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

NCT ID NCT04890093

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.

Disclaimer Read more

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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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.

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