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New hope for kids with tough neuroblastoma: oral drug trial begins

NCT ID NCT00295919

First seen Apr 22, 2026 · Last updated Jun 22, 2026 · Updated 9 times

Summary

This early-stage trial tested an oral powder form of the drug fenretinide (called LXS) in 80 children whose neuroblastoma had returned or resisted standard treatment. The main goals were to find the safest dose and understand side effects. Some patients also received ketoconazole to see if it boosted the drug's levels in the body.

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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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Contacts and locations

Locations

  • AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Scottish Rite Campus

    Atlanta, Georgia, 30342, United States

  • C.S. Mott Children's Hospital at University of Michigan Medical Center

    Ann Arbor, Michigan, 48109-0286, United States

  • Children's Hospital Boston

    Boston, Massachusetts, 02115, United States

  • Children's Hospital and Regional Medical Center - Seattle

    Seattle, Washington, 98105, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104-4318, United States

  • Childrens Hospital Los Angeles

    Los Angeles, California, 90027-0700, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039, United States

  • Cook Children's Medical Center - Fort Worth

    Fort Worth, Texas, 76104, United States

  • Lucile Packard Children's Hospital at Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • UCSF Helen Diller Family Comprehensive Cancer Center

    San Francisco, California, 94143, United States

  • University of Chicago Comer Children's Hospital

    Chicago, Illinois, 60637, United States

What this could mean

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

Active substance

fenretinide LXS oral powder

What this could lead to

If successful, this could point toward a new treatment option for children with neuroblastoma that has come back or not responded to standard therapy.

What could go wrong

This is an early Phase 1 trial focused on safety and dosing, not yet on effectiveness. The drug may not shrink tumors or improve survival, and side effects could be significant.

Conditions

The condition(s) this trial relates to.

neuroblastoma

As listed by the trial registrant

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