A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors

NCT ID: NCT03899792

Last Updated: 2026-01-06

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

36 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-06-13

Study Completion Date

2029-05-31

Brief Summary

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This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric participants with an activating rearranged during transfection (RET) alteration and an advanced solid or primary CNS tumor.

Detailed Description

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This study includes 2 parts: phase 1 (dose escalation) and phase 2 (dose expansion). In phase 1, participants will be enrolled using a rolling 6 dose escalation scheme. The starting dose of LOXO-292 is equivalent to the adult recommended phase 2 dose of 160 milligrams (mg) twice a day (BID). Once the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) is identified, participants will be enrolled to one of four phase 2 dose expansion cohorts depending on tumor histology and tumor genotype. Cycle length will be 28 days.

Conditions

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Medullary Thyroid Cancer Infantile Myofibromatosis Infantile Fibrosarcoma Papillary Thyroid Cancer Soft Tissue Sarcoma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1: Medullary Thyroid Cancer (MTC) Group

Participants in this cohort had MTC and received 160 mg of selpercatinib BID orally on Days 1 through 28 of a 28-day cycle.

The treatment was continued until participants experienced a progressive disease, unacceptable toxicity, or other protocol-defined reason for discontinuation.

Group Type EXPERIMENTAL

Selpercatinib

Intervention Type DRUG

Oral LOXO-292

Cohort 2: Papillary Thyroid Cancer (PTC) Group

Participants in this cohort had PTC and received 160 mg of selpercatinib BID orally on Days 1 through 28 of a 28-day cycle.

The treatment was continued until participants experienced a progressive disease, unacceptable toxicity, or other protocol-defined reason for discontinuation.

Group Type EXPERIMENTAL

Selpercatinib

Intervention Type DRUG

Oral LOXO-292

Cohort 3: Other Cancer Group

Participants in this cohort had other (REarranged during Transfection (RET)-altered non-thyroid) cancer and received 160 mg of selpercatinib BID orally on Days 1 through 28 of a 28-day cycle.

The treatment was continued until participants experienced a progressive disease, unacceptable toxicity, or other protocol-defined reason for discontinuation.

Group Type EXPERIMENTAL

Selpercatinib

Intervention Type DRUG

Oral LOXO-292

Interventions

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Selpercatinib

Oral LOXO-292

Intervention Type DRUG

Other Intervention Names

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LOXO-292 LY3527723

Eligibility Criteria

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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\[s\])
Minimum Eligible Age

6 Months

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Eli Lilly and Company

INDUSTRY

Sponsor Role lead

Loxo Oncology, Inc.

INDUSTRY

Sponsor Role collaborator

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST)

Role: STUDY_DIRECTOR

Eli Lilly and Company

Locations

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Childrens Hospital of Los Angeles

Los Angeles, California, United States

Site Status

The Children's Hospital for Cancer and Blood Disorders

Aurora, Colorado, United States

Site Status

Nemours Children's Health

Orlando, Florida, United States

Site Status

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

University of Minnesota Hospital

Minneapolis, Minnesota, United States

Site Status

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, United States

Site Status

Texas Childrens Hospital

Houston, Texas, United States

Site Status

Seattle Children's Hospital Research Foundation

Seattle, Washington, United States

Site Status

The Children's Hospital at Westmead

Westmead, New South Wales, Australia

Site Status

Royal Children's Hospital

Melbourne, Victoria, Australia

Site Status

The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Rigshospitalet

Copenhagen, , Denmark

Site Status

Gustave Roussy

Villejuif, , France

Site Status

Universitätsklinikum Heidelberg

Heidelberg, Baden-Wurttemberg, Germany

Site Status

Fondazione IRCCS Istituto Nazionale dei Tumori

Milan, Lombardy, Italy

Site Status

Hokkaido University Hospital

Sapporo, Hokkaido, Japan

Site Status

National Cancer Center Hospital

Chuo-ku, Tokyo, Japan

Site Status

Hiroshima University Hospital

Hiroshima, , Japan

Site Status

Kyoto University Hospital

Kyoto, , Japan

Site Status

Seoul National University Hospital

Seoul, Korea, South Korea

Site Status

Hospital Universitari Vall d'Hebron

Barcelona, Barcelona [Barcelona], Spain

Site Status

University College Hospital - London

London, Greater London, United Kingdom

Site Status

Countries

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United States Australia Canada Denmark France Germany Italy Japan South Korea Spain United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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J2G-OX-JZJJ

Identifier Type: OTHER

Identifier Source: secondary_id

LOXO-RET-18036

Identifier Type: OTHER

Identifier Source: secondary_id

2019-000212-28

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

17493

Identifier Type: -

Identifier Source: org_study_id

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