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SJDAWN: Molecular-Based Therapy for Aggressive Brain and Spinal Cord Tumors in Children and Young Adults

About this study

Doctors want to find new treatments for brain tumors that come back after treatment or do not get better with treatment. They know tumors disrupt the natural “checks and balances” of the body’s cells. This disruption forces cells to grow and divide. Doctors refer to this process as “cell cycle activation.” The drugs that inhibit these molecular signals are known as “cell cycle inhibitors.”

Doctors have also identified some drugs that have potential to kill particular brain tumors. However, these drugs need an extra boost to be more effective. Researchers in this study want to find out if giving a combination of cell cycle inhibitors with specific drugs tailored to a particular tumor type can treat these kinds of aggressive tumors.

This study will test a combination drug therapy in children and young adults with tumors of the brain and spinal cord. This study is called a Phase I study because the goal is to find the highest doses of these drugs that can be given together safely.

The first part of the study is a screening phase. In this phase, doctors will test a sample of your tumor to see if it is a good target for one of three treatments in this clinical trial. If the test shows you are eligible, St. Jude will assign you to one of three treatment groups. These treatment groups are called Stratum A, Stratum B and Stratum C.

All study participants will receive a drug called ribociclib, a cell cycle inhibitor. This drug will be given in combination with one of these three additional drugs: gemcitabine, trametinib, or sonidegib. In Stratum A, participants will receive ribociclib and gemcitabine. In Stratum B, participants will receive ribociclib and trametinib. In Stratum C, participants will receive ribociclib and sonidegib. Your specific treatment will depend on your tumor type and the treatment group you are in.

Ribociclib has been approved by the U.S. Food and Drug Administration (FDA) for treating adults with breast cancer. Gemcitabine has been approved by the FDA for treating adults with ovarian, breast, lung and pancreatic cancer. Trametinib and sonidegib have been approved by the FDA to treat adults with certain types of skin cancer. None of these drugs have been approved to treat brain tumors or to treat children with any type of cancer. However, our research suggests that these drugs may have a good effect against brain tumors.

Purpose of this clinical trial

The main goal of this study is to find out the highest dose of the combination medicine that is safe to give children and young adults with these brain tumors. Researchers also want to learn about the good and bad effects of these drug combinations, as well as learn more about how your body absorbs, processes and removes these drugs from your body (pharmacokinetics).

Participants will also have the option to participate in additional studies. These may include additional blood tests and tests on the already removed tumor. These studies will help researchers better understand the molecular makeup of your type of brain tumor and better inform research about your tumor’s response to therapy. You will not get a direct health benefit from these optional studies. However, the results may help people with the same type of tumor in the future. The goal of this research is to eventually improve the ability to match therapy with your type of tumor.

Eligibility overview

  • 1 to 39 years old
  • Recurrent, progressive or refractory medulloblastoma, ependymoma or central nervous system (CNS) tumor

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:

Molecularly-Driven Doublet Therapy for All Children with Refractory or Recurrent CNS Malignant Neoplasms and Young Adults with Refractory or Recurrent SHH Medulloblastoma

Study goal:

The main goal of this study is to find out the highest dose of the combination medicine that is safe to give children and young adults with these brain tumors.


1 to 39 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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