New hope for young sarcoma patients: experimental drug combo targets tough tumors

NCT ID NCT04890093

Terminated Disease control Sponsor: National Cancer Institute (NCI) Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a new drug called PEN-866, which stays in tumor cells longer than normal cells, combined with two standard chemotherapy drugs (vincristine and temozolomide). It is for people aged 12 to 39 with sarcomas (Ewing sarcoma or rhabdomyosarcoma) that have returned or not responded to treatment. The goal is to see if the combination is safe and can shrink tumors.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: PEN-866 (a drug that stays longer in tumor cells) combined with vincristine and temozolomide
What this could lead to: If it works, this could offer a new treatment option for young people with sarcomas that have come back or not responded to standard therapy.
What could go wrong: This trial is early (phase 1/2) and currently suspended, so results are uncertain. The combination may cause side effects or fail to shrink tumors.

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.

Ewing sarcoma neoplasm rhabdomyosarcoma sarcoma

As listed by the trial registrant

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

RHABDOMYOSARCOMA SARCOMA, EWING

Contacts and locations

Locations

Childrens National Medical Center

Washington D.C., District of Columbia, 20010, United States
National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

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