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Published results

SJPDGF: PDGFR Inhibitor Crenolanib in Children/ Young Adults with Diffuse Intrinsic Pontine Glioma or Recurrent High-Grade Glioma

Why was this study done?

This study tests the use of a new drug, crenolanib, to treat 2 types of brain cancer:

  • Newly diagnosed diffuse intrinsic pontine glioma (DIPG)
  • DIPG or high-grade glioma (HGG) that returned after treatment

These brain cancers can be hard to treat. They grow rapidly and are likely to spread. These tumors lie in a part of the brain that makes surgery difficult or impossible.

The usual treatment for DIPG tumors is brain radiation. It often does not cure the cancer. Chemotherapy after radiation does not help patients live longer. Patients need new medications that will fight their tumors better.

In this clinical trial, we tested the new targeted therapy drug crenolanib on patients with DIPG and HGG. Crenolanib helps block the activities of a group of proteins on the surface of cells called platelet-derived growth factor receptor (PDGFR). These proteins help tumors get the blood supply they need to grow and spread.

The study’s main goals were to:

  • Find the highest, safest crenolanib dosage that could treat patients without severe side effects
  • Learn about any side effects caused by the crenolanib
  • Study how the body handles (eliminates) crenolanib
  • Learn how crenolanib works in the body to fight brain cancer
  • Find out if crenolanib can be safely used alone or along with brain radiation
  • Learn if crenolanib, given alone or with radiation, can treat brain tumors better
  • Find out if imaging (MRI) can give new details about how tumors grow
  • Learn how this treatment affects the quality of life of patients and their parents

When was this study done?

The study opened in July 2011 and closed in October 2016.

What did the study consist of?

We treated 2 groups of patients ages 18 months to 21 years old:

  • Patients in Group A were newly diagnosed with DIPG. These patients received radiation and crenolanib for 6 weeks. They continued taking crenolanib for up to 2 years after radiation treatment.
  • Patients in Group B had DIPG or HGG that returned after treatment. They did not have radiation therapy but took crenolanib for up to 2 years.

During the study’s early stages, patients got the lowest crenolanib dosage. If this dose was safe, the dose slowly increased for each new group of patients. This process continued until we found the highest, safe dose.

During their treatment, patients had physical exams, blood and urine tests, MRI of their brains, and EKG (electrocardiogram) tests of their hearts. These tests showed:

  • How much crenolanib was in their body and how quickly their body eliminated it
  • Their response to the drug
  • How it affected their bodies
  • Any side effects

Patients and families also answered survey questions to find out how the treatments affected the patients’ quality of life.

What did we learn from this study?

Crenolanib treatment was well-tolerated and safe. The patient’s total body surface area (BSA) affected how well they eliminated the drug from their bodies. Doctors should consider this when deciding the dosage. Patients who took stomach acid reducers had more exposure to the crenolanib in their bodies. We developed a model for studying this drug safety in children and young adults with DIPG or recurrent HGG. In the future, we plan to look at other information from the results of this study.

What are the next research steps as a result of this study?

Information from this study will help researchers safely perform more clinical trials of crenolanib in children and young adults.

How does this study affect my child?

Every childhood cancer survivor should have long-term follow-up care. In the St. Jude After Completion of Care clinic, we will give your child information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines for your child.

For more information

Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

Publication generated from this study

Population pharmacokinetics of crenolanib in children and young adults with brain tumors. Bisbee C, Campagne O, Gajjar A, Tinkle CL, Stewart CF. Cancer Chemother Pharmacol. 2022 Apr;89(4):459-468. Epub 2022 Feb 25.

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.


Full title:

PDGFR Inhibitor Crenolanib in Children/ Young Adults with Diffuse Intrinsic Pontine Glioma or Recurrent High-Grade Glioma (SJPDGF) 

Study goal:

Find the highest, safest crenolanib dosage that could treat patients without severe side effects

For physicians and researchers

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