Selumetinib Evidence: Trial Results and Peer-Reviewed Publications

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

The clinical evidence base for Selumetinib comprises 99 peer-reviewed publications across 15 journals, 4 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Neurofibromatosis 1, Acquired Immunodeficiency Syndrome, Carcinoma, Non-Small-Cell Lung, and Neoplasms. Most recent publication: Phase I/II Study of the CDK2/9 Inhibitor Fadraciclib in Combination with Chemotherapy in Children with Advanced Malignancies: Arm K of the AcSé-ESMART Trial., Target Oncol, 2026.

Top peer-reviewed publications

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

  1. Results of an open-label phase 1b study of the ERK inhibitor MK-8353 plus the MEK inhibitor selumetinib in patients with advanced or metastatic solid tumors.
    Stathis A, Tolcher AW, Wang JS, et al. · Invest New Drugs · 2023
    PubMed: PMID 37040046 · NCT03745989 · Neoplasm Metastasis
  2. A phase II trial of mTORC1/2 inhibition in STK11 deficient non small cell lung cancer.
    Middleton G, Robbins HL, Fletcher P, et al. · NPJ Precis Oncol · 2025
    PubMed: PMID 40069402 · NCT02664935 · Carcinoma, Non-Small-Cell Lung
  3. MEK inhibitors - novel targeted therapies of neurofibromatosis associated benign and malignant lesions.
    Harder A · Biomark Res · 2021
    PubMed: PMID 33863389 · NCT06620354 · Neurofibromatosis 1
  4. Selumetinib in Children with Inoperable Plexiform Neurofibromas.
    Gross AM, Wolters PL, Dombi E, et al. · N Engl J Med · 2020
    PubMed: PMID 32187457 · NCT06620354 · Neurofibromatosis 1
  5. Activity of Selumetinib in Neurofibromatosis Type 1-Related Plexiform Neurofibromas.
    Dombi E, Baldwin A, Marcus LJ, et al. · N Engl J Med · 2017
    PubMed: PMID 28029918 · NCT06620354 · Neurofibromatosis 1
  6. A phase 1 trial of the MEK inhibitor selumetinib in combination with pembrolizumab for advanced or metastatic solid tumors.
    Chénard-Poirier M, Hansen AR, Gutierrez ME, et al. · Invest New Drugs · 2024
    PubMed: PMID 38483782 · NCT03833427 · Parkinson Disease 4, Autosomal Dominant Lewy Body
  7. Clinical Pharmacokinetics and Pharmacodynamics of Selumetinib.
    Campagne O, Yeo KK, Fangusaro J, et al. · Clin Pharmacokinet · 2021
    PubMed: PMID 33354735 · NCT06620354 · Neurofibromatosis 1
  8. A Review of Selumetinib in the Treatment of Neurofibromatosis Type 1-Related Plexiform Neurofibromas.
    Anderson MK, Johnson M, Thornburg L, et al. · Ann Pharmacother · 2022
    PubMed: PMID 34541874 · NCT06621082 · Neurofibromatosis 1

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
NCT01843062
ASTRA
Complete Remission Rate (Expressed as Percentage of Patients in Complete Remission) at 18 Months Post-RAI Treatment; ITT Analysis Set
At 18 months post-RAI treatment
Placebo + RAI38.5 Percentage of participants
Selumetinib 75 mg BD + RAI40.0 Percentage of participants
NCT01933932
SELECT-1
Carcinoma, Non-Small-Cell LungProgression-Free Survival (PFS)
Measured at baseline until the date of first documented objective disease progression. Estimated final completion : approximately 3 years after first subject in (FSI)
Placebo + Docetaxel2.8 Months
Selumetinib + Docetaxel3.9 Months
NCT01974752
SUMIT
Neoplasm MetastasisAssessment of the Efficacy of Selumetinib in Combination With Dacarbazine Compared With Placebo in Combination With Dacarbazine Measured as Progression Free Survival (PFS) Using BICR According to RECIST 1.1.
From Randomization, then every 6 weeks up until progression or death (whichever is sooner) assessed up to 15th May 2015
Placebo + Dacarbazine 1000 mg/m224 number of progression events
Selumetinib 75 mg BD + Dacarbazine 1000 mg/m282 number of progression events
NCT04924608
KOMET
Neurofibromatosis 1Confirmed Partial and Complete Response Rate (ORR) by End of Cycle 16 Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN.
From first dose up until progression (if it occurs prior to the end of Cycle 16), or the last evaluable assessment up to and including the end of Cycle 16, excluding MRI during prolonged study intervention interruption (defined as interruption >= 28 days)
Placebo5.4 Percentage
Selumetinib 25 mg/m219.7 Percentage

Publications by year

19892026: 99 publications.

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Publications by indication

Neurofibromatosis 1 (29)

  • Pharmacokinetics and Safety of Selumetinib Granule Formulation in Children With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas (SPRINKLE; phase I/II).
    J Clin Oncol · 2026 · PMID 41592259 · NCT05309668
  • Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1 With MEK Inhibitor Selumetinib: A Nonrandomized Clinical Trial.
    JAMA Dermatol · 2026 · PMID 40009403 · NCT02839720
  • Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial.
    Nat Med · 2025 · PMID 39762421 · NCT02407405
  • Efficacy and safety of selumetinib in adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas (KOMET): a multicentre, international, randomised, placebo-controlled, parallel, double-blind, phase 3 study.
    Lancet · 2025 · PMID 40473450 · NCT04924608
  • Effect of food on selumetinib pharmacokinetics and gastrointestinal tolerability in adolescents with neurofibromatosis type 1-related plexiform neurofibromas.
    Neurooncol Adv · 2024 · PMID 38721358 · NCT05101148

