1 and older, any sex, with Neurofibromatosis Type 1 or Plexiform Neurofibroma. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Change From Baseline Target Tumor Volume at 12 MonthsPrimary· Approximately 12 months
To determine the objective response defined as 20% or greater tumor volume reduction. Patients will undergo volumetric assays of their target PN using MRI.
Group
Value
95% CI
Open Label Study of Binimetinib (MEK162)
27
Open Label Study of Binimetinib (MEK162)
17
Incidence of Treatment-Emergent Adverse EventsSecondary· Up to 24 months
To evaluate the toxicity of protracted binimetinib administration in this patient population. Subjects will be monitored continuously for adverse events and serious adverse events throughout the study.
Group
Value
95% CI
Open Label Study of Binimetinib (MEK162)
45
Adverse events — posted to ClinicalTrials.gov
Time frame: 24 Months.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Open Label Study of Binimetinib (MEK162)
Serious: 11/45 (24%)
Deaths: 0/45
Serious adverse events (14 terms)
Reaction
System
Open Label Study of Binime…
Anemia
Blood and lymphatic system disorders
—
Abdominal pain
Gastrointestinal disorders
—
Diarrhea
Gastrointestinal disorders
—
Gastric Hemorrhage
Gastrointestinal disorders
—
Nausea
Gastrointestinal disorders
—
Vomiting
Gastrointestinal disorders
—
Fever
General disorders
—
Anaphylaxis
Immune system disorders
—
Appendicitis
Infections and infestations
—
Skin Infection
Infections and infestations
—
Weight Loss
Metabolism and nutrition disorders
—
Suicide attempt
Psychiatric disorders
—
Hematuria
Renal and urinary disorders
—
Rash maculo-papular
Skin and subcutaneous tissue disorders
—
Other adverse events (200 terms — click to expand)
This is a phase II open label study that will evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib. The primary objective is to determine if there is an adequate level of disease responsiveness to binimetinib in children and adults with NF1 and inoperable plexiform neurofibromas. The objective response to binimetinib is defined as ≥ 20% decrease in tumor volume reduction by 12 courses.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT05554354 — Testing the Use of Fulvestrant and Binimetinib Targeted Treatment for NF1 Mutation in Hormone Receptor-Positive Metastat
· Phase 2
· terminated
NCT05564403 — Study of Chemotherapy, With or Without Binimetinib in Advanced Biliary Tract Cancers in 2nd Line Setting (A ComboMATCH T
· Phase 2
· active not recruiting
NCT04870034 — Binimetinib and Palbociclib Before Surgery for the Treatment of Operable KRAS-Positive Lung, Colorectal, or Pancreatic C
· EARLY_PHASE1
· withdrawn
NCT05554367 — Palbociclib and Binimetinib in RAS-Mutant Cancers, A ComboMATCH Treatment Trial
· Phase 2
· active not recruiting
NCT05304546 — Overcoming Primary Resistance to Immunotherapy in Metastatic Melanoma
· Phase 2
· completed
Other recruiting trials for Neurofibromatosis Type 1
Currently open trials in the same condition.
NCT06507748 — A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillome
· NA
· recruiting
NCT07221331 — Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy)
· recruiting
NCT06880991 — Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis
· recruiting
NCT05388370 — PASS of Paediatric Patients Initiating Selumetinib
· active not recruiting
NCT05309668 — Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-re
· Phase 1, PHASE2
· active not recruiting
Other University of Alabama at Birmingham trials
Trials by the same sponsor.
NCT04922229 — Comparative Effectiveness in the Management of Irreversible Pulpitis
· NA
· not yet recruiting
NCT05060380 — Feasibility of a Novel Resistance Exercise in Individuals With Osteoporosis
· NA
· withdrawn
NCT04768777 — Behavioral Intervention for Physical Activity and Sexual Dysfunction in Multiple Sclerosis
· NA
· not yet recruiting
NCT06320951 — VITAL-IMPACT: Improving Cardiometabolic Health in Black Individuals Through Therapeutic Augmentation of Cyclic Guanosine
· Phase 2
· not yet recruiting
NCT07564934 — Leveraging Extended Reality Exergaming and Telehealth to Improve Physical Activity and Health in Children With Disabilit
· Phase 2
· not yet recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by University of Alabama at Birmingham
Last refreshed: 2 May 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03231306.