Mirdametinib Evidence: Trial Results and Peer-Reviewed Publications

Arteriovenous Malformations (29)

• Clinical outcome assessment in clinical trials of chronic pain treatments.
  Pain Rep · 2024 · PMID 33521482 · NCT05983159
• Interpretation of chronic pain clinical trial outcomes: IMMPACT recommended considerations.
  Pain · 2021 · PMID 32520773 · NCT05983159
• Surgical Management of Intramuscular Venous Malformations.
  J Pediatr Orthop · 2021 · PMID 32815867 · NCT05983159
• The REDCap consortium: Building an international community of software platform partners.
  J Biomed Inform · 2020 · PMID 31078660 · NCT05983159
• Quantification of mandibular sexual dimorphism during adolescence.
  J Anat · 2020 · PMID 30834524 · NCT05983159

Neurofibromatoses (1)

• ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma.
  J Clin Oncol · 2025 · PMID 39514826 · NCT03962543

2025 (2 papers)

1. 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.
   Fangusaro J, Onar-Thomas A, Young Poussaint T, et al. · Neuro Oncol · 2025 · Trial result
   PubMed: PMID 40241281 · NCT06666348 (MIRV)
2. ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma.
   Moertel CL, Hirbe AC, Shuhaiber HH, et al. · J Clin Oncol · 2025 · Derived
   PubMed: PMID 39514826 · NCT03962543 (ReNeu) · Neurofibromatoses

2024 (1 paper)

1. Clinical outcome assessment in clinical trials of chronic pain treatments.
   Patel KV, Amtmann D, Jensen MP, et al. · Pain Rep · 2024 · Background
   PubMed: PMID 33521482 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2021 (4 papers)

1. NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas.
   Weiss BD, Wolters PL, Plotkin SR, et al. · J Clin Oncol · 2021 · Trial result
   PubMed: PMID 33507822 · NCT06666348 (MIRV)
2. Phase I, Open-Label, Dose-Escalation/Dose-Expansion Study of Lifirafenib (BGB-283), an RAF Family Kinase Inhibitor, in Patients With Solid Tumors.
   Desai J, Gan H, Barrow C, et al. · J Clin Oncol · 2021 · Trial result
   PubMed: PMID 32182156 · NCT03905148
3. Interpretation of chronic pain clinical trial outcomes: IMMPACT recommended considerations.
   Smith SM, Dworkin RH, Turk DC, et al. · Pain · 2021 · Background
   PubMed: PMID 32520773 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
4. Surgical Management of Intramuscular Venous Malformations.
   Chen RJ, Vrazas JI, Penington AJ, et al. · J Pediatr Orthop · 2021 · Background
   PubMed: PMID 32815867 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2020 (4 papers)

1. Response assessment in paediatric low-grade glioma: recommendations from the Response Assessment in Pediatric Neuro-Oncology (RAPNO) working group.
   Fangusaro J, Witt O, Hernáiz Driever P, et al. · Lancet Oncol · 2020 · Trial result
   PubMed: PMID 32502457 · NCT06666348 (MIRV)
2. Selumetinib in paediatric patients with BRAF-aberrant or neurofibromatosis type 1-associated recurrent, refractory, or progressive low-grade glioma: a multicentre, phase 2 trial.
   Fangusaro J, Onar-Thomas A, Young Poussaint T, et al. · Lancet Oncol · 2020 · Trial result
   PubMed: PMID 31151904 · NCT06666348 (MIRV)
3. The REDCap consortium: Building an international community of software platform partners.
   Harris PA, Taylor R, Minor BL, et al. · J Biomed Inform · 2020 · Background
   PubMed: PMID 31078660 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
4. Quantification of mandibular sexual dimorphism during adolescence.
   Fan Y, Penington A, Kilpatrick N, et al. · J Anat · 2020 · Background
   PubMed: PMID 30834524 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2019 (3 papers)

1. Modelling 3D craniofacial growth trajectories for population comparison and classification illustrated using sex-differences.
   Matthews HS, Penington AJ, Hardiman R, et al. · Sci Rep · 2019 · Background
   PubMed: PMID 29556038 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
2. Sirolimus is efficacious in treatment for extensive and/or complex slow-flow vascular malformations: a monocentric prospective phase II study.
   Hammer J, Seront E, Duez S, et al. · Orphanet J Rare Dis · 2019 · Background
   PubMed: PMID 30373605 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
3. Mosaic RAS/MAPK variants cause sporadic vascular malformations which respond to targeted therapy.
   Al-Olabi L, Polubothu S, Dowsett K, et al. · J Clin Invest · 2019 · Background
   PubMed: PMID 29461977 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2018 (3 papers)

1. Prolonged antibiotic treatment for infected low flow vascular malformations.
   Wagner KM, Lokmic Z, Penington AJ, et al. · J Pediatr Surg · 2018 · Background
   PubMed: PMID 28599969 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
2. Targeted therapy in patients with PIK3CA-related overgrowth syndrome.
   Venot Q, Blanc T, Rabia SH, et al. · Nature · 2018 · Background
   PubMed: PMID 29899452 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
3. The Public Health Burden of Lymphatic Malformations in Children: National Estimates in the United States, 2000-2009.
   Cheng J, Liu B, Farjat AE, et al. · Lymphat Res Biol · 2018 · Background
   PubMed: PMID 28759318 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2017 (1 paper)

