Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options
NCT ID: NCT02650401
Last Updated: 2025-12-24
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
69 participants
INTERVENTIONAL
2016-05-03
2026-06-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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CNS tumors harboring- NTRK1/2/3, ROS1, ALK
Arm closed for further enrollment
molecular alterations, including gene fusions
Oral entrectinib (RXDX-101)
Entrectinib
TRKA/B/C, ROS1, and ALK inhibitor
Neuroblastoma
Arm closed for further enrollment
Oral entrectinib (RXDX-101)
Entrectinib
TRKA/B/C, ROS1, and ALK inhibitor
Non-neuroblastoma, extracranial solid tumors
Arm closed for further enrollment
harboring - NTRK1/2/3, ROS1, ALK gene fusions
Oral entrectinib (RXDX-101)
Entrectinib
TRKA/B/C, ROS1, and ALK inhibitor
Any participant unable to swallow capsules
Arm closed for further enrollment
Any participant who otherwise meet all other eligibility criteria
Oral entrectinib (RXDX-101)
Entrectinib
TRKA/B/C, ROS1, and ALK inhibitor
Expansion: CNS tumors harboring NTRK1/2/3, ROS1
gene fusions
Oral entrectinib (RXDX-101)
Entrectinib
TRKA/B/C, ROS1, and ALK inhibitor
Expansion: Extracranial solid tumors harboring NTRK1/2/3, ROS1
NTRK 1,2,3 and ROS1 fusions
Oral entrectinib (RXDX-101)
Entrectinib
TRKA/B/C, ROS1, and ALK inhibitor
Extracranial solid tumors harboring NTRK1/2/3,
Arm closed for further enrollment
ROS1, ALK non-gene fusion molecular alterations
Oral entrectinib (RXDX-101)
Entrectinib
TRKA/B/C, ROS1, and ALK inhibitor
Interventions
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Entrectinib
TRKA/B/C, ROS1, and ALK inhibitor
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Phase 1 portion (closed): Participants must have measurable or evaluable disease, as defined by RECIST v1.1
* Phase 2 portion:
* Part B: Participants must have measurable or evaluable disease, as defined by RANO
* Part C (closed): Participants must have measurable or evaluable disease, as defined by RECIST v1.1 ± Curie Scale
* Part D: Participants must have measurable or evaluable disease, as defined by RECIST v1.1
* Part E (closed): Participants must have measurable or evaluable disease, as defined by RECIST v1.1 ± Curie Scale or RANO
2. Tumor type:
* Phase 1 portion:
\* Part A: Relapsed or refractory extracranial solid tumors
* Phase 2 portion
* Part B: Primary brain tumors with NTRK1/2/3 or ROS1 gene fusions; gene fusions are defined as those predicted to translate into a fusion protein with a functional TRKA/B/C or ROS1 kinase domain, without a concomitant second oncodriver as determined by a nucleic acid-based diagnostic testing method
* Part D: Extracranial solid tumors (including NB) with NTRK1/2/3 or ROS1 gene fusions; gene fusions are defined as those predicted to translate into a fusion protein with a functional TRKA/B/C or ROS1 kinase domain, without a concomitant second oncodriver as determined by a nucleic acid-based diagnostic testing method
3. Histologic/molecular diagnosis of malignancy at diagnosis or the time of relapse
4. Archival tumor tissue from diagnosis or, preferably, at relapse
5. Performance status: Lansky or Karnofsky score ≥ 60% and minimum life expectancy of at least 4 weeks
6. Prior therapy: Participants must have a disease that is locally advanced, metastatic, or where surgical resection is likely to result in severe morbidity, and who have no satisfactory treatment options for solid tumors and primary CNS tumors that are neurotrophic tyrosine receptor kinase (NTRK) or ROS1 fusion-positive
7. Participants must have recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to enrollment
8. Adequate organ and neurologic function
9. Females of childbearing potential must have a negative serum pregnancy test during screening and be neither breastfeeding nor intending to become pregnant during study participation. Agreement to remain abstinent or use use combined contraceptive methods prior to study entry, for the duration of study participation and in the following 90 days after discontinuation of study treatment.
