A Phase II Study of Imetelstat (GRN163L) in Children with Recurrent Brain Tumors

Protocol
13-099
Full Title
PBTC-036: A Molecular Biology and Phase II Study of Imetelstat (GRN163L) in Children with Recurrent High-Grade Glioma, Ependymoma, and Diffuse Intrinsic Pontine Glioma
Phase
II
Purpose

The purpose of this study is to assess the safety and effectiveness of the investigational drug imetelstat in children with malignant glioma, diffuse intrinsic pontine glioma, medulloblastoma/primitive neuroectodermal tumor, or ependymoma that has continued to grow or has come back despite standard therapy. This study is being conducted as part of the Pediatric Brain Tumor Consortium, a group of U.S. centers dedicated to advancing the treatment of brain tumors in children.

Imetelstat works by inhibiting the activity of an enzyme called telomerase. This enzyme helps extend the ends of chromosomes (telomeres). Cancer cells must maintain the lengths of their telomeres to continue to grow, and they use telomerase to accomplish this. Telomerase is active in most pediatric solid tumors.

Imetelstat is given intravenously (by vein).

Eligibility

To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • This study is open to patients at least 1 year of age but no older than 21 years.
  • Patients must have malignant glioma, diffuse intrinsic pontine glioma, medulloblastoma/primitive neuroectodermal tumor, or ependymoma that has continued to grow or has come back despite standard therapy.
  • Patients should have recovered from the serious side effects of prior treatments.

For more information and to inquire about eligibility for this study, please contact Dr. Kim Kramer at 212-639-6410.

Disease(s)
Pediatric Brain Tumors: Brain Cancer
Pediatric Brain Tumors: CNS Cancer
Locations
Related Diseases