Clinical trial · Interventional
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
A Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients With Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) That Are Causing Significant Morbidity
NCT03962543CI-TRIAL-00117547ReNeuactive not recruitingPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901). Eligible participants may continue in a long-term follow-up phase.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neurofibromatosis Type 1 (NF1) | — | UNRESOLVED | — |
| Plexiform Neurofibroma | Plexiform Neurofibroma | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Mirdametinib (PD-0325901) oral capsule or dispersible tablet | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Mirdametinib (PD-0325901)
- description
- Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
- interventionNames
- Drug: Mirdametinib (PD-0325901) oral capsule or dispersible tablet
Primary outcomes (1)
- measure
- Confirmed Objective Response Rate at the End of the Treatment Phase.
- timeFrame
- Up to 24 months
- description
- Response will be determined by a blinded centralized review of volumetric MRI. The confirmed objective response rate (complete or partial response) by the end of Treatment Phase (i.e., Cycle 24) is defined as the proportion of participants who have a confirmed ≥ 20% reduction in target tumor volume as compared to baseline as assessed by a BICR, and the response needs to be confirmed by BICR in a consecutive tumor assessment within 2 - 6 months. Partial response is defined as a ≥ 20% reduction in target tumor volume from baseline. Complete response is defined as the complete resolution of the target tumor.
Secondary outcomes (5)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 2 Years
Show eligibility criteria text
Key Inclusion Criteria: * Participant has documented NF1 mutation or a diagnosis of neurofibromatosis type 1 (NF1) using National Institute of Health (NIH) Consensus Conference criteria inclusive of the presence of a plexiform neurofibroma (PN). * Participant has a PN that is causing significant morbidity. * Participant has a PN that cannot be completely surgically removed. * Participant has a target tumor that is amenable to volumetric MRI analysis. * Participant is willing to undergo a tumor biopsy pre and post treatment if ≥ 18 years of age. * Participant has adequate organ and bone marrow function. Key Exclusion Criteria: * Participant has abnormal liver function or history of liver disease. * Participant has lymphoma, leukemia or any malignancy within the past 5 years (except for resected basal/squamous skin carcinomas without metastases within 3 years). * Participant has breast cancer within 10 years. * Participant has active optic glioma or other low-grade glioma requiring treatment. * Participant has abnormal QT interval corrected or other heart disease within 6 months. * Participant has a history of retinal pathology, risk factors for retinal vein occlusion or has a history of glaucoma. * Participant has known malabsorption syndrome or gastrointestinal conditions that would impair absorption of mirdametinib (PD-0325901). * Participant has received NF1 PN-targeted therapy within 45 days. * Participant previously received or is currently receiving therapy with mirdametinib (PD-0325901) or any other MEK1/2 inhibitor. * Participant has received radiation therapy within 6 months or has received radiation to the orbit at any time. * Participant is unable to undergo or tolerate MRI. * Participant has active bacterial, fungal or viral infection. * Participant has experienced other severe acute or chronic medical or psychiatric conditions within 1 year.
References
Publications (1)
- DERIVEDMoertel CL, Hirbe AC, Shuhaiber HH, Bielamowicz K, Sidhu A, Viskochil D, Weber MD, Lokku A, Smith LM, Foreman NK, Hajjar FM, McNall-Knapp RY, Weintraub L, Antony R, Franson AT, Meade J, Schiff D, Walbert T, Ambady P, Bota DA, Campen CJ, Kaur G, Klesse LJ, Maraka S, Moots PL, Nevel K, Bornhorst M, Aguilar-Bonilla A, Chagnon S, Dalvi N, Gupta P, Khatib Z, Metrock LK, Nghiemphu PL, Roberts RD, Robison NJ, Sadighi Z, Stapleton S, Babovic-Vuksanovic D, Gershon TR; ReNeu Trial Investigators; ReNeu Study Investigators. ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma. J Clin Oncol. 2025 Feb 20;43(6):716-729. doi: 10.1200/JCO.24.01034. Epub 2024 Nov 8. PMID 39514826