Promising drug targets rare childhood tumors in new trial

NCT ID NCT02114229

Ongoing Disease control Sponsor: St. Jude Children's Research Hospital Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

atypical teratoid rhabdoid tumor rhabdoid tumor

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ATYPICAL TERATOID/RHABDOID TUMOR MALIGNANT RHABDOID TUMOR

Contacts and locations

Locations

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital and Clinics of Minnesota

Minneapolis, Minnesota, 55102, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Lucille Packard Children's Hospital at Stanford University Medical Center

Palo Alto, California, 94304, United States
Rady Children's Hospital

San Diego, California, 92123, United States
St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
Texas Children's Hospital

Houston, Texas, 77030, United States
UF Cancer Center at Orlando Health

Orlando, Florida, 32806, United States

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