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Showing 1–24 of 183 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Neurofibromatosis Type I Clear all
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 22, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas

NCT00021541
View directory record Official record
Conditions
Neurofibroma, Plexiform, Neurofibromatosis Type I
Interventions
tipifarnib, placebo
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 25 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
11
States / cities
Birmingham, Alabama • Los Angeles, California • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2018 · Synced Jul 22, 2026, 2:27 PM EDT
Completed No phase listed Observational

Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1

NCT04941027
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
40 Years and older
Enrollment
1,046 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
2
States / cities
Baltimore, California • Redwood City, California
Source: ClinicalTrials.gov public record
Updated Dec 1, 2025 · Synced Jul 22, 2026, 2:27 PM EDT
Active, not recruiting No phase listed Observational

Natural History Study of Patients With Neurofibromatosis Type I

NCT00924196
View directory record Official record
Conditions
Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor, Plexiform Neurofibroma, Optic Glioma, Neurofibroma
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
4 Weeks and older
Enrollment
259 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Jul 22, 2026, 2:27 PM EDT
Recruiting No phase listed Observational

Neurofibromatosis Type 1 Tumor Early Detection Study

NCT06515860
View directory record Official record
Conditions
Neurofibromatosis Type 1, Neurofibromatosis 1, Plexiform Neurofibroma, Plexiform Neurofibromas, Malignant Peripheral Nerve Sheath Tumor, Malignant Peripheral Nerve Sheath Tumors, Atypical Neurofibroma
Interventions
Liquid biopsy for MPNST development
Diagnostic Test
Lead sponsor
David Miller
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced Jul 22, 2026, 2:27 PM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic Pain

NCT01633008
View directory record Official record
Conditions
Neurofibromatosis
Interventions
ACT Workshop
Behavioral
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years to 99 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 29, 2019 · Synced Jul 22, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma

NCT01553149
View directory record Official record
Conditions
Neurofibromatosis Type 1, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Childhood Visual Pathway Glioma
Interventions
Lenalidomide, Pharmacological Study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 21 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2023
U.S. locations
113
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 90 more
Source: ClinicalTrials.gov public record
Updated Jan 6, 2025 · Synced Jul 22, 2026, 2:27 PM EDT
Terminated Early Phase 1 Interventional Results available

Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma

NCT02282917
View directory record Official record
Conditions
Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
Interventions
AR-42
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
4
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Jul 22, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

Medical Treatment of "High-Risk" Neurofibromas

NCT00846430
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Peg-Interferon alpha-2b, Celecoxib (Celebrex), Temozolomide (temodar), Vincristine Sulfate (Oncovin)
Drug
Lead sponsor
Corewell Health West
Other
Eligibility
2 Years to 30 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2017
U.S. locations
1
States / cities
Grand Rapids, Michigan
Source: ClinicalTrials.gov public record
Updated Aug 29, 2023 · Synced Jul 22, 2026, 2:27 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST

NCT06735820
View directory record Official record
Conditions
Malignant Peripheral Nerve Sheath Tumor (MPNST), Neurofibromatosis 1 (NF1), Atypical Neurofibroma
Interventions
APG-115, Selumetinib
Drug
Lead sponsor
AeRang Kim
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jul 2, 2025 · Synced Jul 22, 2026, 2:27 PM EDT
Completed No phase listed Observational

Quality of Friendships in Children With Neurofibromatosis

NCT00508235
View directory record Official record
Conditions
Neurofibromatosis
Interventions
Questionnaire
Behavioral
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
8 Years to 18 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 31, 2012 · Synced Jul 22, 2026, 2:27 PM EDT
Terminated Phase 2 Interventional Results available

Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors

NCT03095248
View directory record Official record
Conditions
Neurofibromatosis 2, Vestibular Schwannoma, Meningioma, Ependymoma, Glioma
Interventions
Selumetinib
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
3 Years to 45 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 21, 2025 · Synced Jul 22, 2026, 2:27 PM EDT
Completed No phase listed Observational

Genetic Evaluation of NF1 and Scoliosis Patients

NCT01776125
View directory record Official record
Conditions
Neurofibromatosis 1, Scoliosis
Interventions
Cheek swab
Other
Lead sponsor
University of Minnesota
Other
Eligibility
6 Years to 65 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 31, 2019 · Synced Jul 22, 2026, 2:27 PM EDT
Terminated Phase 2 Interventional Results available

AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine

NCT00326872
View directory record Official record
Conditions
Neurofibromatosis Type 1, Plexiform Neurofibroma, Spinal Cord Neurofibroma
Interventions
Cediranib Maleate
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2016
U.S. locations
9
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2017 · Synced Jul 22, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

NCT05005845
View directory record Official record
Conditions
Cutaneous Neurofibroma, Neurofibromatosis 1
Interventions
NFX-179 gel, Vehicle gel
Drug
Lead sponsor
NFlection Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
23
States / cities
Little Rock, Arkansas • Rogers, Arkansas • Fremont, California + 20 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced Jul 22, 2026, 2:27 PM EDT
Active, not recruiting Phase 3 Interventional Results available

Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

NCT04924608
View directory record Official record
Conditions
Neurofibromatosis 1, Plexiform Neurofibroma (PN)
Interventions
Selumetinib, Placebo
Drug · Other
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years and older
Enrollment
145 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
4
States / cities
Gainesville, Florida • Rockville, Maryland • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2026 · Synced Jul 22, 2026, 2:27 PM EDT
Completed Phase 1 Interventional

Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1

NCT02211768
View directory record Official record
Conditions
Neurofibromatosis, MPNST
Interventions
MRI, FDG-PET/CT scans, [18F]-FLT-PET/CT scans
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
10 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Jul 22, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas

NCT03231306
View directory record Official record
Conditions
Neurofibromatosis Type 1, Plexiform Neurofibroma
Interventions
Binimetinib
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
1 Year and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
22
States / cities
Birmingham, Alabama • Los Angeles, California • Oakland, California + 16 more
Source: ClinicalTrials.gov public record
Updated May 1, 2025 · Synced Jul 22, 2026, 2:27 PM EDT
Completed No phase listed Observational

Adaptation and Quality of Life Among Adults With Neurofibromatosis Type I

NCT00684398
View directory record Official record
Conditions
Neurofibromatosis Type 1, Von Recklinghausen Disease, NF1
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
645 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 15, 2019 · Synced Jul 22, 2026, 2:27 PM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Open Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1

NCT04856514
View directory record Official record
Conditions
Neurofibromatosis 1, Social Skills
Interventions
PEERS
Behavioral
Lead sponsor
University of Wisconsin, Milwaukee
Other
Eligibility
12 Years to 17 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 26, 2021 · Synced Jul 22, 2026, 2:27 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations

NCT02124772
View directory record Official record
Conditions
Cancer
Interventions
Trametinib, Dabrafenib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Month to 17 Years
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
10
States / cities
Phoenix, Arizona • San Francisco, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2021 · Synced Jul 22, 2026, 2:27 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 22, 2026, 2:27 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 22, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2

NCT01419639
View directory record Official record
Conditions
Neurofibromatosis Type II
Interventions
Everolimus (RAD001) , Afinitor®
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
3 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 17, 2017 · Synced Jul 22, 2026, 2:27 PM EDT