RECRUITING Phase 1 / Phase 2 ClinicalTrials.gov

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

Sponsor: Turning Point Therapeutics, Inc. • ClinicalTrials.gov ID: NCT04094610

Registry updated: 2026-09-25

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Description

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.

Interventions

Oral repotrectinib (TPX-0005) (Drug)
Oral repotrectinib (TPX-0005)

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.

Conditions

Locally Advanced Solid Tumors Metastatic Solid Tumors Lymphoma Primary CNS Tumors

Locations

Children's Hospital Los Angeles — Los Angeles, California, United States
University of California at Los Angeles — Los Angeles, California, United States
Children's Hospital Colorado - Anschutz Medical Campus — Aurora, Colorado, United States
Local Institution - 2105 — Orlando, Florida, United States
Local Institution - 2120 — Orlando, Florida, United States
Children's Healthcare of Atlanta - Egleston Hospital — Atlanta, Georgia, United States
Maine Medical Center — Scarborough, Maine, United States
Dana Farber Cancer Institute. — Boston, Massachusetts, United States
Washington University School of Medicine in St. Louis — St Louis, Missouri, United States
Local Institution - 2110 — New Brunswick, New Jersey, United States
Local Institution - 2102 — New York, New York, United States
Levine Children's Hospital- Pediatric Neuro-Oncology — Charlotte, North Carolina, United States
Local Institution - 2112 — Cleveland, Ohio, United States
Local Institution - 2114 — Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia-Center for Childhood Cancer Research — Philadelphia, Pennsylvania, United States
St. Jude Children's Research Hospital — Memphis, Tennessee, United States
The University of Texas Southwestern Medical Center - Harold C Simmons Comprehensive Cancer Center — Dallas, Texas, United States
Baylor College of Medicine — Houston, Texas, United States
Local Institution - 2104 — Houston, Texas, United States
Children's Hospital of Richmond at VCU — Richmond, Virginia, United States
Local Institution - 6104 — Randwick, New South Wales, Australia
Local Institution - 6103 — Westmead, New South Wales, Australia
Children's Health Queensland Hospital and Health Service — South Brisbane, Queensland, Australia
Perth Childrens Hospital — Nedlands, Western Australia, Australia
University Of Calgary — Calgary, Alberta, Canada
Stollery Children'S Hospital — Edmonton, Alberta, Canada
Children'S Hospital Of Eastern Ontario — Ottawa, Ontario, Canada
St Justine Hospital — Montreal, Quebec, Canada
Rigshospitalet - Glostrup — Copenhagen, Denmark
Local Institution - 6111 — Lyon, Rhone, France
Centre Hospitalier Universitaire D'Angers — Angers, France
Centre Hospitalier Universitaire de Bordeaux - Groupe Hospitalier Pellegrin — Bordeaux, France
Institut d Hematologie et d Oncologie Pediatriques — Lyon, France
Hôpitaux Universitaires de Marseille Timone — Marseille, France
Local Institution - 6110 — Marseille, France
Local Institution - 6112 — Nantes, France
Local Institution - 6109 — Paris, France
Institut Gustave-Roussy — Villejuif, France
Local Institution - 6108 — Villejuif, France
Fondazione IRCCS - Istituto Nazionale dei Tumori — Milan, Italy
Local Institution - 6113 — Padova, Italy
Local Institution - 4302 — Rome, Italy
Local Institution - 6114 — Torino, Italy
National University Hospital — Singapore, Singapore
KK Women's and Children's Hospital — Singapore, Singapore
Local Institution - 6303 — Seoul, Seodaemun-gu, South Korea
Seoul National University Hospital — Seoul, South Korea
Asan Medical Center — Seoul, South Korea
Local Institution - 6304 — Seoul, South Korea
Hospital Sant Joan De Deu — Esplugues de Llobregat, Barcelona, Spain
Clínica Universidad de navarra — Pamplona, Navarre, Spain
Local Institution - 6105 — Barcelona, Spain
Hospital Universitari Vall d'Hebron — Barcelona, Spain
Hospital Infantil Universitario Nino Jesus — Madrid, Spain
Local Institution - 6106 — Madrid, Spain
Clinica Universidad de Navarra — Madrid, Spain
HM Sanchinarro University Hospital — Madrid, Spain
Hospital Universitario Virgen del Rocio — Seville, Spain
Hospital Universitario Y Politecnico La Fe — Valencia, Spain
Local Institution - 6107 — Valencia, Spain
Local Institution - 6202 — Taipei, Taiwan
Taipei Medical University Hospital — Taipei, Taiwan
Alder Hey Children's NHS Foundation Trust — Liverpool, England, United Kingdom
Local Institution - 4403 — Birmingham, United Kingdom
University Hospital of Wales — Cardiff, United Kingdom
Royal Hosp. for Children — Glasgow, United Kingdom
The Royal Marsden NHS Foundation Trust — London, United Kingdom
Great Ormond Street Hospital For Children NHS Foundation Trust — London, United Kingdom

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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