Can off-label cancer drugs help children? a national study aims to find out

NCT ID NCT07727694

First seen Jul 27, 2026 · Last updated Jul 28, 2026 · Updated 1 time

Summary

This study collects safety and effectiveness data on cancer medicines used off-label or through compassionate access in children, adolescents, and young adults up to age 25 with pediatric malignancies. Participants are not enrolled in other treatment trials and are discussed by expert tumor boards. The goal is to gather real-world evidence on how these innovative drugs work in younger patients.

What this could mean

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

Active substance
off-label or compassionate-use cancer medicines (targeted therapies, immunotherapies, chemotherapies) approved in adults after 2007
What this could lead to
If successful, this could provide real-world evidence to support safer and more effective use of innovative cancer drugs in children and young adults.
What could go wrong
This is an observational data collection study, not a controlled trial, so it cannot prove cause and effect. Results may be limited by incomplete data or differences in how patients are treated.

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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

  • CHC Montlégia

    NOT_YET_RECRUITING

    Liège, 4000, Belgium

  • Cliniques Universitaires Saint-Luc

    NOT_YET_RECRUITING

    Brussels, 1200, Belgium

  • Hôpital Universitaire des Enfants Reine Fabiola (HUDERF)

    NOT_YET_RECRUITING

    Brussels, 1020, Belgium

  • University Hospital Antwerp

    NOT_YET_RECRUITING

    Antwerp, 2650, Belgium

  • University Hospital Gent

    NOT_YET_RECRUITING

    Ghent, 9000, Belgium

  • University Hospital Leuven

    RECRUITING

    Leuven, 3000, Belgium

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