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Showing 1–23 of 23 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: NF1 Mutation Clear all
Active, not recruiting Phase 2 Interventional Results available

Testing Trametinib as a Potential Targeted Treatment in Cancers With NF1 Genetic Changes (MATCH-Subprotocol S1)

NCT04439318
View directory record Official record
Conditions
Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma, Refractory Malignant Solid Neoplasm, Refractory Multiple Myeloma
Interventions
Trametinib Dimethyl Sulfoxide
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 21, 2026, 6:54 PM EDT
Terminated Phase 2 Interventional Results available

Testing the Use of Fulvestrant and Binimetinib Targeted Treatment for NF1 Mutation in Hormone Receptor-Positive Metastatic Breast Cancer (A ComboMATCH Treatment Trial)

NCT05554354
View directory record Official record
Conditions
Anatomic Stage IV Breast Cancer AJCC v8, Metastatic HER2-Negative Breast Carcinoma, Metastatic Hormone Receptor-Positive Breast Carcinoma
Interventions
Binimetinib, Biopsy Procedure, Biospecimen Collection, Bone Scan, Computed Tomography, Echocardiography Test, Fulvestrant, Magnetic Resonance Imaging, Multigated Acquisition Scan
Drug · Procedure
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
192
States / cities
Birmingham, Alabama • Kingman, Arizona • Arroyo Grande, California + 155 more
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Jul 21, 2026, 6:54 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

RASopathy Biorepository

NCT04395495
View directory record Official record
Conditions
RAS Mutation, Neurofibromatosis 1, Noonan Syndrome, Noonan Syndrome With Multiple Lentigines, Noonan Neurofibromatosis Syndrome, Cardiofaciocutaneous Syndrome, Costello Syndrome, Legius Syndrome, Smith-Kingsmore Syndrome, MTOR Gene Mutation, GATOR-1 Gene Mutation, SYNGAP1-Related Intellectual Disability, DLG4, MAPK1 Gene Mutation
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2065
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Jul 21, 2026, 6:54 PM EDT
Recruiting Phase 1 Interventional

Ulixertinib/Palbociclib in Patients With Advanced Pancreatic and Other Solid Tumors

NCT03454035
View directory record Official record
Conditions
Tumor, Solid, Pancreatic Cancer, Melanoma
Interventions
Ulixertinib, Palbociclib
Drug
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years to 99 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Jul 21, 2026, 6:54 PM EDT
Recruiting Phase 2 Interventional

MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies

NCT05735717
View directory record Official record
Conditions
Hematologic Malignancy, Acute Leukemia, Remission, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, AML, TP53, Intrachromosomal Amplification of Chromosome 21, Cytogenetic Abnormality, CNS Leukemia, Minimal Residual Disease, Myelodysplasia, Juvenile Myelomonocytic Leukemia, Somatic Mutation, PTPN11 Gene Mutation, N-RAS Gene Amplification, Neurofibromatosis 1, NF1 Mutation, CBL Gene Mutation, Monosomy 7, Chromosome Abnormality, Fetal Hemoglobin, Lymphoblastic Lymphoma, High Grade Non-Hodgkin's Lymphoma, Adult
Interventions
Fludarabine, Busulfan, Melphalan, Rituximab, Levetiracetam, Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells, Thymoglobulin, Cyclophosphamide
Drug · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 60 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced Jul 21, 2026, 6:54 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-Esc/Exp RMC4630 & Cobi in Relapsed/Refractory Solid Tumors & RMC4630& Osi in EGFR+ Locally Adv/Meta NSCLC

NCT03989115
View directory record Official record
Conditions
Solid Tumor
Interventions
RMC-4630, Cobimetinib, Drug: Osimertinib
Drug
Lead sponsor
Revolution Medicines, Inc.
Industry
Eligibility
18 Years and older
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
21
States / cities
Scottsdale, Arizona • Duarte, California • Orange, California + 18 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2023 · Synced Jul 21, 2026, 6:54 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Testing Whether Cancers With Specific Mutations Respond Better to Glutaminase Inhibitor, Telaglenastat Hydrochloride, Anti-Cancer Treatment, BeGIN Study

NCT03872427
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm, NF1 Mutation Positive Malignant Peripheral Nerve Sheath Tumor, Unresectable Malignant Solid Neoplasm
Interventions
Biospecimen Collection, Computed Tomography, Magnetic Resonance Imaging, Pharmacodynamic Study, Positron Emission Tomography, Telaglenastat Hydrochloride
Procedure · Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
31
States / cities
Birmingham, Alabama • Aventura, Florida • Coral Gables, Florida + 23 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Jul 21, 2026, 6:54 PM EDT
Recruiting Phase 3 Interventional

