Overview

Eligibility

Design Details

Arms & Interventions

Outcome Measures

Trial Sites

Source: NCT04094610

Recruiting

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

Interventional

Phase 1/2

Baylor College of Medicine

Sponsor: Turning Point Therapeutics

Last Updated: November 19, 2025
Notice- Information is sourced from public registries and may not reflect real-time changes at the local site.

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.

Show Detailed Description

Treatment Category

Targeted Therapy

Required Mutations

ROS1, ALK, NTRK

Trial Started

March 12, 2020

Last Updated by Sponsor

November 19, 2025

Estimated Completion

September 30, 2026

Contact Study

Name:BMS Study Connect Contact Center www.BMSStudyConnect.com

Email:Clinical.Trials@bms.com

Phone:855-907-3286

Name:First line of the email MUST contain the NCT# and Site #.

Eligibility

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.

* 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.

Exclusion Criteria

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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Design Details

AllocationN/A

Intervention ModelSingle Group Assignment

MaskingNone (Open Label)

Number to Enroll75

Arms & Interventions

Arms

Interventions

Type: Experimental

Description: Phase 1 Oral repotrectinib (TPX-0005): Safety and tolerability at different dose levels Phase 2 Oral repotrectinib (TPX-0005): 3 cohorts Cohort 1: TKI-naive NTRK fusion Cohort 2: Prior TKI NTRK fusion Cohort 3: ROS1 gene fusions or other ROS1 aberrations

Interventions:

Oral repotrectinib (TPX-0005)

Outcome Measures

Primary Outcome Measures

Dose limiting toxicities (DLTs) (Phase 1)Within 28 days of the first repotrectinib dose

Define the dose limiting toxicities (DLTs) (Phase 1)

Pediatric Recommended Phase 2 Dose (RP2D) (Phase 1)Within 28 days of the last patient dosed in escalation

To determine the pediatric RP2D (Phase 1)

Overall Response Rate (ORR) (Phase 2)Two to three years after first dose of repotrectinib

To determine the confirmed ORR of repotrectinib (TPX-0005) as assessed by Blinded Independent Central Review (Phase 2)

Secondary Outcome Measures

Overall survival (OS) (Phase 2)Approximately three years

To determine the OS (Phase 2)

Overall Response Rate (ORR) (Phase 1)Approximately three years

To determine the overall response rate (ORR) by Blinded Independent Central Review (BICR) (Phase 1)

Clinical Benefit Rate (CBR) (Phase 1 and Phase 2)Approximately three years

To determine the CBR of repotrectinib (TPX-0005) (Phase 1 and Phase 2)

Time to response (TTR) (Phase 1 and Phase 2)Approximately three years

To determine the TTR of reprotrectinib (TPX-005) (Phase 1 and Phase 2)

Duration of response (DOR) (Phase 1 and Phase 2)Approximately three years

To determine the DOR of repotrectinib (TPX-0005) (Phase 1 and Phase 2)

Intracranial objective response rate (IC-ORR) (Phase 1 and Phase 2)Approximately three years

To determine the IC-ORR of repotrectinib (TPX-005) (Phase 1 and Phase 2)

Central Nervous System Progression-Free Survival (CNS-PFS) (Phase 2)Approximately three years

CNS-PFS in subjects with measurable brain metastases (Phase 2)

Progression-free survival (PFS) (Phase 2)Approximately three years

To determine the PFS (Phase 2)

Maximum concentration of repotrectinib in plasma (Cmax)Pre-dose and up to 24 hours post-dose on Day 1 and Day 15 in Cycle 1 (each cycle is 28 days)

To determine the Cmax

Area under the concentration versus time curve of repotrectinib in plasma (AUC)Pre-dose and up to 24 hours post-dose on Day 1 and Day 15 in Cycle 1 (each cycle is 28 days)

