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Showing 1–24 of 183 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Neurofibromatosis 1 Clear all
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 Aug 3, 2026 · Synced Sep 23, 2026, 10:02 PM EDT
Terminated Phase 2 Interventional

Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)

NCT01968590
View directory record Official record
Conditions
Neurofibromatosis Type 1 (NF1)
Interventions
Cholecalciferol
Drug
Lead sponsor
University of Utah
Other
Eligibility
25 Years to 40 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
2
States / cities
Cincinnati, Ohio • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 24, 2022 · Synced Sep 23, 2026, 10:02 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 Sep 23, 2026, 10:02 PM EDT
Completed Phase 3 Interventional Results available

Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain

NCT02471339
View directory record Official record
Conditions
Neurofibromatosis Type 1, Plexiform Neurofibromas
Interventions
Acceptance and Commitment Therapy (ACT)
Behavioral
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
16 Years to 59 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 13, 2022 · Synced Sep 23, 2026, 10:02 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Everolimus for Children With NF1 Chemotherapy-Refractory Radiographic Progressive Low Grade Gliomas

NCT01158651
View directory record Official record
Conditions
Glioma
Interventions
RAD001 (Everolimus)
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
1 Year to 21 Years
Enrollment
23 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2017
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 Feb 4, 2020 · Synced Sep 23, 2026, 10:02 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 Sep 23, 2026, 10:02 PM EDT
Completed Phase 2 Interventional Results available

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

NCT04435665
View directory record Official record
Conditions
Neurofibromatosis 1, Cutaneous Neurofibroma
Interventions
NFX-179 Gel, Vehicle Gel
Drug
Lead sponsor
NFlection Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
6
States / cities
Fremont, California • New Brighton, Minnesota • Rochester, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 8, 2022 · Synced Sep 23, 2026, 10:02 PM EDT
Terminated No phase listed Observational

US Selumetinib Registry

NCT05683678
View directory record Official record
Conditions
Neurofibromatosis Type 1, Plexiform Neurofibromas
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 18 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
10
States / cities
Phoenix, Arizona • Los Angeles, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2025 · Synced Sep 23, 2026, 10:02 PM EDT
Terminated Phase 2 Interventional

Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

NCT02728388
View directory record Official record
Conditions
NEUROFIBROMATOSIS 1
Interventions
aminolevulinic acid
Drug
Lead sponsor
Donald Basel
Other
Eligibility
14 Years to 30 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Sep 23, 2026, 10:02 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 Sep 23, 2026, 10:02 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 Sep 23, 2026, 10:02 PM EDT
Completed Not applicable Interventional Results available

Innovation in the Treatment of Persistent Pain in Adults With NF1: Implementation of the iCanCope Mobile Application- Clinical Trial

NCT04561765
View directory record Official record
Conditions
Neurofibromatosis 1, Chronic Pain
Interventions
iCanCope, iCanCope+Contingency Management
Device
Lead sponsor
Yale University
Other
Eligibility
18 Years to 72 Years
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 29, 2025 · Synced Sep 23, 2026, 10:02 PM EDT
Completed Early Phase 1 Interventional Results available

Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas

NCT01275586
View directory record Official record
Conditions
Neurofibromatosis, NF1, Neurofibromas
Interventions
Tasigna
Drug
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Apr 11, 2017 · Synced Sep 23, 2026, 10:02 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors

NCT01362803
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibromatosis Type 1, NF1, Neurofibroma, Plexiform
Interventions
AZD6244
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 18 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2032
U.S. locations
4
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Sep 23, 2026, 10:02 PM EDT
No Longer Available No phase listed Expanded access

Compassionate Use Arm - ABI541 ABI for 10 NF2 Patients

NCT02589912
View directory record Official record
Conditions
Neurofibromatosis Type 2, Severe Profound Sensorineural Hearing Loss
Interventions
Nucleus Profile ABI541 Auditory Brainstem Implant
Device
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
12 Years to 80 Years
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 5, 2019 · Synced Sep 23, 2026, 10:02 PM EDT
Terminated Not applicable Interventional Results available

Auditory Brainstem Implant (ABI) in Adult Non-Neurofibromatosis Type 2 Subjects

NCT01736267
View directory record Official record
Conditions
Bilateral Hearing Loss for Causes Other Than Tumors
Interventions
Nucleus 24 (discontinued) and ABI541 Auditory Brainstem Implants (ABI)
Device
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 5, 2020 · Synced Sep 23, 2026, 10:02 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma

NCT01089101
View directory record Official record
Conditions
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Neurofibromatosis Type 1, Recurrent Visual Pathway Glioma, Refractory Neurofibromatosis Type 1, Refractory Visual Pathway Glioma
Interventions
Biospecimen Collection, Selumetinib
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 21 Years
Enrollment
217 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2027
U.S. locations
17
States / cities
Los Angeles, California • Palo Alto, California • San Francisco, California + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 20, 2026 · Synced Sep 23, 2026, 10:02 PM EDT
Active, not recruiting Not applicable Interventional

Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1

NCT03820778
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibroma, Atypical Neurofibroma, Atypical Neurofibromatosis, Plexiform Neurofibroma, Von Recklinghausen Disease
Interventions
Whole Body MRI
Diagnostic Test
Lead sponsor
Children's National Research Institute
Other
Eligibility
8 Years to 30 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 1, 2021 · Synced Sep 23, 2026, 10:02 PM EDT
Completed Phase 1 Interventional

Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas

NCT00053937
View directory record Official record
Conditions
Neurofibromatosis Type 1, Precancerous Condition
Interventions
pirfenidone
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 21 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
15
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2015 · Synced Sep 23, 2026, 10:02 PM EDT
Suspended Not applicable Interventional

Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1-associated Cutaneous Neurofibromas (cNFs)

NCT06934928
View directory record Official record
Conditions
Neurofibromatosis 1 (NF1), Neurofibromatosis Type I
Interventions
GentleMax Pro (with skin cooling), GentleMax Pro (without skin cooling)
Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Sep 23, 2026, 10:02 PM EDT
Completed Phase 2 Interventional Results available

Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma

NCT02839720
View directory record Official record
Conditions
Cutaneous Neurofibroma, Neurofibromatosis Type 1, Optic Nerve Glioma
Interventions
Laboratory Biomarker Analysis, Selumetinib Sulfate
Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
2
States / cities
Birmingham, Alabama • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 5, 2023 · Synced Sep 23, 2026, 10:02 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1

NCT05912400
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Blood draw, FACIT-F and Pain Scales
Diagnostic Test · Other
Lead sponsor
University of Arkansas
Other
Eligibility
18 Years and older
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated May 7, 2025 · Synced Sep 23, 2026, 10:02 PM EDT
Completed Phase 1 Interventional

Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

NCT04730583
View directory record Official record
Conditions
Cutaneous Neurofibroma
Interventions
Kybella, 1064nm laser, 755nm Alexandrite Laser
Drug · Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 85 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 20, 2025 · Synced Sep 23, 2026, 10:02 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas

NCT02831257
View directory record Official record
Conditions
Neurofibromatosis 2, Meningioma
Interventions
AZD2014
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2020
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 12, 2022 · Synced Sep 23, 2026, 10:02 PM EDT