Mirdametinib

DRUG15 trials

Sponsors

Springworks Therapeutics Inc., BeiGene, St. Jude Children's Research Hospital, Memorial Sloan Kettering Cancer Center, Murdoch Childrens Research Institute

Conditions

Advanced Unresectable MelanomaArteriovenous Malformation (AVM)Arteriovenous MalformationsBreast CancerBreast Cancer Stage IVCentral Nervous System TumorsCutaneous NeurofibromaCystic Hygroma

Phase 1

Study of the Safety and Pharmacokinetics of BGB-283 (Lifirafenib) and PD-0325901 (Mirdametinib) in Participants With Advanced or Refractory Solid Tumors

CompletedNCT03905148

BeiGeneSolid Tumor, Adult

Start: 2019-05-01End: 2025-10-23Updated: 2026-01-07

SJ901: Evaluation of Mirdametinib in Children, Adolescents, and Young Adults With Low-Grade Glioma

RecruitingNCT04923126

St. Jude Children's Research HospitalLow-Grade Glioma, Progressive Low-Grade Glioma, Recurrent Low-Grade Glioma

Start: 2021-06-21End: 2031-06-01Target: 132Updated: 2026-09-11

A Study of Mirdametinib on Its Own or in Combination With Fulvestrant in People With Solid Tumor Cancer

CompletedNCT05054374

Memorial Sloan Kettering Cancer CenterBreast Cancer, Breast Cancer Stage IV, HER2-negative Breast Cancer +4

Start: 2021-09-14End: 2023-10-26Updated: 2025-05-23

Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).

RecruitingNCT06159166

Johns Hopkins UniversityCutaneous Neurofibroma, Monotherapy, NF1

Start: 2024-02-12End: 2028-11-15Target: 24Updated: 2026-04-13

Phase 1/2 Study of Mirdametinib + Vinblastine for Newly Diagnosed/Previously Untreated PLGG + Activation of MAPK

RecruitingNCT06666348

St. Justine's HospitalPediatric Low-grade Glioma

Start: 2026-01-12End: 2033-04-01Target: 50Updated: 2026-08-11

A Study of Mirdametinib in Combination With Palbociclib in People With Liposarcoma

RecruitingNCT06843967

Memorial Sloan Kettering Cancer CenterDedifferentiated Liposarcoma, Liposarcoma, Myxoid Liposarcoma +6

Start: 2025-02-19End: 2028-08-19Target: 54Updated: 2026-09-16

Combination Therapy (Mirdametinib and Sirolimus) for RAS Mutated Relapsed Refractory Multiple Myeloma

SuspendedNCT06876142

National Cancer Institute (NCI)Multiple Myeloma, Relapsed and/or Refractory Multiple Myeloma (RRMM)

Start: 2026-09-23End: 2033-01-01Target: 54Updated: 2026-09-18

A Phase 1B, Open-Label Study of Mirdametinib in Infants and Toddlers with Neurofibromatosis Type 1 associated Plexiform Neurofibromas

Not yet recruitingCTIS2025-522000-24-00

Springworks Therapeutics Inc.Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN)

Target: 8Updated: 2025-12-19

To Assess the Enzyme Inducing Effects of Carbamazepine on the PK of Mirdametinib in Healthy Participants

CompletedNCT07279233

SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, GermanyHealthy, Volunteer

Start: 2025-12-15End: 2026-02-27Updated: 2026-05-08

A Study of Mirdametinib in People With Central Nervous System Tumors

RecruitingNCT07539441

Memorial Sloan Kettering Cancer CenterCentral Nervous System Tumors, Glioma

Start: 2026-04-10End: 2029-10-10Target: 26Updated: 2026-09-03

Phase 2

A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations

RecruitingNCT05983159

Murdoch Childrens Research InstituteArteriovenous Malformation (AVM), Arteriovenous Malformations, Cystic Hygroma +15

Start: 2024-09-13End: 2027-05-01Target: 50Updated: 2026-05-05

Mirdametinib in Histiocytic Disorders

RecruitingNCT06153173

Children's Hospital Medical Center, CincinnatiHistiocytic Disorders, Juvenile Xanthogranuloma (JXG), Langerhans Cell Histiocytosis (LCH) +1

Start: 2024-02-05End: 2031-03-31Target: 40Updated: 2025-09-16

Testing the Effectiveness of the Anti-cancer Drug, Mirdametinib, in Treating Relapsed, Refractory Chronic Lymphocytic Leukemia

RecruitingNCT07061951

National Cancer Institute (NCI)Recurrent Chronic Lymphocytic Leukemia, Recurrent Small Lymphocytic Lymphoma, Refractory Chronic Lymphocytic Leukemia +1

Start: 2026-05-27End: 2030-02-01Target: 20Updated: 2026-09-11

Mirdametinib in Patients With Advanced NF1-mutant Melanoma

RecruitingNCT07237100

Kevin Kim, MDAdvanced Unresectable Melanoma, Metastatic Melanoma

Start: 2025-10-13End: 2030-10-13Target: 10Updated: 2025-11-19

Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma

Not yet recruitingNCT07521657

University of Alabama at BirminghamNeurofibromatosis 1 (NF1)

Start: 2026-08-01End: 2031-08-01Target: 55Updated: 2026-04-13

Related Papers

A phase Ib/II study of mirdametinib in combination with palbociclib in patients with advanced dedifferentiated liposarcoma.

Journal of Clinical Oncology2026-05-28

A phase 1b/2 study of mirdametinib in combination with sirolimus for patients with RAS-mutated relapsed/refractory multiple myeloma.

Journal of Clinical Oncology2026-05-28

CTNI-70. RESULTS FROM THE PHASE 1 AND PHASE 1 EXPANSION COHORTS OF SJ901: A PHASE 1/2 TRIAL OF SINGLE-AGENT MIRDAMETINIB (PD-0325901) IN CHILDREN, ADOLESCENTS, AND YOUNG ADULTS WITH LOW-GRADE GLIOMA

Neuro-Oncology2024-11-011 citations

LGG-53. RESULTS FROM THE PHASE 1 AND PHASE 1 EXPANSION COHORTS OF SJ901: A PHASE 1/2 TRIAL OF SINGLE-AGENT MIRDAMETINIB (PD-0325901) IN CHILDREN, ADOLESCENTS, AND YOUNG ADULTS WITH LOW-GRADE GLIOMA

Neuro-Oncology2024-06-183 citations

LGG-25. ADVANCING RESPONSE ASSESSMENT OF LOW-GRADE GLIOMAS: IDENTIFYING VOLUME CORRELATIONS OF BI-PERPENDICULAR DIAMETER CHANGES WITH MACHINE LEARNING

Neuro-Oncology2024-06-18

LGG-22. SJ901: Phase I/II evaluation of single agent mirdametinib (PD-0325901), a brain-penetrant MEK1/2 inhibitor, for the treatment of children, adolescents, and young adults with low-grade glioma (LGG)

Neuro-Oncology2022-06-018 citations

Abstract 6415: RAF dimer inhibitor lifirafenib enhances the antitumor activity of MEK inhibitor mirdametinib in RAS mutant tumors

Cancer Research2020-08-154 citations