To determine the AUC

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Trial Sites

This study has 52 trial sites

Baylor College of Medicine

Houston, Texas, US

Name:Matthew McEvoy, Site 2118

Recruiting

Houston, Texas, 77030 US

Baylor College of Medicine

Recruiting

Villejuif, 94805 FR

Institut Gustave-Roussy

Recruiting

Los Angeles, California, 90095 US

University of California at Los Angeles

Recruiting

London, Greater London, SW3 6JJ GB

The Royal Marsden Hospital

Recruiting

Singapore, 119228 SG

National University Hospital

Recruiting

Barcelona, Barcelona, 8035 ES

Hospital Universitario Valle de Hebrón

Recruiting

Taipei, 11031 TW

Taipei Medical University Hospital

Recruiting

Singapore, 229899 SG

KK Women's and Children's Hospital

Recruiting

Scarborough, Maine, 04074 US

Maine Medical Center

Recruiting

Cardiff, Wales, CF14 4XN GB

University Hospital of Wales

Recruiting

Sevilla, Sevilla, 41013 ES

Hospital Universitario Virgen del Rocío

Recruiting

València, València, 46026 ES

Hospital Universitario y Politécnico La Fe

Recruiting

Milan, 20133 IT

Fondazione IRCCS - Istituto Nazionale dei Tumori

Recruiting

Boston, Massachusetts, 02215 US

Dana Farber Cancer Institute.

Recruiting

Taipei, 100225 TW

National Taiwan University Hospital

Recruiting

Madrid, Madrid, 28009 ES

Hospital Universitario Infantil Niño Jesús

Recruiting

Madrid, Madrid, 28022 ES

Clínica Universidad de Navarra

Recruiting

Nedlands, Western Australia, 6009 AU

Perth Childrens Hospital

Recruiting

London, Greater London, WC1N 3JH GB

Great Ormond Street Hospital for Children

Recruiting

Ottawa, Ontario, K1H 8L1 CA

Children'S Hospital Of Eastern Ontario

Recruiting

Edmonton, Alberta, T6G 2B7 CA

Stollery Children'S Hospital

Recruiting

Los Angeles, California, 90027-6062 US

Children's Hospital Los Angeles

Recruiting

Aurora, Colorado, 80045 US

Children's Hospital Colorado - Anschutz Medical Campus

Recruiting

Memphis, Tennessee, 38015 US

St. Jude Children's Research Hospital

Recruiting

Dallas, Texas, 75390 US

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

Recruiting

Randwick, New South Wales, 2031 AU

Local Institution - 6104

Not yet recruiting

Lyon, Rhone, 69008 FR

Local Institution - 6111

Not yet recruiting

Nantes, 44093 FR

Local Institution - 6112

Not yet recruiting

Paris, 75005 FR

Local Institution - 6109

Not yet recruiting

Villejuif, 94805 FR

Local Institution - 6108

Not yet recruiting

Rome, 00165 IT

Local Institution - 4302

Not yet recruiting

Barcelona, 08014 ES

Local Institution - 6105

Recruiting

South Brisbane, Queensland, 4101 AU

Children's Health Queensland Hospital and Health Service

Recruiting

Copenhagen, 2100 DK

Rigshospitalet - Glostrup

Recruiting

Calgary, Alberta, T3B 6A8 CA

University Of Calgary

Recruiting

Angers, 49933 FR

Centre Hospitalier Universitaire D'Angers

Recruiting

Madrid, 28050 ES

HM Sanchinarro University Hospital

Recruiting

Liverpool, England, L12 2AP GB

Alder Hey Children's NHS Foundation Trust

Recruiting

Glasgow, G51 4TF GB

Royal Hosp. for Children

Recruiting

Bordeaux, 33076 FR

Centre Hospitalier Universitaire de Bordeaux - Groupe Hospitalier Pellegrin

Recruiting

Esplugues de Llobregat, Barcelona, 08950 ES

Hospital Sant Joan De Deu

Recruiting

Atlanta, Georgia, 30329 US

Children's Healthcare of Atlanta - Egleston Hospital

Recruiting

Philadelphia, Pennsylvania, 19104 US

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

Recruiting

Seoul, 03080 KR

Seoul National University Hospital

Recruiting

Seoul, 05505 KR

Asan Medical Center

Recruiting

St Louis, Missouri, 63110 US

Washington University School of Medicine in St. Louis

Recruiting

Lyon, 69373 FR

Institut d Hematologie et d Oncologie Pediatriques

Recruiting

Marseille, 13385 FR

Hôpitaux Universitaires de Marseille Timone

Not yet recruiting

Marseille, 13385 FR

Local Institution - 6110

Recruiting

Pamplona, Navarre, 31008 ES

Clínica Universidad de navarra

Recruiting

Charlotte, North Carolina, 28203 US

Levine Children's Hospital- Pediatric Neuro-Oncology

Recruiting

Montreal, Quebec, H3T 1C5 CA

St Justine Hospital

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