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

NCT ID NCT03899792

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

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

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

36 people

The number who actually took part.

Started

Jun 2019

Expected to finish

May 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 months to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Advanced or metastatic solid or primary CNS tumor which has failed standard of care therapies * Evidence of an activating RET gene alteration in the tumor and/or blood * Measurable or non-measurable disease * Karnofsky (participants 16 years and older) or Lansky (participants younger than 16) performance score of at least 50 * Participant with primary CNS tumors or cerebral metastases must be neurologically stable for 7 days prior and must not have required increasing doses of steroids within the last 7 days * Adequate hematologic, hepatic and renal function. * Ability to receive study drug therapy orally or via gastric access * Willingness of men and women of reproductive potential to observe conventional and effective birth control Exclusion Criteria: * Major surgery within two weeks prior to planned start of LOXO-292 * Clinically significant, uncontrolled cardiac, cardiovascular disease or history of myocardial infarction within 6 months prior to planned start of LOXO-292 * Active uncontrolled systemic bacterial, viral, fungal or parasitic infection * Clinically significant active malabsorption syndrome * Pregnancy or lactation * Uncontrolled symptomatic hyperthyroidism or hypothyroidism (i.e. the participant required a modification to current thyroid medication in the 7 days before start of LOXO-292) * Uncontrolled symptomatic hypercalcemia or hypocalcemia * Known hypersensitivity to any of the components of the investigational agent, LOXO-292 or Ora-Sweet® SF and OraPlus®, for participants who will receive LOXO-292 suspension * Prior treatment with a selective RET inhibitor(s) (including investigational selective RET inhibitor\[s\])

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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

More trials for these conditions

Other studies related to the condition(s) this trial covers.