1. Phase II Weekly Vinblastine for Chemotherapy-Naïve Children With Progressive Low-Grade Glioma: A Canadian Pediatric Brain Tumor Consortium Study.
   Lassaletta A, Scheinemann K, Zelcer SM, et al. · J Clin Oncol · 2017 · Trial result
   PubMed: PMID 27573663 · NCT06666348 (MIRV)

2016 (1 paper)

1. Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies.
   Adams DM, Trenor CC, Hammill AM, et al. · Pediatrics · 2016 · Background
   PubMed: PMID 26783326 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2015 (2 papers)

1. Clinical delineation and natural history of the PIK3CA-related overgrowth spectrum.
   Keppler-Noreuil KM, Sapp JC, Lindhurst MJ, et al. · Am J Med Genet A · 2015 · Background
   PubMed: PMID 24782230 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
2. Characterization of the novel and specific PI3Kα inhibitor NVP-BYL719 and development of the patient stratification strategy for clinical trials.
   Fritsch C, Huang A, Chatenay-Rivauday C, et al. · Mol Cancer Ther · 2015 · Background
   PubMed: PMID 24608574 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2014 (2 papers)

1. Assessing PIK3CA and PTEN in early-phase trials with PI3K/AKT/mTOR inhibitors.
   Janku F, Hong DS, Fu S, et al. · Cell Rep · 2014 · Background
   PubMed: PMID 24440717 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
2. Recommendations for imaging tumor response in neurofibromatosis clinical trials.
   Dombi E, Ardern-Holmes SL, Babovic-Vuksanovic D, et al. · Neurology · 2014 · Background
   PubMed: PMID 24249804 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2012 (3 papers)

1. MAPK pathway activation in pilocytic astrocytoma.
   Jones DT, Gronych J, Lichter P, et al. · Cell Mol Life Sci · 2012 · Trial result
   PubMed: PMID 22159586 · NCT06666348 (MIRV)
2. ERK1/2 MAP kinases: structure, function, and regulation.
   Roskoski R · Pharmacol Res · 2012 · Background
   PubMed: PMID 22569528 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
3. Sirolimus for the treatment of complicated vascular anomalies in children.
   Hammill AM, Wentzel M, Gupta A, et al. · Pediatr Blood Cancer · 2012 · Background
   PubMed: PMID 21445948 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2010 (2 papers)

1. Pediatric low-grade gliomas.
   Sievert AJ, Fisher MJ · J Child Neurol · 2010 · Trial result
   PubMed: PMID 19841428 · NCT06666348 (MIRV)
2. Extracranial arteriovenous malformations: natural progression and recurrence after treatment.
   Liu AS, Mulliken JB, Zurakowski D, et al. · Plast Reconstr Surg · 2010 · Background
   PubMed: PMID 20335868 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2009 (3 papers)

1. Pain outcomes: a brief review of instruments and techniques.
   Younger J, McCue R, Mackey S, et al. · Curr Pain Headache Rep · 2009 · Background
   PubMed: PMID 19126370 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
2. Research electronic data capture (REDCap)--a metadata-driven methodology and workflow process for providing translational research informatics support.
   Harris PA, Taylor R, Thielke R, et al. · J Biomed Inform · 2009 · Background
   PubMed: PMID 18929686 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations
3. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1).
   Eisenhauer EA, Therasse P, Bogaerts J, et al. · Eur J Cancer · 2009 · Background
   PubMed: PMID 19097774 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2008 (1 paper)

1. Propranolol for severe hemangiomas of infancy.
   Léauté-Labrèze C, Dumas de la Roque E, Hubiche T, et al. · N Engl J Med · 2008 · Background
   PubMed: PMID 18550886 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2007 (1 paper)

1. Vascular malformations: Part I.
   Garzon MC, Huang JT, Enjolras O, et al. · J Am Acad Dermatol · 2007 · Background
   PubMed: PMID 17317485 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2006 (1 paper)

1. Systematic review of the psychometric properties, interpretability and feasibility of self-report pain intensity measures for use in clinical trials in children and adolescents.
   Stinson JN, Kavanagh T, Yamada J, et al. · Pain · 2006 · Background
   PubMed: PMID 16777328 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2004 (1 paper)

1. Core outcome domains for chronic pain clinical trials: IMMPACT recommendations.
   Turk DC, Dworkin RH, Allen RR, et al. · Pain · 2004 · Background
   PubMed: PMID 14659516 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

2000 (1 paper)

1. New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada.
   Therasse P, Arbuck SG, Eisenhauer EA, et al. · J Natl Cancer Inst · 2000 · Background
   PubMed: PMID 10655437 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

1999 (1 paper)

1. Constitutive activation of the 41-/43-kDa mitogen-activated protein kinase signaling pathway in human tumors.
   Hoshino R, Chatani Y, Yamori T, et al. · Oncogene · 1999 · Background
   PubMed: PMID 9989833 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations

1993 (1 paper)

1. Carboplatin and vincristine for recurrent and newly diagnosed low-grade gliomas of childhood.
   Packer RJ, Lange B, Ater J, et al. · J Clin Oncol · 1993 · Trial result
   PubMed: PMID 8487049 · NCT06666348 (MIRV)

1990 (1 paper)

1. Requirement for integration of signals from two distinct phosphorylation pathways for activation of MAP kinase.
   Anderson NG, Maller JL, Tonks NK, et al. · Nature · 1990 · Background
   PubMed: PMID 2154696 · NCT05983159 (TARGET-VM) · Arteriovenous Malformations