Acquired Immunodeficiency Syndrome (14)

  • A phase II open-label randomized study to assess the efficacy and safety of selumetinib (AZD6244 [ARRY-142886]) versus capecitabine in patients with advanced or metastatic pancreatic cancer who have failed first-line gemcitabine therapy.
    Invest New Drugs · 2012 · PMID 21594619 · NCT01752569
  • The first-in-human study of the hydrogen sulfate (Hyd-sulfate) capsule of the MEK1/2 inhibitor AZD6244 (ARRY-142886): a phase I open-label multicenter trial in patients with advanced cancer.
    Clin Cancer Res · 2010 · PMID 20179232 · NCT01752569
  • British HIV Association guidelines for HIV-associated malignancies 2008.
    HIV Med · 2009 · PMID 18705759 · NCT01752569
  • Reactivation of Kaposi's sarcoma-associated herpesvirus from latency requires MEK/ERK, JNK and p38 multiple mitogen-activated protein kinase pathways.
    Virology · 2008 · PMID 17964626 · NCT01752569
  • Phase I pharmacokinetic and pharmacodynamic study of the oral, small-molecule mitogen-activated protein kinase kinase 1/2 inhibitor AZD6244 (ARRY-142886) in patients with advanced cancers.
    J Clin Oncol · 2008 · PMID 18390968 · NCT01752569

Carcinoma, Non-Small-Cell Lung (13)

  • Osimertinib plus datopotamab deruxtecan in patients with EGFR-mutated advanced NSCLC after progression on first-line osimertinib: ORCHARD.
    Ann Oncol · 2026 · PMID 41780641 · NCT03944772
  • Osimertinib Plus Savolitinib in Patients With EGFR-Mutated Advanced NSCLC With MET Alterations After First-Line Osimertinib: Clinical Outcomes, Safety, and Biomarker Analysis: A Brief Report.
    J Thorac Oncol · 2026 · PMID 41130408 · NCT03944772
  • A phase II trial of mTORC1/2 inhibition in STK11 deficient non small cell lung cancer.
    NPJ Precis Oncol · 2025 · PMID 40069402 · NCT02664935
  • ORCHARD: Osimertinib Plus Necitumumab in Patients With Epidermal Growth Factor Receptor-Mutated Advanced Non-Small Cell Lung Cancer With a Secondary Epidermal Growth Factor Receptor Alteration Whose Disease Had Progressed on First-Line Osimertinib.
    JCO Precis Oncol · 2025 · PMID 40466026 · NCT03944772
  • Durvalumab Versus Chemotherapy as First-line Treatment for Metastatic NSCLC With Tumor PD-L1 Expression of 25% or Higher: Results From the Randomized Phase 3 PEARL Study.
    J Thorac Oncol · 2025 · PMID 39521433 · NCT03944772

Neoplasms (6)

  • Phase I/II Study of the CDK2/9 Inhibitor Fadraciclib in Combination with Chemotherapy in Children with Advanced Malignancies: Arm K of the AcSé-ESMART Trial.
    Target Oncol · 2026 · PMID 41838385 · NCT02813135
  • The European MAPPYACTS Trial: Precision Medicine Program in Pediatric and Adolescent Patients with Recurrent Malignancies.
    Cancer Discov · 2022 · PMID 35292802 · NCT02813135
  • Phase II and biomarker study of programmed cell death protein 1 inhibitor nivolumab and metronomic cyclophosphamide in paediatric relapsed/refractory solid tumours: Arm G of AcSé-ESMART, a trial of the European Innovative Therapies for Children With Cancer Consortium.
    Eur J Cancer · 2021 · PMID 33892407 · NCT02813135
  • First-in-child phase I/II study of the dual mTORC1/2 inhibitor vistusertib (AZD2014) as monotherapy and in combination with topotecan-temozolomide in children with advanced malignancies: arms E and F of the AcSé-ESMART trial.
    Eur J Cancer · 2021 · PMID 34543871 · NCT02813135
  • Phase I or II Study of Ribociclib in Combination With Topotecan-Temozolomide or Everolimus in Children With Advanced Malignancies: Arms A and B of the AcSé-ESMART Trial.
    J Clin Oncol · 2021 · PMID 34347542 · NCT02813135

Neoplasm Metastasis (3)

  • Results of an open-label phase 1b study of the ERK inhibitor MK-8353 plus the MEK inhibitor selumetinib in patients with advanced or metastatic solid tumors.
    Invest New Drugs · 2023 · PMID 37040046 · NCT03745989
  • Selumetinib in Combination With Dacarbazine in Patients With Metastatic Uveal Melanoma: A Phase III, Multicenter, Randomized Trial (SUMIT).
    J Clin Oncol · 2019 · PMID 29528792 · NCT01974752
  • Study design and rationale for a randomised, placebo-controlled, double-blind study to assess the efficacy of selumetinib (AZD6244; ARRY-142886) in combination with dacarbazine in patients with metastatic uveal melanoma (SUMIT).
    BMC Cancer · 2016 · PMID 26059332 · NCT01974752

Sarcoma (3)

  • Germline Cancer Predisposition Results From the National Cancer Institute-Children's Oncology Group Pediatric MATCH Trial.
    JCO Precis Oncol · 2025 · PMID 41166674 · NCT03155620
  • Actionable Tumor Alterations and Treatment Protocol Enrollment of Pediatric and Young Adult Patients With Refractory Cancers in the National Cancer Institute-Children's Oncology Group Pediatric MATCH Trial.
    J Clin Oncol · 2022 · PMID 35353553 · NCT03155620
  • Progress Update in Pediatric Renal Tumors.
    Curr Oncol Rep · 2022 · PMID 33591402 · NCT03155620

