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New hope for kids with rare cancers: targeted drug repotrectinib enters trial

NCT ID NCT04094610

Disease control Sponsor: Turning Point Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Sep 04, 2026 · Updated 1 time

Summary

This study tests a drug called repotrectinib in children and young adults (up to age 25) with advanced solid tumors, lymphoma, or brain tumors that have specific gene changes (ALK, ROS1, or NTRK1-3). The goal is to find the safest dose and see if the drug shrinks tumors. Participants must have a confirmed gene change and be in good enough health to receive the treatment.

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

About 75 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2020

Expected to finish

Sep 2027

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

Up to 25 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.

Key Inclusion Criteria: 1. Documented genetic ROS1 point mutation, fusion, or amplification or NTRK1-3 fusion as identified by local testing in a Clinical Laboratory Improvement Amendments (CLIA) laboratory in the US or equivalently accredited diagnostic lab outside the United States (US) is required. 2. Phase 1: Age \<12 years; Phase 2: Age 12- 25 years 3. Prior cytotoxic chemotherapy is allowed. 4. Prior immunotherapy is allowed. 5. Resolution of all acute toxic effects (excluding alopecia) of any prior anti-cancer therapy to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 4.03 Grade less than or equal to 1. 6. All subjects must have measurable disease by RECIST v1.1 or Response Assessment in Neuro-Oncology (RANO) criteria at time of enrollment. 7. Subjects with a primary CNS tumor or CNS metastases must be neurologically stable on a stable or decreasing dose of steroids for at least 7 days prior to enrollment. 8. Subjects must have a Lansky (\< 16 years) or Karnofsky (≥ 16 years) score of at least 50. 9. Life expectancy greater than or equal to 12 weeks, in the investigator's opinion. 10. Adequate hematologic, renal and hepatic function. Phase 2 Inclusion Criteria: 1. Cohort Specific Inclusion Criteria: * Cohort 1: Subjects with NTRK fusion gene positive (NTRK+) advanced solid tumors (including primary CNS tumors), that are tropomyosin receptor kinase (TRK) TKI naïve; * Cohort 2: subjects with NTRK+ advanced solid tumors (including primary CNS tumors), that are TRK TKI pre-treated; * Cohort 3: subjects with advanced solid tumors with ROS1 gene fusions or other ROS1 aberrations (including amplifications and point mutations) with measurable disease. 2. Subjects in Cohorts 1 and 2 must have prospectively confirmed measurable disease by BICR prior to enrollment. Key Exclusion Criteria (Phase 1 and Phase 2): 1. Subjects with neuroblastoma with only bone marrow disease evaluable by bone marrow aspiration only. 2. Major surgery within 14 days (2 weeks) of start of repotrectinib treatment. Central venous access (Broviac, Mediport, etc.) placement does not meet criteria for major surgery. 3. Known active infections requiring ongoing treatment (bacterial, fungal, viral including HIV positivity). 4. Gastrointestinal disease (e.g., Crohn's disease, ulcerative colitis, or short gut syndrome) or other malabsorption syndromes that would impact drug absorption. 5. Any of the following cardiac criteria: * Mean resting corrected QT interval (ECG interval measured from the onset of the QRS complex to the end of the T wave) for heart rate (QTc) \> 480 msec obtained from three ECGs, using the screening clinic ECG machine-derived QTc value * Any clinically important abnormalities in rhythm, conduction, or morphology of resting ECG (e.g., complete left bundle branch block, third degree heart block, second degree heart block, PR interval \> 250 msec) * Any factors that increase the risk of QTc prolongation or risk of arrhythmic events such as heart failure, congenital long QT syndrome, family history of long QT syndrome, or any concomitant medication known to prolong the QT interval 6. Peripheral neuropathy of CTCAE ≥grade 2. 7. Subjects being treated with or anticipating the need for treatment with strong CYP3A4 inhibitors or inducers. 8. Any potential allergies to repotrectinib and/or its excipients.

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Conditions

The condition(s) this trial relates to.

ALK-positive anaplastic large cell lymphoma anaplastic large cell lymphoma central nervous system cancer central nervous system neoplasm glioblastoma glioma inflammatory myofibroblastic tumor lymphoma metastatic malignant neoplasm neoplasm Neoplasm Metastasis NTRK fusion positive cancer primary brain neoplasm sarcoma solitary fibrous tumor

As listed by the trial registrant

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

Locally advanced solid tumors Lymphoma Metastatic solid tumors Primary CNS tumors

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    53 sites in 11 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Alder Hey Children's NHS Foundation Trust

    RECRUITING

    Liverpool, England, L12 2AP, United Kingdom

  • Asan Medical Center

    RECRUITING

    Seoul, 05505, South Korea

  • Baylor College of Medicine

    RECRUITING

    Houston, Texas, 77030, United States

  • Centre Hospitalier Universitaire D'Angers

    RECRUITING

    Angers, 49933, France

  • Centre Hospitalier Universitaire de Bordeaux - Groupe Hospitalier Pellegrin

    RECRUITING

    Bordeaux, 33076, France

  • Children'S Hospital Of Eastern Ontario

    RECRUITING

    Ottawa, Ontario, K1H 8L1, Canada

  • Children's Health Queensland Hospital and Health Service

    RECRUITING

    South Brisbane, Queensland, 4101, Australia

  • Children's Healthcare of Atlanta - Egleston Hospital

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Children's Hospital Colorado - Anschutz Medical Campus

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    RECRUITING

    Los Angeles, California, 90027-6062, United States

  • Children's Hospital of Philadelphia-Center for Childhood Cancer Research

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of Richmond at VCU

    WITHDRAWN

    Richmond, Virginia, 23219, United States

  • Clinica Universidad de Navarra

    RECRUITING

    Madrid, 28022, Spain

  • Clínica Universidad de navarra

    RECRUITING

    Pamplona, Navarre, 31008, Spain

  • Dana Farber Cancer Institute.