A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma

NCT04166409
View directory record Official record
Conditions
Low Grade Astrocytoma, Low Grade Glioma, Metastatic Low Grade Astrocytoma, Metastatic Low Grade Glioma, WHO Grade 1 Glioma, WHO Grade 2 Glioma
Interventions
Biospecimen Collection, Carboplatin, Echocardiography Test, Magnetic Resonance Imaging, Questionnaire Administration, Selumetinib Sulfate, Vincristine Sulfate
Procedure · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 21 Years
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
124
States / cities
Birmingham, Alabama • Mesa, Arizona • Phoenix, Arizona + 101 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Jul 21, 2026, 6:54 PM EDT
Recruiting Phase 1 Interventional

PAS-004 in Patients With Advanced Solid Tumors

NCT06299839
View directory record Official record
Conditions
RAS Mutation, NF1 Mutation, RAF Mutation, Advanced Solid Tumors
Interventions
PAS-004 Capsules, PAS-004 Tablets
Drug
Lead sponsor
Pasithea Therapeutics Corp.
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
4
States / cities
Austin, Texas • Irving, Texas • San Antonio, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 2, 2025 · Synced Jul 21, 2026, 6:54 PM EDT
Approved For Marketing No phase listed Expanded access

An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1

NCT03259633
View directory record Official record
Conditions
NF type1 With Inoperable Plexiform Neurofibromas
Interventions
Selumetinib
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
2 Years to 130 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Apr 30, 2020 · Synced Jul 21, 2026, 6:54 PM EDT
Terminated Phase 1 Interventional

Study of DCC-3084 in Participants With Advanced Malignancies Driven by the Mitogen-Activated Protein Kinase (MAPK) Pathway

NCT06287463
View directory record Official record
Conditions
Advanced Solid Tumor, RAF Mutation, RAS Mutation, NF1 Mutation, Non-Small Cell Lung Cancer, Pancreatic Ductal Adenocarcinoma, Melanoma, BRAF Gene Mutation, CRAF Gene Mutation, Castration-Resistant Prostate Cancer (CRPC)
Interventions
DCC-3084
Drug
Lead sponsor
Deciphera Pharmaceuticals, LLC
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
9
States / cities
Los Angeles, California • San Francisco, California • Denver, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Jul 21, 2026, 6:54 PM EDT
Terminated Phase 2Phase 3 Interventional

Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas

NCT05130866
View directory record Official record
Conditions
Neurofibromatosis Type 2
Interventions
REC-2282, Placebo
Drug
Lead sponsor
Recursion Pharmaceuticals Inc.
Industry
Eligibility
12 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
14
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2025 · Synced Jul 21, 2026, 6:54 PM EDT
Completed Phase 2 Interventional Results available

Everolimus in Patients With Advanced Solid Malignancies With TSC1, TSC2, NF1, NF2, or STK11 Mutations

NCT02352844
View directory record Official record
Conditions
Solid Malignancy, Solid Tumor
Interventions
Everolimus
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 1, 2018 · Synced Jul 21, 2026, 6:54 PM EDT
Completed Phase 2 Interventional Results available

Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults

NCT02101736
View directory record Official record
Conditions
NF1, Neurofibromatosis, Plexiform Neurofibromas
Interventions
Cabozantinib
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
3 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
12
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated May 23, 2023 · Synced Jul 21, 2026, 6:54 PM EDT
Recruiting Phase 2 Interventional

Mirdametinib in Patients With Advanced NF1-mutant Melanoma

NCT07237100
View directory record Official record
Conditions
Advanced Unresectable Melanoma, Metastatic Melanoma
Interventions
Mirdametinib
Drug
Lead sponsor
Kevin Kim, MD
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Nov 18, 2025 · Synced Jul 21, 2026, 6:54 PM EDT
Terminated Phase 1 Interventional

PF-07284892 in Participants With Advanced Solid Tumors

NCT04800822
View directory record Official record
Conditions
Solid Tumor
Interventions
PF-07284892, lorlatinib, binimetinib, cetuximab, encorafenib
Drug · Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
11
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Detroit, Michigan + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Jul 21, 2026, 6:54 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Compare Treatment With the Drug Selumetinib Alone Versus Selumetinib and Vinblastine in Patients With Recurrent or Progressive Low-Grade Glioma