Publications by journal

Trial-results highlights

For Carcinoma, Non-Small-Cell Lung, the SELECT-1 study (NCT01933932) investigated Selumetinib in combination with Docetaxel. This trial measured Progression-Free Survival (PFS), defined as the time from baseline until the date of first documented objective disease progression. The median PFS was 2.8 Months for participants receiving Placebo + Docetaxel, and 3.9 Months for those receiving Selumetinib + Docetaxel. For Neoplasm Metastasis, the SUMIT study (NCT01974752) assessed the efficacy of Selumetinib in combination with Dacarbazine compared with placebo in combination with Dacarbazine. This study measured Progression Free Survival (PFS) using Blinded Independent Central Review (BICR) according to RECIST 1.1. In this study, there were 24 progression events in the Placebo + Dacarbazine 1000 mg/m2 arm, and 82 progression events in the Selumetinib 75 mg BD + Dacarbazine 1000 mg/m2 arm.

In Neurofibromatosis 1, the KOMET study (NCT04924608) evaluated Selumetinib 25 mg/m2 in participants with symptomatic, inoperable plexiform neurofibromas (PN). This trial assessed the Confirmed Partial and Complete Response Rate (ORR) by the end of Cycle 16, using volumetric MRI analysis as determined by Independent Central Review (ICR) per REiNS Criteria. The ORR was 5.4 Percentage for the Placebo arm and 19.7 Percentage for the Selumetinib 25 mg/m2 arm. The ASTRA study (NCT01843062) investigated Selumetinib 75 mg BD + RAI. In this trial, the Complete Remission Rate at 18 Months Post-RAI Treatment was 38.5 Percentage of participants in the Placebo + RAI arm, and 40.0 Percentage of participants in the Selumetinib 75 mg BD + RAI arm.

All values presented are sourced from primary registry reporting; individual publications should be consulted for comprehensive clinical decision-making.

All Selumetinib publications (99)

2026 (5 papers)

  1. Phase I/II Study of the CDK2/9 Inhibitor Fadraciclib in Combination with Chemotherapy in Children with Advanced Malignancies: Arm K of the AcSé-ESMART Trial.
    Rubio-San-Simón A, Marshall LV, Karamouza E, et al. · Target Oncol · 2026 · Derived
  2. Osimertinib plus datopotamab deruxtecan in patients with EGFR-mutated advanced NSCLC after progression on first-line osimertinib: ORCHARD.
    Riess JW, Yu HA, Le X, et al. · Ann Oncol · 2026 · Derived
    PubMed: PMID 41780641 · NCT03944772 (ORCHARD) · Carcinoma, Non-Small-Cell Lung
  3. Osimertinib Plus Savolitinib in Patients With EGFR-Mutated Advanced NSCLC With MET Alterations After First-Line Osimertinib: Clinical Outcomes, Safety, and Biomarker Analysis: A Brief Report.
    Le X, Baik C, Cho BC, et al. · J Thorac Oncol · 2026 · Derived
    PubMed: PMID 41130408 · NCT03944772 (ORCHARD) · Carcinoma, Non-Small-Cell Lung
  4. Pharmacokinetics and Safety of Selumetinib Granule Formulation in Children With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas (SPRINKLE; phase I/II).
    Hernáiz Driever P, Kordes UR, Brecht IB, et al. · J Clin Oncol · 2026 · Derived
  5. Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1 With MEK Inhibitor Selumetinib: A Nonrandomized Clinical Trial.
    Gross AM, Reid OH, Baldwin LA, et al. · JAMA Dermatol · 2026 · Derived
    PubMed: PMID 40009403 · NCT02839720 · Neurofibromatosis 1

2025 (6 papers)

  1. A phase II trial of mTORC1/2 inhibition in STK11 deficient non small cell lung cancer.
    Middleton G, Robbins HL, Fletcher P, et al. · NPJ Precis Oncol · 2025 · Trial result
    PubMed: PMID 40069402 · NCT02664935 · Carcinoma, Non-Small-Cell Lung
  2. Germline Cancer Predisposition Results From the National Cancer Institute-Children's Oncology Group Pediatric MATCH Trial.
    Scollon S, Plon SE, Joffe S, et al. · JCO Precis Oncol · 2025 · Derived
  3. ORCHARD: Osimertinib Plus Necitumumab in Patients With Epidermal Growth Factor Receptor-Mutated Advanced Non-Small Cell Lung Cancer With a Secondary Epidermal Growth Factor Receptor Alteration Whose Disease Had Progressed on First-Line Osimertinib.
    Riess JW, de Langen AJ, Ponce S, et al. · JCO Precis Oncol · 2025 · Derived
    PubMed: PMID 40466026 · NCT03944772 (ORCHARD) · Carcinoma, Non-Small-Cell Lung
  4. Durvalumab Versus Chemotherapy as First-line Treatment for Metastatic NSCLC With Tumor PD-L1 Expression of 25% or Higher: Results From the Randomized Phase 3 PEARL Study.
    Lu S, Wu L, Wang Q, et al. · J Thorac Oncol · 2025 · Derived
    PubMed: PMID 39521433 · NCT03944772 (ORCHARD) · Carcinoma, Non-Small-Cell Lung
  5. Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial.
    Gross AM, O'Sullivan Coyne G, Dombi E, et al. · Nat Med · 2025 · Derived
    PubMed: PMID 39762421 · NCT02407405 · Neurofibromatosis 1
  6. Efficacy and safety of selumetinib in adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas (KOMET): a multicentre, international, randomised, placebo-controlled, parallel, double-blind, phase 3 study.
    Chen AP, Coyne GO, Wolters PL, et al. · Lancet · 2025 · Derived
    PubMed: PMID 40473450 · NCT04924608 (KOMET) · Neurofibromatosis 1