    RECRUITING

    Boston, Massachusetts, 02215, United States

  • Fondazione IRCCS - Istituto Nazionale dei Tumori

    RECRUITING

    Milan, 20133, Italy

  • Great Ormond Street Hospital For Children NHS Foundation Trust

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • HM Sanchinarro University Hospital

    RECRUITING

    Madrid, 28050, Spain

  • Hospital Infantil Universitario Nino Jesus

    RECRUITING

    Madrid, 28009, Spain

  • Hospital Sant Joan De Deu

    RECRUITING

    Esplugues de Llobregat, Barcelona, 08950, Spain

  • Hospital Universitari Vall d'Hebron

    RECRUITING

    Barcelona, 8035, Spain

  • Hospital Universitario Virgen del Rocio

    RECRUITING

    Seville, 41013, Spain

  • Hospital Universitario Y Politecnico La Fe

    RECRUITING

    Valencia, 46026, Spain

  • Hôpitaux Universitaires de Marseille Timone

    RECRUITING

    Marseille, 13385, France

  • Institut Gustave-Roussy

    RECRUITING

    Villejuif, 94805, France

  • Institut d Hematologie et d Oncologie Pediatriques

    RECRUITING

    Lyon, 69373, France

  • KK Women's and Children's Hospital

    RECRUITING

    Singapore, 229899, Singapore

  • Levine Children's Hospital- Pediatric Neuro-Oncology

    RECRUITING

    Charlotte, North Carolina, 28203, United States

  • Local Institution - 2102

    COMPLETED

    New York, New York, 10065, United States

  • Local Institution - 2104

    COMPLETED

    Houston, Texas, 77030, United States

  • Local Institution - 2105

    COMPLETED

    Orlando, Florida, 32806, United States

  • Local Institution - 2110

    COMPLETED

    New Brunswick, New Jersey, 08901, United States

  • Local Institution - 2112

    COMPLETED

    Cleveland, Ohio, 44195, United States

  • Local Institution - 2114

    COMPLETED

    Hershey, Pennsylvania, 17033, United States

  • Local Institution - 2120

    COMPLETED

    Orlando, Florida, 32827, United States

  • Local Institution - 4302

    NOT_YET_RECRUITING

    Rome, 00165, Italy

  • Local Institution - 4403

    COMPLETED

    Birmingham, B4 6DH, United Kingdom

  • Local Institution - 6103

    COMPLETED

    Westmead, New South Wales, 0, Australia

  • Local Institution - 6104

    RECRUITING

    Randwick, New South Wales, 2031, Australia

  • Local Institution - 6105

    NOT_YET_RECRUITING

    Barcelona, 08014, Spain

  • Local Institution - 6106

    NOT_YET_RECRUITING

    Madrid, 28009, Spain

  • Local Institution - 6107

    NOT_YET_RECRUITING

    Valencia, 46026, Spain

  • Local Institution - 6108

    NOT_YET_RECRUITING

    Villejuif, 94805, France

  • Local Institution - 6109

    NOT_YET_RECRUITING

    Paris, 75005, France

  • Local Institution - 6110

    NOT_YET_RECRUITING

    Marseille, 13385, France

  • Local Institution - 6111

    NOT_YET_RECRUITING

    Lyon, Rhone, 69008, France

  • Local Institution - 6112

    NOT_YET_RECRUITING

    Nantes, 44093, France

  • Local Institution - 6113

    WITHDRAWN

    Padova, 35128, Italy

  • Local Institution - 6114

    WITHDRAWN

    Torino, 10126, Italy

  • Local Institution - 6202

    COMPLETED

    Taipei, 100225, Taiwan

  • Local Institution - 6303

    COMPLETED

    Seoul, Seodaemun-gu, 03722, South Korea

  • Local Institution - 6304

    COMPLETED

    Seoul, 06351, South Korea

  • Maine Medical Center

    RECRUITING

    Scarborough, Maine, 04074, United States

  • National University Hospital

    RECRUITING

    Singapore, 119228, Singapore

  • Perth Childrens Hospital

    RECRUITING

    Nedlands, Western Australia, 6009, Australia

  • Rigshospitalet - Glostrup

    RECRUITING

    Copenhagen, 2100, Denmark

  • Royal Hosp. for Children

    RECRUITING

    Glasgow, G51 4TF, United Kingdom

  • Seoul National University Hospital

    RECRUITING

    Seoul, 03080, South Korea

  • St Justine Hospital

    RECRUITING

    Montreal, Quebec, H3T 1C5, Canada

  • St. Jude Children's Research Hospital

    RECRUITING

    Memphis, Tennessee, 38015, United States

  • Stollery Children'S Hospital

    RECRUITING

    Edmonton, Alberta, T6G 2B7, Canada

  • Taipei Medical University Hospital

    RECRUITING

    Taipei, 11031, Taiwan

  • The Royal Marsden NHS Foundation Trust

    RECRUITING

    London, SW3 6JJ, United Kingdom

  • The University of Texas Southwestern Medical Center - Harold C Simmons Comprehensive Cancer Center

    RECRUITING

    Dallas, Texas, 75390, United States

  • University Hospital of Wales

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • University Of Calgary

    RECRUITING

    Calgary, Alberta, T3B 6A8, Canada

  • University of California at Los Angeles

    RECRUITING

    Los Angeles, California, 90095, United States

  • Washington University School of Medicine in St. Louis

    RECRUITING

    St Louis, Missouri, 63110, United States

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