10. For male participants with a female partner of childbearing potential or a pregnant female partner: Agreement to remain abstinent or use a condom during the treatment period and for at least 3 months after the last dose of study drug
Exclusion Criteria
2. Known congenital long QT syndrome
3. History of recent (3 months) symptomatic congestive heart failure or ejection fraction ≤50% at screening
4. Known active infections
5. Familial or personal history of congenital bone disorders, bone metabolism alterations or osteopenia
6. Receiving Enzyme Inducing Antiepileptic Drugs (EIAEDs) within 14 days of first dose.
7. Prior treatment with approved or investigational TRK or ROS1 inhibitors
8. Known hypersensitivity to entrectinib or any of the other excipients of the investigational medicinal product
9. Patients with NB with bone marrow space-only disease
10. Incomplete recovery from acute effects of any surgery prior to treatment.
11. Active gastrointestinal disease or other malabsorption syndromes that would impact drug absorption.
12. Other severe acute or chronic medical or psychiatric condition or lab abnormality that may increase the risk associated with study participation, drug administration or may interfere with the interpretation of study results.
0 Years
18 Years
ALL
No
Sponsors
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Hoffmann-La Roche
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trials
Role: STUDY_DIRECTOR
Hoffmann-La Roche
Locations
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University of California San Diego
La Jolla, California, United States
UCSF Benioff Children's Hospital
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Egleston Children's Hospital at Emory University Atlanta
Atlanta, Georgia, United States
University of Chicago
Chicago, Illinois, United States
Johns Hopkins University
Baltimore, Maryland, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
Washington University,St. Louis Children's Hospital
St Louis, Missouri, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Oregon Health & Science Uni
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
St. Jude Children'S Research Hospital
Memphis, Tennessee, United States
Texas Children's Cancer and Hematology Center
Houston, Texas, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
The Hospital for Sick Children
Toronto, Ontario, Canada
Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
Beijing Children's Hospital, Capital Medical University
Beijing, , China
Centre Leon Berard
Lyon, , France
Hôpital de la Timone, Oncologie Pédiatrique
Marseille, , France
Universitaetsklinikum Heidelberg
Heidelberg, Baden-Wurttemberg, Germany
Hong Kong Children's Hospital
Hong Kong, , Hong Kong
Fondazione IRCCS Istituto Nazionale dei Tumori
Milan, Lombardy, Italy
Hospital Infantil Universitario Nino Jesus
Madrid, , Spain
Royal Marsden Hospital (Sutton)
London, , United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne, , United Kingdom
Countries
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References
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Desai AV, Bagchi A, Armstrong AE, van Tilburg CM, Basu EM, Robinson GW, Wang H, Casanova M, Andre N, Campbell-Hewson Q, Wu Y, Cardenas A, Ci B, Ryklansky C, Devlin CE, Meneses-Lorente G, Wulff J, Hutchinson KE, Gajjar A, Fox E. Efficacy and safety of entrectinib in children with extracranial solid or central nervous system (CNS) tumours harbouring NTRK or ROS1 fusions. Eur J Cancer. 2025 May 2;220:115308. doi: 10.1016/j.ejca.2025.115308. Epub 2025 Feb 22.
Desai AV, Robinson GW, Gauvain K, Basu EM, Macy ME, Maese L, Whipple NS, Sabnis AJ, Foster JH, Shusterman S, Yoon J, Weiss BD, Abdelbaki MS, Armstrong AE, Cash T, Pratilas CA, Corradini N, Marshall LV, Farid-Kapadia M, Chohan S, Devlin C, Meneses-Lorente G, Cardenas A, Hutchinson KE, Bergthold G, Caron H, Chow Maneval E, Gajjar A, Fox E. Entrectinib in children and young adults with solid or primary CNS tumors harboring NTRK, ROS1, or ALK aberrations (STARTRK-NG). Neuro Oncol. 2022 Oct 3;24(10):1776-1789. doi: 10.1093/neuonc/noac087.
Doebele RC, Drilon A, Paz-Ares L, Siena S, Shaw AT, Farago AF, Blakely CM, Seto T, Cho BC, Tosi D, Besse B, Chawla SP, Bazhenova L, Krauss JC, Chae YK, Barve M, Garrido-Laguna I, Liu SV, Conkling P, John T, Fakih M, Sigal D, Loong HH, Buchschacher GL Jr, Garrido P, Nieva J, Steuer C, Overbeck TR, Bowles DW, Fox E, Riehl T, Chow-Maneval E, Simmons B, Cui N, Johnson A, Eng S, Wilson TR, Demetri GD; trial investigators. Entrectinib in patients with advanced or metastatic NTRK fusion-positive solid tumours: integrated analysis of three phase 1-2 trials. Lancet Oncol. 2020 Feb;21(2):271-282. doi: 10.1016/S1470-2045(19)30691-6. Epub 2019 Dec 11.
Other Identifiers
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CO40778
Identifier Type: OTHER
Identifier Source: secondary_id
2023-505088-35-00
Identifier Type: CTIS
Identifier Source: secondary_id
RXDX-101-03
Identifier Type: -
Identifier Source: org_study_id