Promising drug targets rare childhood tumors in new trial
NCT ID NCT02114229
First seen Mar 22, 2026 · Last updated Jun 22, 2026 · Updated 14 times
Summary
This study tests a drug called alisertib in children and young adults (up to age 22) with rare, aggressive tumors called atypical teratoid rhabdoid tumors (AT/RT) in the brain or malignant rhabdoid tumors (MRT) elsewhere in the body. For patients whose cancer has come back or not responded to treatment, alisertib is given alone. For newly diagnosed patients, it is combined with chemotherapy and sometimes radiation. The goal is to see if the drug can shrink tumors or keep them from growing.
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital and Clinics of Minnesota
Minneapolis, Minnesota, 55102, United States
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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Lucille Packard Children's Hospital at Stanford University Medical Center
Palo Alto, California, 94304, United States
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Rady Children's Hospital
San Diego, California, 92123, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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UF Cancer Center at Orlando Health
Orlando, Florida, 32806, United States
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
alisertib
What this could lead to
If successful, this could provide a new treatment option for children with these rare, aggressive tumors, potentially improving survival and delaying disease progression.
What could go wrong
This is a phase 2 trial, so results are still early. The drug may not work for all patients, and side effects like low blood counts and mouth sores are possible. The study is no longer recruiting, so final results are pending.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.