TriangleNCT02650401 · CITED IN SOURCE DOCUMENTS
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
An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.
- Phase
- PHASE1, PHASE2
- Status at capture
- ACTIVE NOT RECRUITING
- Registry last update
- 2026-06-23
This is an open-label, Phase 1/2 multicenter dose escalation study in pediatric patients with relapsed or refractory extracranial solid tumors (Phase 1), with additional expansion cohorts (Phase 2) in patients with primary brain tumors harboring NTRK1/2/3 or ROS1 gene fusions, and extracranial solid tumors harboring NTRK1/2/3 or ROS1 gene fusions.
Open the original ClinicalTrials.gov record → · Download preserved record
What did the study report?
Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.
No posted result sections were captured. Registered plans do not establish that a treatment works.
Who could take part
- eligibility Criteria
- Inclusion Criteria: 1. Disease status: * 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: 1. Receiving other experimental therapy 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.
- healthy Volunteers
- false
- maximum Age
- 18 Years
- minimum Age
- 0 Years
- sex
- ALL
- std Ages
- CHILD
- ADULT
Treatment arms and interventions
- arm Groups
- description
- Arm closed for further enrollment ROS1, ALK non-gene fusion molecular alterations Oral entrectinib (RXDX-101)
- intervention Names
- Drug: Entrectinib
- label
- Extracranial solid tumors harboring NTRK1/2/3,
- type
- ACTIVE_COMPARATOR
- description
- Arm closed for further enrollment molecular alterations, including gene fusions Oral entrectinib (RXDX-101)
- intervention Names
- Drug: Entrectinib
- label
- CNS tumors harboring- NTRK1/2/3, ROS1, ALK
- type
- ACTIVE_COMPARATOR
- description
- Arm closed for further enrollment Oral entrectinib (RXDX-101)
- intervention Names
- Drug: Entrectinib
- label
- Neuroblastoma
- type
- ACTIVE_COMPARATOR
- description
- Arm closed for further enrollment harboring - NTRK1/2/3, ROS1, ALK gene fusions Oral entrectinib (RXDX-101)
- intervention Names
- Drug: Entrectinib
- label
- Non-neuroblastoma, extracranial solid tumors
- type
- ACTIVE_COMPARATOR
- description
- Arm closed for further enrollment Any participant who otherwise meet all other eligibility criteria Oral entrectinib (RXDX-101)
- intervention Names
- Drug: Entrectinib
- label
- Any participant unable to swallow capsules
- type
- ACTIVE_COMPARATOR
- description
- gene fusions Oral entrectinib (RXDX-101)
- intervention Names
- Drug: Entrectinib
- label
- Expansion: CNS tumors harboring NTRK1/2/3, ROS1
- type
- ACTIVE_COMPARATOR
- description
- NTRK 1,2,3 and ROS1 fusions Oral entrectinib (RXDX-101)
- intervention Names
- Drug: Entrectinib
- label
- Expansion: Extracranial solid tumors harboring NTRK1/2/3, ROS1
- type
- ACTIVE_COMPARATOR
- interventions
- arm Group Labels
- Any participant unable to swallow capsules
- CNS tumors harboring- NTRK1/2/3, ROS1, ALK
- Expansion: CNS tumors harboring NTRK1/2/3, ROS1
- Expansion: Extracranial solid tumors harboring NTRK1/2/3, ROS1
- Extracranial solid tumors harboring NTRK1/2/3,
- Neuroblastoma
- Non-neuroblastoma, extracranial solid tumors
- description
- TRKA/B/C, ROS1, and ALK inhibitor
- name
- Entrectinib
- other Names
- RXDX-101
- type
- DRUG
Study design
- allocation
- NON_RANDOMIZED
- intervention Model
- SINGLE_GROUP
- masking Info
- masking
- NONE
- primary Purpose
- TREATMENT
Enrollment
- count
- 69
- type
- ACTUAL
Registered outcome plans (not posted results)
- primary Outcomes
- description
- Assessed by National Cancer Institute Common Terminology for Adverse Events Criteria (NCI CTCAE v4.03)
- measure
- Maximum Tolerated Dose (MTD)
- time Frame
- Approximately 6 months
- description
- Assessed by NCI CTCAE v4.03
- measure
- Recommended Phase 2 Dose (RP2D) of F1 Formulation In Pediatric Participants Able To Swallow Intact Capsules
- time Frame
- Approximately 6 months
- description
- Assessed by NCI CTCAE v4.03
- measure
