Trial reportNeuro-oncology2026
TRAM-01: A phase 2 study of trametinib for pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas.
Trial report in Neuro-oncology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.
What it found
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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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
5 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- Article
- TEAD inhibitors re-sensitize drug-resistant NF1 MPNST cells to MEK inhibitors.microPublication biology · 2026Article
- TheCancers · 2024Review
- Measuring Safety and Outcomes for the Use of Compassionate and Off-Label Therapies for Children, Adolescents, and Young Adults With Cancer in the SACHA-France Study.JAMA network open · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
35 authors.
Funding
Abstract
backgroundPlexiform neurofibromas (PNs) are observed in up to 50% of patients with neurofibromatosis type 1 (NF1). Trametinib has been used widely to treat PNs but limited data has been reported on its efficacy within a clinical trial. We conducted a phase 2 trial with trametinib for patients with NF1 and inoperable PNs.
methodsPatients with NF1 and inoperable PNs aged ≥1 month to ≤25 years when starting trametinib were eligible. Patients received trametinib once daily orally continuously for a maximum of 18 cycles of 28 days. Patients completed patient-reported outcome measures. Response evaluations were performed after cycle 3, 6, 12 and at the end of treatment and then every 6 months. The PN volumes were centrally quantified using a semi-automatic 3D segmentation method.
resultsForty-six patients were recruited. The median age was 11.1 years (range 0.7-19.8). The median baseline tumor volume was 104.6 ml (range 16.3 to 650.1 ml). Volumetric assessment demonstrated an overall response rate (volume decrease of ≥20%) in 47.8% (22/46) of patients. Median volume change was -19.3% (range -80.5 to 3.5). Forty-one patients (89.1%) completed the planned treatment. The 3 years progression free survival and the event free survival were respectively 73.1% (95% CI 53.3%-85.5%) and 47.1% (95% CI 28.8-63.5).
conclusionsWe report outcomes and volumetric response of PNs treated with trametinib within a phase 2 trial. Based on these results, trametinib is effective and overall well tolerated.
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