE COMPARATOR

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.

Drug: etoposide

Interventions

oral

Also known as: OSI-774, Tarceva
Erlotinib

oral

Also known as: VP-16
Etoposide

Eligibility Criteria

Age1 Year - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Recurrent of refractory ependymoma or subependymoma
  • Performance Status (PS): Lansky ≥ 50% for patients ≤ 10 years of age or Karnofsky ≥ 50% for patients \>10 years of age
  • Measurable disease, defined as 1 measurable lesion that can be accurately measured in 2 planes that has not received radiation therapy within 12 weeks
  • Recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy
  • ≥ 1 year to ≤ 21 years
  • Serum creatinine for patients ≤ 5 years in age is ≤ 0.8 mg/dL or Creatinine Clearance/Glomerular Filtration Rate (GFR) ≥ 70 mL/min/m\^2
  • Serum creatinine for patients \> 5 and ≤ 10 years in age is ≤ 1.0 mg/dL or Creatinine Clearance/GFR ≥ 70 mL/min/m\^2
  • Serum creatinine for patients \> 10 and ≤ 15 years in age is ≤ 1.2 mg/dL or Creatinine Clearance/GFR ≥ 70 mL/min/m\^2
  • Serum creatinine for patients \> 15 years in age is ≤ 1.5 mg/dL or Creatinine Clearance/GFR ≥ 70 mL/min/m\^2
  • Total bilirubin is ≤ 1.5 x upper limit of normal for age
  • Alanine aminotransferase (ALT) ≤ 3 x upper limit of normal
  • Absolute neutrophil count \> 1000/µL
  • Platelet count \> 100,000/µL
  • Hemoglobin \> 8 gm/dL
  • Neurologically stable for at least 7 days prior to randomization
  • +2 more criteria

You may not qualify if:

  • Previously received epidermal growth factor receptor (EGFR)-targeted therapy
  • Previously received oral etoposide
  • Received craniospinal radiotherapy within 24 weeks prior to randomization
  • Received field radiotherapy to the target lesion within 12 weeks prior to randomization
  • Received symptomatic metastatic disease within 14 days prior to randomization
  • Received myelosuppressive chemotherapy within 21 days before randomization
  • Received growth factors within 7 days prior to randomization
  • Participating in another investigational drug trial
  • Received a biologic agent within 7 days prior to randomization
  • Received a monoclonal antibody within 28 days prior to randomization
  • Taking cytochrome P450 (CYP)3A4 or CYP1A2 inhibitors/inducers within 14 days prior to randomization
  • Taking proton pump inhibitors within 14 days prior to randomization
  • Smoking during treatment
  • Pregnant or breast-feeding females

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (28)

University of Alabama at Birmingham Dept. of Pediatric-Hematology/Oncology

Birmingham, Alabama, 35233, United States

Location

Center for Cancer and Blood Disorders-Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Children's Hospital of Orange County (CHOC)

Orange, California, 92868, United States

Location

Stanford University and Lucile Packard Children's Hospital

Palo Alto, California, 94304, United States

Location

Children's Hospital Center for Cancer and Blood Disorders

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center - D.C. Center for Cancer and Blood Disorders

Washington D.C., District of Columbia, 20010, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Emory University Children's Healthcare of Atlanta Aflac Cancer Center & Blood Disorders

Atlanta, Georgia, 30322, United States

Location

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21287, United States

Location

Dana-Farber Cancer Institute Department of Pediatric Neuro-oncology

Boston, Massachusetts, 02115, United States

Location

Amplatz Children's Hospital University of Minnesota Medical Center- Pediatric Hematology Oncology

Minneapolis, Minnesota, 55455, United States

Location

Steven D Hassenfeld Children's Center - New York University

New York, New York, 10016, United States

Location

Columbia University Children's Hospital of New York Presbyterian Child & Adolescent Oncology Center

New York, New York, 10032, United States

Location

Oregon Health & Sciences University Doembecher Children's Hospital

Portland, Oregon, 97124, United States

Location

Penn State Hershey Children's Hospital

Hershey, Pennsylvania, 17110, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Children's Medical Center, Dallas Center for Cancer and Blood Disorders

Dallas, Texas, 75235, United States

Location

University of Wisconsin Pediatric Hematology/Oncology Department

Madison, Wisconsin, 53705-2275, United States

Location

Strollery Children's Hospital Division of Hematology/Oncology

Edmonton, Alberta, T6G 2B7, Canada

Location

Children's and Women's Health Center of BC Division of Hematology/ Oncology/ BMT

Vancouver, British Columbia, V6H 3V4, Canada

Location

Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

Royal Hospital for Sick Children

Glasgow, G3 8SJ, United Kingdom

Location

Paediatric Oncology and Haematology Offices

Leeds, LS1 3EX, United Kingdom

Location

Alder Hey Children's NHS Foundation Trust Department of Oncology

Liverpool, L12 1AP, United Kingdom

Location

Royal Manchester Children's Hospital Ward 84

Manchester, M13 9W2, United Kingdom

Location

University of Nottingham Children's Brain Tumour Research Centre

Nottingham, NG7 2UH, United Kingdom

Location

Royal Marsden Hospital

Sutton, SM2 5PT, United Kingdom

Location

Related Links

MeSH Terms

Conditions

RecurrenceFamilial ependymomaEpendymoma

Interventions

Erlotinib HydrochlorideEtoposide

Condition Hierarchy (Ancestors)

Disease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsGliomaNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

QuinazolinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsPodophyllotoxinTetrahydronaphthalenesNaphthalenesPolycyclic Aromatic HydrocarbonsHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsOrganic ChemicalsPolycyclic CompoundsGlucosidesGlycosidesCarbohydrates

Limitations and Caveats

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.

Results Point of Contact

Title
Senior Medical Director, Medical Oncology
Organization
Astellas Pharma Global Development, Inc

Study Officials

  • Medical Monitor

    Astellas Pharma Global Development

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 10, 2009

First Posted

December 15, 2009

Study Start

September 27, 2010

Primary Completion

November 26, 2012

Study Completion

November 26, 2012

Last Updated

December 12, 2024

Results First Posted

January 8, 2014

Record last verified: 2024-11

Data Sharing

IPD Sharing
Will not share

Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Locations