2024 (5 papers)

  1. A phase 1 trial of the MEK inhibitor selumetinib in combination with pembrolizumab for advanced or metastatic solid tumors.
    Chénard-Poirier M, Hansen AR, Gutierrez ME, et al. · Invest New Drugs · 2024 · Trial result
    PubMed: PMID 38483782 · NCT03833427 · Parkinson Disease 4, Autosomal Dominant Lewy Body
  2. Effect of food on selumetinib pharmacokinetics and gastrointestinal tolerability in adolescents with neurofibromatosis type 1-related plexiform neurofibromas.
    Viskochil D, Wysocki M, Learoyd M, et al. · Neurooncol Adv · 2024 · Derived
    PubMed: PMID 38721358 · NCT05101148 · Neurofibromatosis 1
  3. Phase 1 trial of navitoclax and sorafenib in patients with relapsed or refractory solid tumors with hepatocellular carcinoma expansion cohort.
    Emiloju OE, Yin J, Koubek E, et al. · Invest New Drugs · 2024 · Derived
  4. snRNA-seq of human cutaneous neurofibromas before and after selumetinib treatment implicates role of altered Schwann cell states, inter-cellular signaling, and extracellular matrix in treatment response.
    Church C, Fay CX, Kriukov E, et al. · Acta Neuropathol Commun · 2024 · Derived
    PubMed: PMID 38907342 · NCT02839720 · Neurofibromatosis 1
  5. Safety and efficacy of selumetinib in pediatric and adult patients with neurofibromatosis type 1 and plexiform neurofibroma.
    Kim H, Yoon HM, Kim EK, et al. · Neuro Oncol · 2024 · Background

2023 (2 papers)

  1. Results of an open-label phase 1b study of the ERK inhibitor MK-8353 plus the MEK inhibitor selumetinib in patients with advanced or metastatic solid tumors.
    Stathis A, Tolcher AW, Wang JS, et al. · Invest New Drugs · 2023 · Trial result
    PubMed: PMID 37040046 · NCT03745989 · Neoplasm Metastasis
  2. Long-term safety and efficacy of selumetinib in children with neurofibromatosis type 1 on a phase 1/2 trial for inoperable plexiform neurofibromas.
    Gross AM, Dombi E, Wolters PL, et al. · Neuro Oncol · 2023 · Background

2022 (14 papers)

  1. A Review of Selumetinib in the Treatment of Neurofibromatosis Type 1-Related Plexiform Neurofibromas.
    Anderson MK, Johnson M, Thornburg L, et al. · Ann Pharmacother · 2022 · Trial result
    PubMed: PMID 34541874 · NCT06621082 · Neurofibromatosis 1
  2. Biomarker-Directed Phase II Platform Study in Patients With EGFR Sensitizing Mutation-Positive Advanced/Metastatic Non-Small Cell Lung Cancer Whose Disease Has Progressed on First-Line Osimertinib Therapy (ORCHARD).
    Yu HA, Goldberg SB, Le X, et al. · Clin Lung Cancer · 2022 · Derived
    PubMed: PMID 34389237 · NCT03944772 (ORCHARD) · Carcinoma, Non-Small-Cell Lung
  3. Actionable Tumor Alterations and Treatment Protocol Enrollment of Pediatric and Young Adult Patients With Refractory Cancers in the National Cancer Institute-Children's Oncology Group Pediatric MATCH Trial.
    Parsons DW, Janeway KA, Patton DR, et al. · J Clin Oncol · 2022 · Derived
  4. Harnessing cell-free DNA: plasma circulating tumour DNA for liquid biopsy in genitourinary cancers.
    Maia MC, Salgia M, Pal SK, et al. · Nat Rev Urol · 2022 · Derived
  5. Phase II study of selumetinib, an orally active inhibitor of MEK1 and MEK2 kinases, in KRASG12R-mutant pancreatic ductal adenocarcinoma.
    Kenney C, Kunst T, Webb S, et al. · Invest New Drugs · 2022 · Derived
  6. Progress Update in Pediatric Renal Tumors.
    Jain J, Sutton KS, Hong AL, et al. · Curr Oncol Rep · 2022 · Derived
  7. Selumetinib Plus Adjuvant Radioactive Iodine in Patients With High-Risk Differentiated Thyroid Cancer: A Phase III, Randomized, Placebo-Controlled Trial (ASTRA).
    Ho AL, Dedecjus M, Wirth LJ, et al. · J Clin Oncol · 2022 · Derived
  8. Phase II Study of Selumetinib in Children and Young Adults With Tumors Harboring Activating Mitogen-Activated Protein Kinase Pathway Genetic Alterations: Arm E of the NCI-COG Pediatric MATCH Trial.
    Eckstein OS, Allen CE, Williams PM, et al. · J Clin Oncol · 2022 · Derived
    PubMed: PMID 35363510 · NCT03213691 · Central Nervous System Neoplasms
  9. Robotic Nerve Sheath Tumor Resection With Intraoperative Neuromonitoring: Case Series and Systematic Review.
    Curry BP, Alvarez R, Widemann BC, et al. · Oper Neurosurg · 2022 · Derived
    PubMed: PMID 35007270 · NCT02407405 · Neurofibromatosis 1
  10. The European MAPPYACTS Trial: Precision Medicine Program in Pediatric and Adolescent Patients with Recurrent Malignancies.
    Berlanga P, Pierron G, Lacroix L, et al. · Cancer Discov · 2022 · Derived
  11. Osimertinib Plus Durvalumab in Patients With EGFR-Mutated, Advanced NSCLC: A Phase 1b, Open-Label, Multicenter Trial.
    Ahn MJ, Cho BC, Ou X, et al. · J Thorac Oncol · 2022 · Derived
  12. Clinical and humanistic burden among pediatric patients with neurofibromatosis type 1 and plexiform neurofibroma in the USA.
    Yang X, Yoo HK, Amin S, et al. · Childs Nerv Syst · 2022 · Background
  13. Selumetinib in combination with dexamethasone for the treatment of relapsed/refractory RAS-pathway mutated paediatric and adult acute lymphoblastic leukaemia (SeluDex): study protocol for an international, parallel-group, dose-finding with expansion phase I/II trial.
    Menne T, Slade D, Savage J, et al. · BMJ Open · 2022 · Background
    PubMed: PMID 35246426 · NCT03705507 (SeluDex) · Precursor Cell Lymphoblastic Leukemia-Lymphoma
  14. Selumetinib in children with neurofibromatosis type 1 and asymptomatic inoperable plexiform neurofibroma at risk for developing tumor-related morbidity.
    Gross AM, Glassberg B, Wolters PL, et al. · Neuro Oncol · 2022 · Background

