New hope for neuroblastoma: targeted pill shows promise in early trial

NCT ID NCT03107988

First seen Nov 01, 2025 · Last updated Apr 28, 2026 · Updated 24 times

Summary

This early-phase study tested a daily pill called lorlatinib in 65 people with ALK-driven neuroblastoma, a rare nerve cancer that had come back or not responded to standard treatments. The goal was to find the safest dose and check for side effects, both when given alone and with chemotherapy. While not a cure, this targeted approach aims to better control the disease in patients who have few other options.

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Contacts and locations

Locations

  • C.S Mott Children's Hospital

    Ann Arbor, Michigan, 48109, United States

  • Children Hospital of Colorado

    Aurora, Colorado, 80045, United States

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027-0700, 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 Boston, Dana-Farber Cancer Institute.

    Boston, Massachusetts, 02115, United States

  • Cook Children's Healthcare System

    Fort Worth, Texas, 76104, United States

  • Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • Institut Curie

    Paris, Cedex, 05, France

  • Royal Marsden Hospital

    Sutton, Surrey, SM25NG, United Kingdom

  • 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

Conditions

Explore the condition pages connected to this study.