New hope for kids with relapsed neuroblastoma: targeted drug lorlatinib enters first pediatric trial
NCT ID NCT03107988
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.
Disclaimer
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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.
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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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
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Locations
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C.S Mott Children's Hospital
Ann Arbor, Michigan, 48109, United States
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Children Hospital of Colorado
Aurora, Colorado, 80045, United States
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027-0700, United States
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Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, 98105, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4318, United States
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Childrens Hospital Boston, Dana-Farber Cancer Institute.
Boston, Massachusetts, 02115, United States
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Cook Children's Healthcare System
Fort Worth, Texas, 76104, United States
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Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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Institut Curie
Paris, Cedex, 05, France
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Royal Marsden Hospital
Sutton, Surrey, SM25NG, United Kingdom
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UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94143, United States
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University of Chicago, Comer Children's Hospital
Chicago, Illinois, 60637, United States