2021 (13 papers)

  1. MEK inhibitors - novel targeted therapies of neurofibromatosis associated benign and malignant lesions.
    Harder A · Biomark Res · 2021 · Trial result
    PubMed: PMID 33863389 · NCT06620354 · Neurofibromatosis 1
  2. Clinical Pharmacokinetics and Pharmacodynamics of Selumetinib.
    Campagne O, Yeo KK, Fangusaro J, et al. · Clin Pharmacokinet · 2021 · Trial result
    PubMed: PMID 33354735 · NCT06620354 · Neurofibromatosis 1
  3. Savolitinib ± Osimertinib in Japanese Patients with Advanced Solid Malignancies or EGFRm NSCLC: Ph1b TATTON Part C.
    Yoh K, Hirashima T, Saka H, et al. · Target Oncol · 2021 · Derived
  4. Systems approach to rational combination therapy: PARP inhibitors.
    Sun C, Fang Y, Labrie M, et al. · Biochem Soc Trans · 2021 · Derived
    PubMed: PMID 32379297 · NCT03801369 · Triple Negative Breast Neoplasms
  5. Phase II and biomarker study of programmed cell death protein 1 inhibitor nivolumab and metronomic cyclophosphamide in paediatric relapsed/refractory solid tumours: Arm G of AcSé-ESMART, a trial of the European Innovative Therapies for Children With Cancer Consortium.
    Pasqualini C, Rubino J, Brard C, et al. · Eur J Cancer · 2021 · Derived
  6. TATTON: a multi-arm, phase Ib trial of osimertinib combined with selumetinib, savolitinib, or durvalumab in EGFR-mutant lung cancer.
    Oxnard GR, Yang JC, Yu H, et al. · Ann Oncol · 2021 · Derived
  7. Outcome and molecular landscape of patients with PIK3CA-mutated metastatic breast cancer.
    Mosele F, Stefanovska B, Lusque A, et al. · Ann Oncol · 2021 · Derived
  8. First-in-child phase I/II study of the dual mTORC1/2 inhibitor vistusertib (AZD2014) as monotherapy and in combination with topotecan-temozolomide in children with advanced malignancies: arms E and F of the AcSé-ESMART trial.
    Morscher RJ, Brard C, Berlanga P, et al. · Eur J Cancer · 2021 · Derived
  9. Isoform- and Phosphorylation-specific Multiplexed Quantitative Pharmacodynamics of Drugs Targeting PI3K and MAPK Signaling in Xenograft Models and Clinical Biopsies.
    Herrick WG, Kilpatrick CL, Hollingshead MG, et al. · Mol Cancer Ther · 2021 · Derived
    PubMed: PMID 33536190 · NCT02407405 · Neurofibromatosis 1
  10. MEK inhibitors in RASopathies.
    Bergqvist C, Wolkenstein P · Curr Opin Oncol · 2021 · Derived
  11. Phase I or II Study of Ribociclib in Combination With Topotecan-Temozolomide or Everolimus in Children With Advanced Malignancies: Arms A and B of the AcSé-ESMART Trial.
    Bautista F, Paoletti X, Rubino J, et al. · J Clin Oncol · 2021 · Derived
  12. Response to letter to the editor: "All models are wrong; some models are useful".
    Arnold A, Rodriguez F, Eberhart CG, et al. · Neuro Oncol · 2021 · Derived
  13. An adaptive, biomarker-directed platform study of durvalumab in combination with targeted therapies in advanced urothelial cancer.
    Powles T, Carroll D, Chowdhury S, et al. · Nat Med · 2021 · Background

2020 (8 papers)