- Recommended Phase 2 Dose (RP2D) of F06 Formulation In Pediatric Participants Able To Swallow Intact Capsules
- time Frame
- Approximately 6 months
- description
- Assessed by NCI CTCAE v4.03
- measure
- Recommended Phase 2 Dose (RP2D) of F06 Formulation In Pediatric In Participants Dosed Via Feeding Tube (Nasogastric Tube Or Gastric Tube)
- time Frame
- Approximately 6 months
- description
- Assessed by NCI CTCAE v4.03
- measure
- Recommended Phase 2 Dose (RP2D) Of Minitablets/F15 Formulation In Pediatric Participants Unable To Swallow Intact Capsules
- time Frame
- Approximately 6 months
- description
- Assessed by RANO per the BICR
- measure
- Cohort B: Objective Response Rate (ORR)
- time Frame
- Approximately 6 months
- description
- Assessed by RECIST v1.1 per the BICR
- measure
- Cohort D: ORR
- time Frame
- Approximately 6 months
- secondary Outcomes
- description
- AE, ECG and Labs assessed by NCI CTCAE v4.03
- measure
- Safety and Tolerability - AE, ECG and Labs assessed by NCI CTCAE v4.03
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- Maximum observed plasma drug concentration (Cmax) using F1 Formulation
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- Maximum observed plasma drug concentration (Cmax) using F06 Formulation given intact
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- Maximum observed plasma drug concentration (Cmax) using F06 Formulation administered via feeding tube
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- Maximum observed plasma drug concentration (Cmax) using minitablets/F15
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- Time to Cmax, by inspection (Tmax) using F1 Formulation
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- Time to Cmax, by inspection (Tmax) using F06 Formulation given intact
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- Time to Cmax, by inspection (Tmax) using F06 Formulation administered via feeding tube
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- Time to Cmax, by inspection (Tmax) using minitablets/F15
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- AUC at steady state (AUCss) using F1 Formulation
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- AUC at steady state (AUCss) using F06 Formulation given intact
- time Frame
- Approximately 24 months
- description
- Assessed by plasma concentrations obtained on Days 1, 2, 8, 15, 22 (Cycle 1), Days 1, 2 (Cycle 2) and on Day 1 of every cycle thereafter
- measure
- AUC at steady state (AUCss) using F06 Formulation administered via feeding tube
- time Frame
- Approximately 24 months
Full study description
- brief Summary
- This is an open-label, Phase 1/2 multicenter dose escalation study in pediatric patients with relapsed or refractory extracranial solid tumors (Phase 1), with additional expansion cohorts (Phase 2) in patients with primary brain tumors harboring NTRK1/2/3 or ROS1 gene fusions, and extracranial solid tumors harboring NTRK1/2/3 or ROS1 gene fusions.
Source references
- references
- citation
- 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.
- pmid
- 40086048
- type
- DERIVED
- citation
- 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.
- pmid
- 35395680
- type
- DERIVED
- citation
- 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.
- pmid
- 31838007
- type
- DERIVED
Source notices and limitations
Discovery and provenance
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- atric patients with unresectable or metastatic solid tumors with a NTRK gene fusion enrolled in one of two multicenter, open-label clinical trials: STARTRK-NG (NCT02650401) and TAPISTRY (NCT04589845). To be included in the analysis, patients were required to have received at least 1 dose of ROZLYTREK; measurable or evaluable dise
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- Updated: February 12, 2025
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- February 12, 2025
Preserved source evidence · Independent clinical review pending · Not medical advice