New hope for kids with relapsed neuroblastoma: targeted drug lorlatinib enters first pediatric trial

NCT ID NCT03107988

Completed Disease control Sponsor: New Approaches to Neuroblastoma Therapy Consortium Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This early-stage trial tested a drug called lorlatinib in 65 children and young adults with a type of nerve cancer (neuroblastoma) that had come back or not responded to treatment. The drug targets a specific genetic change (ALK) found in their tumors. The study looked at safety, the right dose, and how the body processes the drug, both alone and with chemotherapy.

What this could mean

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

Active substance: Lorlatinib (an oral drug that targets ALK/ROS1 proteins), sometimes combined with chemotherapy drugs cyclophosphamide, topotecan, and filgrastim
What this could lead to: If successful, this could lead to a new treatment option for children with a hard-to-treat form of 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, so it is too soon to know if lorlatinib will work well. Side effects from the drug or chemotherapy may be significant, and the study is small.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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.

NEUROBLASTOMA

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

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