  1. The National Lung Matrix Trial of personalized therapy in lung cancer.
    Middleton G, Fletcher P, Popat S, et al. · Nature · 2020 · Trial result
    PubMed: PMID 32669708 · NCT02664935 · Carcinoma, Non-Small-Cell Lung
  2. Selumetinib in patients receiving standard pemetrexed and platinum-based chemotherapy for advanced or metastatic KRAS wildtype or unknown non-squamous non-small cell lung cancer: A randomized, multicenter, phase II study. Canadian Cancer Trials Group (CCTG) IND.219.
    Melosky B, Bradbury P, Tu D, et al. · Lung Cancer · 2020 · Trial result
    PubMed: PMID 31200828 · NCT02337530 · Carcinoma, Non-Small-Cell Lung
  3. Selumetinib in Children with Inoperable Plexiform Neurofibromas.
    Gross AM, Wolters PL, Dombi E, et al. · N Engl J Med · 2020 · Trial result
    PubMed: PMID 32187457 · NCT06620354 · Neurofibromatosis 1
  4. Canadian Cancer Trials Group (CCTG) IND215: A phase Ib study of Selumetinib in patients with untreated advanced or metastatic NSCLC who are receiving standard chemotherapy regimens.
    Goffin JR, Nicholas G, Mates M, et al. · Invest New Drugs · 2020 · Trial result
    PubMed: PMID 30317534 · NCT01783197 · Carcinoma, Non-Small-Cell Lung
  5. Osimertinib plus savolitinib in patients with EGFR mutation-positive, MET-amplified, non-small-cell lung cancer after progression on EGFR tyrosine kinase inhibitors: interim results from a multicentre, open-label, phase 1b study.
    Sequist LV, Han JY, Ahn MJ, et al. · Lancet Oncol · 2020 · Derived
  6. Targeting MET in EGFR resistance in non-small-cell lung cancer-ready for daily practice?
    Schmid S, Früh M, Peters S, et al. · Lancet Oncol · 2020 · Derived
    PubMed: PMID 32027845 · NCT03944772 (ORCHARD) · Carcinoma, Non-Small-Cell Lung
  7. Sequential or combined designs for Phase I/II clinical trials? A simulation study.
    Rossoni C, Bardet A, Geoerger B, et al. · Clin Trials · 2020 · Derived
  8. 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 · Derived
    PubMed: PMID 31151904 · NCT01089101 · Neurofibromatosis 1

2019 (5 papers)

  1. Health Supervision for Children With Neurofibromatosis Type 1.
    Miller DT, Freedenberg D, Schorry E, et al. · Pediatrics · 2019 · Trial result
    PubMed: PMID 31010905 · NCT06621082 · Neurofibromatosis 1
  2. The Diagnosis and Management of Neurofibromatosis Type 1.
    Ly KI, Blakeley JO · Med Clin North Am · 2019 · Trial result
    PubMed: PMID 31582003 · NCT06620354 · Neurofibromatosis 1
  3. Association of plexiform neurofibroma volume changes and development of clinical morbidities in neurofibromatosis 1.
    Gross AM, Singh G, Akshintala S, et al. · Neuro Oncol · 2019 · Trial result
    PubMed: PMID 29718344 · NCT06620354 · Neurofibromatosis 1
  4. Resection of small plexiform neurofibromas in neurofibromatosis type 1 children.
    Friedrich RE, Schmelzle R, Hartmann M, et al. · World J Surg Oncol · 2019 · Trial result
    PubMed: PMID 15683544 · NCT06620354 · Neurofibromatosis 1
  5. Selumetinib in Combination With Dacarbazine in Patients With Metastatic Uveal Melanoma: A Phase III, Multicenter, Randomized Trial (SUMIT).
    Carvajal RD, Piperno-Neumann S, Kapiteijn E, et al. · J Clin Oncol · 2019 · Derived
    PubMed: PMID 29528792 · NCT01974752 (SUMIT) · Neoplasm Metastasis

2018 (5 papers)

  1. Neurofibromatosis type 1.
    Gutmann DH, Ferner RE, Listernick RH, et al. · Nat Rev Dis Primers · 2018 · Trial result
    PubMed: PMID 28230061 · NCT06621082 · Neurofibromatosis 1
  2. Sphenoid dysplasia in neurofibromatosis type 1: a new technique for repair.
    Di Rocc C, Samii A, Tamburrini G, et al. · Childs Nerv Syst · 2018 · Trial result
    PubMed: PMID 28409229 · NCT06621082 · Neurofibromatosis 1
  3. Comparison of the Pharmacokinetics of the Phase II and Phase III Capsule Formulations of Selumetinib and the Effects of Food on Exposure: Results From Two Randomized Crossover Trials in Healthy Male Subjects.
    Tomkinson H, McBride E, Martin P, et al. · Clin Ther · 2018 · Derived
  4. SELECT-2: a phase II, double-blind, randomized, placebo-controlled study to assess the efficacy of selumetinib plus docetaxel as a second-line treatment of patients with advanced or metastatic non-small-cell lung cancer.
    Soria JC, Fülöp A, Maciel C, et al. · Ann Oncol · 2018 · Derived
    PubMed: PMID 29045535 · NCT01750281 (SELECT-2) · Carcinoma, Non-Small-Cell Lung
  5. A phase I trial of the MEK inhibitor selumetinib (AZD6244) in pediatric patients with recurrent or refractory low-grade glioma: a Pediatric Brain Tumor Consortium (PBTC) study.
    Banerjee A, Jakacki RI, Onar-Thomas A, et al. · Neuro Oncol · 2018 · Background

2017 (6 papers)

  1. Activity of Selumetinib in Neurofibromatosis Type 1-Related Plexiform Neurofibromas.
    Dombi E, Baldwin A, Marcus LJ, et al. · N Engl J Med · 2017 · Trial result
    PubMed: PMID 28029918 · NCT06620354 · Neurofibromatosis 1
  2. Therapeutic advances for the tumors associated with neurofibromatosis type 1, type 2, and schwannomatosis.
    Blakeley JO, Plotkin SR · Neuro Oncol · 2017 · Trial result
    PubMed: PMID 26851632 · NCT06621082 · Neurofibromatosis 1
  3. Selumetinib Plus Docetaxel Compared With Docetaxel Alone and Progression-Free Survival in Patients With KRAS-Mutant Advanced Non-Small Cell Lung Cancer: The SELECT-1 Randomized Clinical Trial.
    Jänne PA, van den Heuvel MM, Barlesi F, et al. · JAMA · 2017 · Derived
    PubMed: PMID 28492898 · NCT01933932 (SELECT-1) · Carcinoma, Non-Small-Cell Lung
  4. Metabolism, Excretion, and Pharmacokinetics of Selumetinib, an MEK1/2 inhibitor, in Healthy Adult Male Subjects.
    Dymond AW, Howes C, Pattison C, et al. · Clin Ther · 2017 · Derived
  5. Effect of Selumetinib and MK-2206 vs Oxaliplatin and Fluorouracil in Patients With Metastatic Pancreatic Cancer After Prior Therapy: SWOG S1115 Study Randomized Clinical Trial.
    Chung V, McDonough S, Philip PA, et al. · JAMA Oncol · 2017 · Derived
    PubMed: PMID 27978579 · NCT01658943 (S1115) · Pancreatic Neoplasms
  6. Relationship between physician-adjudicated adverse events and patient-reported health-related quality of life in a phase II clinical trial (NCT01143402) of patients with metastatic uveal melanoma.
    Atkinson TM, Hay JL, Shoushtari A, et al. · J Cancer Res Clin Oncol · 2017 · Derived
    PubMed: PMID 27921276 · NCT01143402 · Uveal Melanoma

