Combination drug approach aims to tackle tough childhood cancer

NCT ID NCT04299113

First seen Jul 17, 2026 · Last updated Jul 17, 2026

Summary

This early-phase trial tests a combination of two drugs—mocetinostat and vinorelbine—in children, adolescents, and young adults with rhabdomyosarcoma that has spread, cannot be surgically removed, or has returned after treatment. Mocetinostat works by blocking enzymes that help cancer cells grow, while vinorelbine is a chemotherapy drug that stops cancer cells from dividing. The study aims to find the safest and most effective dose of this combination.

What this could mean

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

Active substance
mocetinostat and vinorelbine
What this could lead to
If successful, this combination could offer a new treatment option for children and young adults with rhabdomyosarcoma that has not responded to standard therapy.
What could go wrong
This is an early-phase trial with a small number of participants, so the benefits and risks are not yet well understood. The combination may cause significant side effects or may not work better than existing treatments.

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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.

Contacts and locations

Locations

  • Rebecca Phelan

    Los Angeles, California, 90095, United States

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