A Phase I Study of Pazopanib as a Single Agent for Children With Refractory Solid Tumors

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01130623
Recruitment Status : Withdrawn
First Posted : May 26, 2010
Last Update Posted : July 2, 2017
Information provided by:
National Institutes of Health Clinical Center (CC)

Brief Summary:


- Pazopanib, a drug that inhibits the growth of new blood vessels in tumors, was recently approved by the Food and Drug Administration to treat advanced kidney cancer in adults. Pazopanib has been used in only a small number of adults, and more research is needed on whether it is safe and effective to use in children. Researchers are interested in determining safe and effective treatment doses of pazopanib in children, and in other studies will examine which form of pazopanib treatment (tablet or liquid) is most effective and well tolerated.


  • To determine a safe and effective dose of pazopanib to treat solid tumors in children.
  • To study the effects of pazopanib on blood cells, blood flow, and human development.


- Children, adolescents, and young adults between 1 and 21 years of age who have been diagnosed with solid tumors that have not responded to treatment.


  • Eligible participants will be screened with a physical examination, blood and tumor samples, and imaging studies.
  • Participants will receive pazopanib tablets for 28-day cycles of treatment. Pazopanib should be taken on an empty stomach, at least 1hour before or 2 hours after a meal. Participants may receive pazopanib for up to 24 cycles unless the tumor does not respond or participants develop serious side effects.
  • Blood samples will be taken on days 1, 15, 22, and 27 of the first cycle of pazopanib, with additional samples taken every 8 weeks during subsequent cycles.
  • An optional part of the study will collect additional blood samples at regular intervals for 24 hours after the first dose of pazopanib and at regular intervals after another dose during the second or third week of the first treatment cycle.

Condition or disease Intervention/treatment Phase
Sarcoma Neuroblastoma Wilms Tumor Osteosarcoma Brain Tumor Drug: Pazopanib (GW786034) Phase 1

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Study of Pazopanib as a Single Agent for Children With Relapsed or Refractory Solid Tumors, Including CNS Tumors
Study Start Date : May 10, 2010
Actual Primary Completion Date : November 18, 2011
Actual Study Completion Date : November 18, 2011

Primary Outcome Measures :
  1. To estimate MTD and Phase II dose of oral pazopanib.
  2. To define the toxicities of oral pazopanib tablet or suspension.
  3. To characterize PKs.

Secondary Outcome Measures :
  1. To define antitumor and biologic activity and explore changes in tumor vascular permeability. To assess VEGF haplotype/phenotype relationships and explore concentration-effect with biomarkers and clinical outcomes.

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  • Eligibility:
  • Part 1 (Phase I Dose Escalation) and 2a (Suspension Formulation Component):

    --Patients greater than 12 months and less than or equal to 21 years of age with measureable or evaluable relapsed or refractory solid tumors including CNS tumors with histologic verification except in patients with intrinsic brain stem tumors, optic pathway gliomas, or patients with pineal tumors and elevation of tumor markers.

  • Part 2b (Expanded Imaging Cohort):

    --Patients greater than 2 years and less than or equal to 25 years of age with histologically verified relapsed or refractory soft tissue sarcoma, desmoplastic small round cell tumor or extraosseus Ewing sarcoma with measurable disease (greater than or equal to 2 cm) in the head, neck, extremity or fixed within the thorax, abdomen or pelvis.

  • Performance score: Karnofsky greater than or equal to 50% for patients 16 years of age; Lansky greater than or equal to 50 for patients less than or equal to 16 years of age.
  • Must have fully recovered from acute toxic effects from all prior therapy which have been completed within the specified prior time frame. Have adequate organ function as determined by laboratory evaluation.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01130623

United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Cancer Institute (NCI)

Responsible Party: CTEP/National Cancer Institute, National Institutes of Health Identifier: NCT01130623     History of Changes
Other Study ID Numbers: 100120
First Posted: May 26, 2010    Key Record Dates
Last Update Posted: July 2, 2017
Last Verified: November 18, 2011

Keywords provided by National Institutes of Health Clinical Center (CC):
Wilms Tumor
VEGF Inhibitor
Maximum Tolerated Dose
Dose Escalation
Antitumor Activity
Pediatric Cancer
Solid Tumor
Brain Tumor

Additional relevant MeSH terms:
Wilms Tumor
Brain Neoplasms
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Central Nervous System Neoplasms
Nervous System Neoplasms
Neoplasms by Site
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Bone Tissue
Neoplasms, Connective Tissue
Neoplasms, Complex and Mixed
Kidney Neoplasms
Urologic Neoplasms
Urogenital Neoplasms
Neoplastic Syndromes, Hereditary
Kidney Diseases
Urologic Diseases