2016 (4 papers)

  1. The National Lung Matrix Trial: translating the biology of stratification in advanced non-small-cell lung cancer.
    Middleton G, Crack LR, Popat S, et al. · Ann Oncol · 2016 · Derived
  2. Study Design and Rationale for a Randomized, Placebo-Controlled, Double-Blind Study to Assess the Efficacy and Safety of Selumetinib in Combination With Docetaxel as Second-Line Treatment in Patients With KRAS-Mutant Advanced Non-Small Cell Lung Cancer (SELECT-1).
    Jänne PA, Mann H, Ghiorghiu D, et al. · Clin Lung Cancer · 2016 · Derived
    PubMed: PMID 26837474 · NCT01933932 (SELECT-1) · Carcinoma, Non-Small-Cell Lung
  3. Study design and rationale for a randomised, placebo-controlled, double-blind study to assess the efficacy of selumetinib (AZD6244; ARRY-142886) in combination with dacarbazine in patients with metastatic uveal melanoma (SUMIT).
    Carvajal RD, Schwartz GK, Mann H, et al. · BMC Cancer · 2016 · Derived
    PubMed: PMID 26059332 · NCT01974752 (SUMIT) · Neoplasm Metastasis
  4. A phase 1b study of Selumetinib in combination with Cisplatin and Gemcitabine in advanced or metastatic biliary tract cancer: the ABC-04 study.
    Bridgewater J, Lopes A, Beare S, et al. · BMC Cancer · 2016 · Derived
    PubMed: PMID 26912134 · NCT01242605 (ABC-04) · Biliary Tract Neoplasms

2015 (2 papers)

  1. Fulvestrant with or without selumetinib, a MEK 1/2 inhibitor, in breast cancer progressing after aromatase inhibitor therapy: a multicentre randomised placebo-controlled double-blind phase II trial, SAKK 21/08.
    Zaman K, Winterhalder R, Mamot C, et al. · Eur J Cancer · 2015 · Trial result
    PubMed: PMID 25892646 · NCT01160718 · Breast Neoplasms
  2. A phase II evaluation of selumetinib (AZD6244, ARRY-142886), a selective MEK-1/2 inhibitor in the treatment of recurrent or persistent endometrial cancer: an NRG Oncology/Gynecologic Oncology Group study.
    Coleman RL, Sill MW, Thaker PH, et al. · Gynecol Oncol · 2015 · Derived

2014 (2 papers)

  1. Phase II study of the oral MEK inhibitor selumetinib in advanced acute myelogenous leukemia: a University of Chicago phase II consortium trial.
    Jain N, Curran E, Iyengar NM, et al. · Clin Cancer Res · 2014 · Trial result
    PubMed: PMID 24178622 · NCT00588809 · Leukemia, Myeloid, Acute
  2. Effect of selumetinib vs chemotherapy on progression-free survival in uveal melanoma: a randomized clinical trial.
    Carvajal RD, Sosman JA, Quevedo JF, et al. · JAMA · 2014 · Derived
    PubMed: PMID 24938562 · NCT01143402 · Uveal Melanoma

2013 (3 papers)

  1. Social-emotional functioning of children and adolescents with neurofibromatosis type 1 and plexiform neurofibromas: relationships with cognitive, disease, and environmental variables.
    Martin S, Wolters P, Baldwin A, et al. · J Pediatr Psychol · 2013 · Trial result
    PubMed: PMID 22353803 · NCT06620354 · Neurofibromatosis 1
  2. Selumetinib in women with recurrent low-grade serous carcinoma of the ovary or peritoneum: an open-label, single-arm, phase 2 study.
    Farley J, Brady WE, Vathipadiekal V, et al. · Lancet Oncol · 2013 · Derived
    PubMed: PMID 23261356 · NCT00551070 · Cystadenocarcinoma, Serous
  3. Dropped head syndrome: report of three cases during treatment with a MEK inhibitor.
    Chen X, Schwartz GK, DeAngelis LM, et al. · Neurology · 2013 · Derived
    PubMed: PMID 23077008 · NCT01143402 · Uveal Melanoma

2012 (3 papers)

  1. Pediatric plexiform neurofibromas: impact on morbidity and mortality in neurofibromatosis type 1.
    Prada CE, Rangwala FA, Martin LJ, et al. · J Pediatr · 2012 · Trial result
    PubMed: PMID 21996156 · NCT06620354 · Neurofibromatosis 1
  2. Phase II efficacy and pharmacogenomic study of Selumetinib (AZD6244; ARRY-142886) in iodine-131 refractory papillary thyroid carcinoma with or without follicular elements.
    Hayes DN, Lucas AS, Tanvetyanon T, et al. · Clin Cancer Res · 2012 · Background
    PubMed: PMID 22241789 · NCT00559949 · Thyroid Cancer, Papillary
  3. A phase II open-label randomized study to assess the efficacy and safety of selumetinib (AZD6244 [ARRY-142886]) versus capecitabine in patients with advanced or metastatic pancreatic cancer who have failed first-line gemcitabine therapy.
    Bodoky G, Timcheva C, Spigel DR, et al. · Invest New Drugs · 2012 · Background
    PubMed: PMID 21594619 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome

