NCT04094610From ClinicalTrials.govRecruiting

A Study of Repotrectinib in Pediatric and Young Adult Subjects Harboring ALK, ROS1, OR NTRK1-3 Alterations

A Phase 1/2, Open-label, Safety, Tolerability, Pharmacokinetics, and Anti-tumor Activity Study of Repotrectinib in Pediatric and Young Adult Subjects With Advanced or Metastatic Malignancies Harboring ALK, ROS1, or NTRK1-3 Alterations (CARE)

Locally Advanced Solid Tumors
Metastatic Solid Tumors
Lymphoma
Primary CNS Tumors

At a glance

Phase: Phase 1
Study type: Interventional
Sponsor: Turning Point Therapeutics, Inc.
Enrolment target: 75
Started: 12 March 2020
Main results due: 30 September 2026
Study sites: 68
Registry updated: 25 September 2026

Can you take part?

Up to 25 years.
Open to any sex.
You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full eligibility criteria

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.

What this study is about

In the sponsor’s own words, from the registry.

Phase 1 will evaluate the safety and tolerability at different dose levels of repotrectinib in pediatric and young adult subjects with advanced or metastatic malignancies harboring anaplastic lymphoma kinase (ALK), receptor tyrosine kinase encoded by the gene ROS1 (ROS1), or neurotrophic receptor kinase genes encoding TRK kinase family (NTRK1-3) alterations to estimate the Maximum Tolerated Dose (MTD) or Maximum Administered Dose (MAD) and select the Pediatric Recommended Phase 2 Dose (RP2D).

Phase 2 will determine the anti-tumor activity of repotrectinib in pediatric and young adult subjects with advanced or metastatic malignancies harboring ROS1 or NTRK1-3 alterations.

Study sites(68)

Children's Hospital Los Angeles
Los Angeles, California, United States
Recruiting
University of California at Los Angeles
Los Angeles, California, United States
Recruiting
Children's Hospital Colorado - Anschutz Medical Campus
Aurora, Colorado, United States
Recruiting
Local Institution - 2105
Orlando, Florida, United States
Completed
Local Institution - 2120
Orlando, Florida, United States
Completed
Children's Healthcare of Atlanta - Egleston Hospital
Atlanta, Georgia, United States
Recruiting
Maine Medical Center
Scarborough, Maine, United States
Recruiting
Dana Farber Cancer Institute.
Boston, Massachusetts, United States
Recruiting
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States
Recruiting
Local Institution - 2110
New Brunswick, New Jersey, United States
Completed
Local Institution - 2102
New York, New York, United States
Completed
Levine Children's Hospital- Pediatric Neuro-Oncology
Charlotte, North Carolina, United States
Recruiting
Local Institution - 2112
Cleveland, Ohio, United States
Completed
Local Institution - 2114
Hershey, Pennsylvania, United States
Completed
Children's Hospital of Philadelphia-Center for Childhood Cancer Research
Philadelphia, Pennsylvania, United States
Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
Recruiting
The University of Texas Southwestern Medical Center - Harold C Simmons Comprehensive Cancer Center
Dallas, Texas, United States
Recruiting
Baylor College of Medicine
Houston, Texas, United States
Recruiting
Local Institution - 2104
Houston, Texas, United States
Completed
Children's Hospital of Richmond at VCU
Richmond, Virginia, United States
Withdrawn
Local Institution - 6104
Randwick, New South Wales, Australia
Recruiting
Local Institution - 6103
Westmead, New South Wales, Australia
Completed
Children's Health Queensland Hospital and Health Service
South Brisbane, Queensland, Australia
Recruiting
Perth Childrens Hospital
Nedlands, Western Australia, Australia
Recruiting
University Of Calgary
Calgary, Alberta, Canada
Recruiting
Stollery Children'S Hospital
Edmonton, Alberta, Canada
Recruiting
Children'S Hospital Of Eastern Ontario
Ottawa, Ontario, Canada
Recruiting
St Justine Hospital
Montreal, Quebec, Canada
Recruiting
Rigshospitalet - Glostrup
Copenhagen, Denmark
Recruiting
Local Institution - 6111
Lyon, Rhone, France
Not yet recruiting
Centre Hospitalier Universitaire D'Angers
Angers, France
Recruiting
Centre Hospitalier Universitaire de Bordeaux - Groupe Hospitalier Pellegrin
Bordeaux, France
Recruiting
Institut d Hematologie et d Oncologie Pediatriques
Lyon, France
Recruiting
Hôpitaux Universitaires de Marseille Timone
Marseille, France
Recruiting
Local Institution - 6110
Marseille, France
Not yet recruiting
Local Institution - 6112
Nantes, France
Not yet recruiting
Local Institution - 6109
Paris, France
Not yet recruiting
Institut Gustave-Roussy
Villejuif, France
Recruiting
Local Institution - 6108
Villejuif, France
Not yet recruiting
Fondazione IRCCS - Istituto Nazionale dei Tumori
Milan, Italy
Recruiting
Local Institution - 6113
Padova, Italy
Withdrawn
Local Institution - 4302
Rome, Italy
Not yet recruiting
Local Institution - 6114
Torino, Italy
Withdrawn
National University Hospital
Singapore, Singapore
Recruiting
KK Women's and Children's Hospital
Singapore, Singapore
Recruiting
Local Institution - 6303
Seoul, Seodaemun-gu, South Korea
Completed
Seoul National University Hospital
Seoul, South Korea
Recruiting
Asan Medical Center
Seoul, South Korea
Recruiting
Local Institution - 6304
Seoul, South Korea
Completed
Hospital Sant Joan De Deu
Esplugues de Llobregat, Barcelona, Spain
Recruiting
Clínica Universidad de navarra
Pamplona, Navarre, Spain
Recruiting
Local Institution - 6105
Barcelona, Spain
Not yet recruiting
Hospital Universitari Vall d'Hebron
Barcelona, Spain
Recruiting
Hospital Infantil Universitario Nino Jesus
Madrid, Spain
Recruiting
Local Institution - 6106
Madrid, Spain
Not yet recruiting
Clinica Universidad de Navarra
Madrid, Spain
Recruiting
HM Sanchinarro University Hospital
Madrid, Spain
Recruiting
Hospital Universitario Virgen del Rocio
Seville, Spain
Recruiting
Hospital Universitario Y Politecnico La Fe
Valencia, Spain
Recruiting
Local Institution - 6107
Valencia, Spain
Not yet recruiting

Showing 60 of 68 sites — filter to narrow it down. 44 of the 68 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT04094610. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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