NCT04576117
View directory record Official record
Conditions
Recurrent Low Grade Astrocytoma, Recurrent WHO Grade 2 Glioma, Refractory Low Grade Astrocytoma, Refractory Low Grade Glioma, Refractory WHO Grade 1 Glioma
Interventions
Biospecimen Collection, Magnetic Resonance Imaging, Quality-of-Life Assessment, Questionnaire Administration, Selumetinib Sulfate, Vinblastine Sulfate
Procedure · Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 25 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
91
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Loma Linda, California + 77 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Jul 21, 2026, 6:54 PM EDT
Active, not recruiting Phase 1 Interventional

A Study of Avutometinib for People With Solid Tumor Cancers

NCT06104488
View directory record Official record
Conditions
Refractory Cancer, CNS Tumors, CNS Tumor, Adult, CNS Tumor, Childhood, MAP Kinase Family Gene Mutation, NF1, Plexiform Neurofibroma, Low-grade Glioma, Optic Pathway Gliomas, Neuroblastoma, Primary Brain Tumor, Solid Tumor, Solid Tumor, Adult, Solid Carcinoma, Central Nervous System Tumor
Interventions
Avutometinib
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
3 Years to 30 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
2
States / cities
Atlanta, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Jul 21, 2026, 6:54 PM EDT
Recruiting Phase 1 Interventional

PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas

NCT06961565
View directory record Official record
Conditions
NF1 Mutation, Neurofibroma Plexiform, Neurofibroma, Plexiform, Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs), Neurofibromatosis Type 1 (NF1)
Interventions
PAS-004 Tablets
Drug
Lead sponsor
Pasithea Therapeutics Corp.
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Dec 3, 2025 · Synced Jul 21, 2026, 6:54 PM EDT
Active, not recruiting Phase 4 Interventional

Pediatric Long-Term Follow-up and Rollover Study

NCT03975829
View directory record Official record
Conditions
Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood, Anaplastic Oligodendroglioma, Glioblastoma, Pilocytic Astrocytoma, Giant Cell Astrocytoma, Pleomorphic Xanthoastrocytoma, Anaplastic Pleomorphic Xanthoastrocytoma, Angiocentric Glioma, Chordoid Glioma of Third Ventricle, Gangliocytoma, Ganglioglioma, Anaplastic Ganglioglioma, Dysplastic Gangliocytoma of Cerebrellum, Desmoplastic Infantile Astrocytoma and Ganglioglioma, Papillary Glioneuronal Tumor, Rosette-forming Glioneuronal Tumor, Central Neurocytoma, Extraventricular Neurocytoma, Cerebellar Liponeurocytoma, Neurofibromatosis Type 1
Interventions
dabrafenib, trametinib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Year to 99 Years
Enrollment
163 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
12
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 21, 2026, 6:54 PM EDT
Terminated Phase 2 Interventional Results available

A Basket Trial of an ERK1/2 Inhibitor (LY3214996) in Combination With Abemaciclib.

NCT04534283
View directory record Official record
Conditions
Cancer, Cancer Metastatic, BRAF V600E, MEK1 Gene Mutation, MEK2 Gene Mutation, ERK Mutation, RAF1 Gene Mutation
Interventions
Abemaciclib, LY3214996
Drug
Lead sponsor
Anita Turk
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Aug 6, 2024 · Synced Jul 21, 2026, 6:54 PM EDT
Terminated Phase 1 Interventional

Safety and Effectiveness of ABM-168 in Adults with Advanced Solid Tumors.

NCT05831995
View directory record Official record
Conditions
Advanced Solid Tumor, RAS Mutation, RAF Mutation, NF1 Mutation
Interventions
ABM-168
Drug
Lead sponsor
ABM Therapeutics Corporation
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
6
States / cities
San Francisco, California • Indianapolis, Indiana • New Brunswick, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 5, 2024 · Synced Jul 21, 2026, 6:54 PM EDT
Terminated Phase 2 Interventional Results available

Study of Mutation-Targeted Therapy With Sunitinib or Everolimus in People With Advanced Low- or Intermediate-Grade Neuroendocrine Tumors of the Gastrointestinal Tract and Pancreas With or Without Cytoreductive Surgery

NCT02315625
View directory record Official record
Conditions
Neuroendocrine Tumors, Neuroendocrine Carcinoma, Neuroendocrine Neoplasms, Carcinoma, Neuroendocrine, Neuroendocrine Tumors of the Gastrointestinal Tract and Pancreas
Interventions
Sunitinib, Everolimus
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2020 · Synced Jul 21, 2026, 6:54 PM EDT