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New oral drug shows promise for kids with rare RET-Driven cancers

NCT ID NCT03899792

Ongoing Disease control Sponsor: Eli Lilly and Company Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests an oral drug called selpercatinib (LOXO-292) in children with advanced solid or brain tumors that have a specific RET gene change. The trial has two parts: first finding a safe dose, then measuring how many children's tumors shrink. About 36 children whose cancers have not responded to standard treatments are participating.

What this could mean

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

Active substance

Selpercatinib (also called LOXO-292), an oral drug that targets RET gene changes

What this could lead to

If successful, this could provide a new targeted treatment option for children with certain hard-to-treat cancers that have RET gene alterations.

What could go wrong

This is an early-phase trial with only 36 participants, so results may not apply broadly. The drug may cause side effects or not shrink tumors in many children.

Disclaimer Read more

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.

breast neoplasm bronchial neoplasm bronchogenic carcinoma Carcinoma, Medullary central nervous system cancer colonic neoplasm congenital fibrosarcoma endocrine system disorder Head and Neck Neoplasms infantile myofibromatosis lung disorder lung neoplasm malignant endocrine neoplasm medullary thyroid gland carcinoma neoplasm Neoplasms by Site non-small cell lung carcinoma primary brain neoplasm respiratory system disorder respiratory tract neoplasm sarcoma soft tissue sarcoma Thoracic Neoplasms Thyroid Cancer, Papillary thyroid gland disorder thyroid gland papillary carcinoma Thyroid Neoplasms

As listed by the trial registrant

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

INFANTILE FIBROSARCOMA INFANTILE MYOFIBROMATOSIS MEDULLARY THYROID CANCER PAPILLARY THYROID CANCER SOFT TISSUE SARCOMA

Contacts and locations

Locations

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Childrens Hospital of Los Angeles

    Los Angeles, California, 90027, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039, United States

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Fondazione IRCCS Istituto Nazionale dei Tumori

    Milan, Lombardy, 20133, Italy

  • Gustave Roussy

    Villejuif, 94805, France

  • Hiroshima University Hospital

    Hiroshima, 734-8551, Japan

  • Hokkaido University Hospital

    Sapporo, Hokkaido, 060-8648, Japan

  • Hospital Universitari Vall d'Hebron

    Barcelona, Barcelona [Barcelona], 8035, Spain

  • Kyoto University Hospital

    Kyoto, 606-8507, Japan

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • National Cancer Center Hospital

    Chuo-ku, Tokyo, 104-0045, Japan

  • Nemours Children's Health

    Orlando, Florida, 32827, United States

  • Rigshospitalet

    Copenhagen, 2200, Denmark

  • Royal Children's Hospital

    Melbourne, Victoria, 3052, Australia

  • Seattle Children's Hospital Research Foundation

    Seattle, Washington, 98105, United States

  • Seoul National University Hospital

    Seoul, Korea, 03080, South Korea

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • Texas Childrens Hospital

    Houston, Texas, 77025, United States

  • The Children's Hospital at Westmead

    Westmead, New South Wales, 2145, Australia

  • The Children's Hospital for Cancer and Blood Disorders

    Aurora, Colorado, 80045, United States

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • University College Hospital - London

    London, Greater London, NW1 2BU, United Kingdom

  • University of Minnesota Hospital

    Minneapolis, Minnesota, 55455, United States

  • University of Texas Southwestern Medical Center at Dallas

    Dallas, Texas, 75390-9063, United States

  • Universitätsklinikum Heidelberg

    Heidelberg, Baden-Wurttemberg, 69115, Germany