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PBTC47: A Clinical Trial of the Drug, Panobinostat, in Children with Brain Tumors

About this study

Diffuse intrinsic pontine glioma, also known as DIPG, is a tumor that starts in the brain stem above the back of the neck. DIPG is a very stubborn cancer. It is hard to treat because it is located in a very sensitive area of the body that controls breathing and other important functions. In most cases, doctors do not want to operate on a tumor in this part of the body because of the risk to the brain. Most doctors recommend treatment with high-energy X-rays or other type of radiation. Treatment may also include chemotherapy. Even with these treatments, the number of children who survive DIPG is very low.

Researchers are studying new drugs that may do a better job of fighting this type of cancer. One of these experimental drugs is called panobinostat. This drug has been approved by the U.S. Food and Drug Administration (FDA) for use in adults with a type of cancer called multiple myeloma but has not been approved for children. Its use for children with DIPG is considered experimental.

Based on laboratory and animal studies, researchers believe panobinostat may kill DIPG tumor cells.

Purpose of this study

The main goal of this study is to find out if children with DIPG can safely take panobinostat and learn the best dose that will kill their tumors. Researchers also want to learn what kinds of side effects happen in children with DIPG when they take this drug and how their bodies handle this drug.

Eligibility overview

  • Diagnosis of DIPG
  • At least 2 but not more than 22 years old
  • Able to swallow capsules whole

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.

Overview

Full title:

Phase 1 Trial of Panobinostat in Children With Diffuse Intrinsic Pontine Glioma

Study goal:

The main goal of this study is to find out if children with DIPG can safely take panobinostat and learn the best dose that will kill their tumors.

Age:

At least 2 but not more than 22 years old

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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