2011 (1 paper)

  1. Multi-institutional phase II study of selumetinib in patients with metastatic biliary cancers.
    Bekaii-Saab T, Phelps MA, Li X, et al. · J Clin Oncol · 2011 · Trial result
    PubMed: PMID 21519026 · NCT00553332 · Carcinoma, Hepatocellular

2010 (1 paper)

  1. The first-in-human study of the hydrogen sulfate (Hyd-sulfate) capsule of the MEK1/2 inhibitor AZD6244 (ARRY-142886): a phase I open-label multicenter trial in patients with advanced cancer.
    Banerji U, Camidge DR, Verheul HM, et al. · Clin Cancer Res · 2010 · Background
    PubMed: PMID 20179232 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome

2009 (1 paper)

  1. British HIV Association guidelines for HIV-associated malignancies 2008.
    Bower M, Collins S, Cottrill C, et al. · HIV Med · 2009 · Background
    PubMed: PMID 18705759 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome

2008 (2 papers)

  1. Reactivation of Kaposi's sarcoma-associated herpesvirus from latency requires MEK/ERK, JNK and p38 multiple mitogen-activated protein kinase pathways.
    Xie J, Ajibade AO, Ye F, et al. · Virology · 2008 · Background
    PubMed: PMID 17964626 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome
  2. Phase I pharmacokinetic and pharmacodynamic study of the oral, small-molecule mitogen-activated protein kinase kinase 1/2 inhibitor AZD6244 (ARRY-142886) in patients with advanced cancers.
    Adjei AA, Cohen RB, Franklin W, et al. · J Clin Oncol · 2008 · Background
    PubMed: PMID 18390968 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome

2007 (4 papers)

  1. Guidelines for the diagnosis and management of individuals with neurofibromatosis 1.
    Ferner RE, Huson SM, Thomas N, et al. · J Med Genet · 2007 · Trial result
    PubMed: PMID 17105749 · NCT06620354 · Neurofibromatosis 1
  2. Kaposi's sarcoma-associated herpesvirus-encoded interleukin-6 and G-protein-coupled receptor regulate angiopoietin-2 expression in lymphatic endothelial cells.
    Vart RJ, Nikitenko LL, Lagos D, et al. · Cancer Res · 2007 · Background
    PubMed: PMID 17483315 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome
  3. Management of AIDS-related Kaposi's sarcoma.
    Di Lorenzo G, Konstantinopoulos PA, Pantanowitz L, et al. · Lancet Oncol · 2007 · Background
    PubMed: PMID 17267331 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome
  4. A randomized, double-blind study of pegylated liposomal doxorubicin for the treatment of AIDS-related Kaposi's sarcoma.
    Cooley T, Henry D, Tonda M, et al. · Oncologist · 2007 · Background
    PubMed: PMID 17227906 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome

2006 (1 paper)

  1. A prognostic index for AIDS-associated Kaposi's sarcoma in the era of highly active antiretroviral therapy.
    Stebbing J, Sanitt A, Nelson M, et al. · Lancet · 2006 · Background
    PubMed: PMID 16679162 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome

2005 (1 paper)

  1. ERK1/2 and MEK1/2 induced by Kaposi's sarcoma-associated herpesvirus (human herpesvirus 8) early during infection of target cells are essential for expression of viral genes and for establishment of infection.
    Sharma-Walia N, Krishnan HH, Naranatt PP, et al. · J Virol · 2005 · Background
    PubMed: PMID 16051824 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome

2004 (2 papers)

  1. Kaposi sarcoma herpesvirus-induced cellular reprogramming contributes to the lymphatic endothelial gene expression in Kaposi sarcoma.
    Wang HW, Trotter MW, Lagos D, et al. · Nat Genet · 2004 · Background
    PubMed: PMID 15220918 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome
  2. Revised recommendations of the International Working Group for Diagnosis, Standardization of Response Criteria, Treatment Outcomes, and Reporting Standards for Therapeutic Trials in Acute Myeloid Leukemia.
    Cheson BD, Bennett JM, Kopecky KJ, et al. · J Clin Oncol · 2004 · Background
    PubMed: PMID 14673054 · NCT00588809 · Leukemia, Myeloid, Acute

2002 (1 paper)

  1. Multicenter trial of low-dose paclitaxel in patients with advanced AIDS-related Kaposi sarcoma.
    Tulpule A, Groopman J, Saville MW, et al. · Cancer · 2002 · Background
    PubMed: PMID 12115328 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome

1999 (1 paper)

  1. Regulation of human immunodeficiency virus type 1 infectivity by the ERK mitogen-activated protein kinase signaling pathway.
    Yang X, Gabuzda D · J Virol · 1999 · Background
    PubMed: PMID 10074203 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome

1989 (1 paper)

  1. Kaposi's sarcoma in the acquired immune deficiency syndrome: a proposal for uniform evaluation, response, and staging criteria. AIDS Clinical Trials Group Oncology Committee.
    Krown SE, Metroka C, Wernz JC, et al. · J Clin Oncol · 1989 · Background
    PubMed: PMID 2671281 · NCT01752569 (SCART) · Acquired Immunodeficiency Syndrome

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).