Vorasidenib Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated:

The clinical evidence base for Vorasidenib comprises 12 peer-reviewed publications across 11 journals, 1 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Glioma. Most recent publication: Vorasidenib in IDH1-mutant or IDH2-mutant low-grade glioma (INDIGO): secondary and exploratory endpoints from a randomised, double-blind, placebo-controlled, phase 3 trial., Lancet Oncol, 2025.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

  1. Vorasidenib in IDH1- or IDH2-Mutant Low-Grade Glioma.
    Mellinghoff IK, van den Bent MJ, Blumenthal DT, et al. · N Engl J Med · 2023
  2. Vorasidenib in IDH1-mutant or IDH2-mutant low-grade glioma (INDIGO): secondary and exploratory endpoints from a randomised, double-blind, placebo-controlled, phase 3 trial.
    Cloughesy TF, van den Bent MJ, Touat M, et al. · Lancet Oncol · 2025
  3. Long-term efficacy of early versus delayed radiotherapy for low-grade astrocytoma and oligodendroglioma in adults: the EORTC 22845 randomised trial.
    van den Bent MJ, Afra D, de Witte O, et al. · Lancet · 2005
  4. New perspectives: Glioma in adult patients.
    Silvani A · Tumori · 2023
  5. Evaluation of early metabolic changes following vorasidenib using FET PET in patients with IDH-mutant gliomas.
    Galldiks N, Werner JM, Stetter I, et al. · Neurooncol Adv · 2025
  6. The role of patient-reported outcome measures in the continuum of cancer clinical care: ESMO Clinical Practice Guideline.
    Di Maio M, Basch E, Denis F, et al. · Ann Oncol · 2022
  7. Guidelines for Inclusion of Patient-Reported Outcomes in Clinical Trial Protocols: The SPIRIT-PRO Extension.
    Calvert M, Kyte D, Mercieca-Bebber R, et al. · JAMA · 2018
  8. Patient-Reported Outcome Measures in Cancer Care: An Updated Systematic Review and Meta-Analysis.
    Balitsky AK, Rayner D, Britto J, et al. · JAMA Netw Open · 2024

Primary-outcome results across pivotal trials

Per-arm reported values from Phase 2/3 and Phase 3 trials with results posted to ClinicalTrials.gov.

TrialIndicationPrimary endpointArmValue
NCT04164901GliomaProgression-Free Survival (PFS)
Up to approximately 30 months
Matching Placebo11.1 months
Vorasidenib27.7 months

Publications by year

20052025: 12 publications.

2005
1
2018
1
2019
1
2020
1
2021
2
2022
1
2023
2
2024
1
2025
2

Publications by indication

Glioma (12)

  • Vorasidenib in IDH1-mutant or IDH2-mutant low-grade glioma (INDIGO): secondary and exploratory endpoints from a randomised, double-blind, placebo-controlled, phase 3 trial.
    Lancet Oncol · 2025 · PMID 41175888 · NCT04164901
  • Evaluation of early metabolic changes following vorasidenib using FET PET in patients with IDH-mutant gliomas.
    Neurooncol Adv · 2025 · PMID 39737092 · NCT07469735
  • Patient-Reported Outcome Measures in Cancer Care: An Updated Systematic Review and Meta-Analysis.
    JAMA Netw Open · 2024 · PMID 39136947 · NCT07547163
  • Vorasidenib in IDH1- or IDH2-Mutant Low-Grade Glioma.
    N Engl J Med · 2023 · PMID 37272516 · NCT04164901
  • New perspectives: Glioma in adult patients.
    Tumori · 2023 · PMID 36964665 · NCT07547163

Publications by journal

Trial-results highlights

Vorasidenib has been investigated in clinical trials for its potential use in treating glioma. A pivotal study, NCT04164901, focused on patients with glioma and evaluated a key measure known as Progression-Free Survival (PFS). PFS is an important endpoint in cancer research, reflecting the period during and after treatment when a patient lives without their disease worsening or without death. In this particular trial, the observation period for PFS extended up to approximately 30 months.

Within the NCT04164901 study, specific median Progression-Free Survival times were reported for the different treatment groups. Patients who received vorasidenib experienced a median PFS of 27.7 months. In comparison, patients in the placebo arm of the same NCT04164901 trial had a median PFS of 11.1 months.

All values presented are sourced from primary registry reporting. For comprehensive clinical information and informed decision-making, individual published papers should be consulted.

All Vorasidenib publications (12)

2025 (2 papers)

  1. Vorasidenib in IDH1-mutant or IDH2-mutant low-grade glioma (INDIGO): secondary and exploratory endpoints from a randomised, double-blind, placebo-controlled, phase 3 trial.
    Cloughesy TF, van den Bent MJ, Touat M, et al. · Lancet Oncol · 2025 · Derived
  2. Evaluation of early metabolic changes following vorasidenib using FET PET in patients with IDH-mutant gliomas.
    Galldiks N, Werner JM, Stetter I, et al. · Neurooncol Adv · 2025 · Background

2024 (1 paper)

  1. Patient-Reported Outcome Measures in Cancer Care: An Updated Systematic Review and Meta-Analysis.
    Balitsky AK, Rayner D, Britto J, et al. · JAMA Netw Open · 2024 · Background

2023 (2 papers)

  1. Vorasidenib in IDH1- or IDH2-Mutant Low-Grade Glioma.
    Mellinghoff IK, van den Bent MJ, Blumenthal DT, et al. · N Engl J Med · 2023 · Trial result
  2. New perspectives: Glioma in adult patients.
    Silvani A · Tumori · 2023 · Background

2022 (1 paper)

  1. The role of patient-reported outcome measures in the continuum of cancer clinical care: ESMO Clinical Practice Guideline.
    Di Maio M, Basch E, Denis F, et al. · Ann Oncol · 2022 · Background

2021 (2 papers)

  1. The 2021 WHO Classification of Tumors of the Central Nervous System: a summary.
    Louis DN, Perry A, Wesseling P, et al. · Neuro Oncol · 2021 · Background
  2. Comprehensive Genomic Analysis in NRG Oncology/RTOG 9802: A Phase III Trial of Radiation Versus Radiation Plus Procarbazine, Lomustine (CCNU), and Vincristine in High-Risk Low-Grade Glioma.
    Bell EH, Zhang P, Shaw EG, et al. · J Clin Oncol · 2021 · Background

2020 (1 paper)

  1. Developing a Quality of Cancer Survivorship Care Framework: Implications for Clinical Care, Research, and Policy.
    Nekhlyudov L, Mollica MA, Jacobsen PB, et al. · J Natl Cancer Inst · 2020 · Background

2019 (1 paper)

  1. Comparative Assessment of Clinical Benefit Using the ESMO-Magnitude of Clinical Benefit Scale Version 1.1 and the ASCO Value Framework Net Health Benefit Score.
    Cherny NI, de Vries EGE, Dafni U, et al. · J Clin Oncol · 2019 · Background

2018 (1 paper)

  1. Guidelines for Inclusion of Patient-Reported Outcomes in Clinical Trial Protocols: The SPIRIT-PRO Extension.
    Calvert M, Kyte D, Mercieca-Bebber R, et al. · JAMA · 2018 · Background

2005 (1 paper)

  1. Long-term efficacy of early versus delayed radiotherapy for low-grade astrocytoma and oligodendroglioma in adults: the EORTC 22845 randomised trial.
    van den Bent MJ, Afra D, de Witte O, et al. · Lancet · 2005 · Background

Sources and methodology

This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).