Trial reportJournal of clinical oncology : official journal of the American Society of Clinical Oncology2023
Efficacy and Safety of Trametinib Monotherapy or in Combination With Dabrafenib in Pediatric
Trial report in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02124772 (An Open-Label, Dose-Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescents Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Children and Adolescents With Cancers Harboring V600 Mutations), which is not on this map. Cited by 131 papers, 8 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
An Open-Label, Dose-Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescents Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Children and Adolescents With Cancers Harboring V600 Mutations
Who cites it
131 citing papers in PubMed, 8 syntheses or guidelines pooled it, 158 citations in OpenAlex.
- Intratumoral and intracranial hemorrhage associated with MAPK-pathway targeted therapy: a systematic review and mechanistic synthesis.Journal of neuro-oncology · 2026Pooled it
- Basket and umbrella trials in pediatric precision medicine: a systematic review of designs, opportunities, and challenges.BMC medical research methodology · 2026Pooled it
- 2025 American Thyroid Association Management Guidelines for Adult Patients with Differentiated Thyroid Cancer.Thyroid : official journal of the American Thyroid Association · 2025Guideline
- Efficacy and safety of dabrafenib plus trametinib in pediatric versus adult gliomas: a systematic review and meta-analysis.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2025Pooled it
- Trametinib as a targeted treatment in cardiac and lymphatic presentations of Noonan syndrome.Frontiers in pediatrics · 2025Pooled it
- Dabrafenib plus trametinib in low-grade versus high-grade gliomas: a systematic review and meta-analysis.BMC cancer · 2024Pooled it
- The effects of dabrafenib and/or trametinib treatment in Braf V600-mutant glioma: a systematic review and meta-analysis.Neurosurgical review · 2024Pooled it
- The safety and efficacy of dabrafenib and trametinib in patients with glioma: A systematic review and meta-analysis.European journal of clinical pharmacology · 2024Pooled it
- Phase II Study of Dabrafenib and Trametinib in Patients With Tumors WithJCO precision oncology · 2026Trial
- A phase 2 PBTC study of selumetinib for recurrent/progressive pediatric low-grade glioma: Strata 2, 5, and 6 with long-term outcomes on strata 1, 3, and 4.Neuro-oncology · 2025Trial
- Phase II study of vemurafenib in children and young adults with tumors harboring BRAF V600 mutations: NCI-COG pediatric MATCH trial (APEC1621) Arm G.The oncologist · 2024Trial
- The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial.Nature medicine · 2024Trial
- Phase II Trial of Dabrafenib Plus Trametinib in Relapsed/RefractoryJournal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023Trial
- Efficacy and safety of trametinib use in pediatric brain tumors in a low- to middle-income country.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Article
- Suprasellar Desmoplastic Infantile Ganglioglioma in a 7-Month-Old Infant: A Rare Diagnostic Consideration in Infant Suprasellar Masses.Clinical case reports · 2026Article
- Tempered optimism: Advances in the precision medicine era for pediatric low-grade glioma.Neuro-oncology · 2026Review
- Precision Medicine in Dermatology: MEK Inhibition and MAPK Pathway Modulation for Congenital Melanocytic Nevi.Biomedicines · 2026Review
- Postoperative Seizure Prophylaxis in Intracranial Tumors: Evaluating the Role of Anti-Seizure Medication in Seizure-Naïve Patients.Current neurology and neuroscience reports · 2026Review
- Dual targeting of interlocked PTK2B and MAPK signaling triggers synergistic ferroptosis via autophagic flux disruption in BRAFCell communication and signaling : CCS · 2026Article
- Mitochondrial gene expression-based model for the prediction of thyroid cancer prognosis.Endocrine connections · 2026Article
71 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
11 authors at 7 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purpose
methodsThis is a four-part, phase I/II study (ClinicalTrials.gov identifier: NCT02124772) in patients age < 18 years with relapsed/refractory malignancies: trametinib monotherapy dose finding (part A) and disease-specific expansion (part B), and dabrafenib + trametinib dose finding (part C) and disease-specific expansion (part D). The primary objective assessed in all patients in parts A and C was to determine pediatric dosing on the basis of steady-state pharmacokinetics. Disease-specific efficacy and safety (across parts A-D) were secondary objectives.
resultsOverall, 139 patients received trametinib (n = 91) or dabrafenib + trametinib (n = 48). Trametinib dose-limiting toxicities in > 1 patient (part A) included mucosal inflammation (n = 3) and hyponatremia (n = 2). There were no dose-limiting toxicities with combination therapy (part C). The recommended phase II dose of trametinib, with or without dabrafenib, was 0.032 mg/kg once daily for patients age < 6 years and 0.025 mg/kg once daily for patients age ≥ 6 years; dabrafenib dosing in the combination was as previously identified for monotherapy. In 49 patients with
conclusionThe trial design provided efficient evaluation of pediatric dosing, safety, and efficacy of single-agent and combination targeted therapy. Age-based and weight-based dosing of trametinib with or without dabrafenib achieved target concentrations with manageable safety and demonstrated clinical efficacy and tolerability in
Indexed as
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What Socratic holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.