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ClinicalTrials.gov public records Last synced Oct 21, 2026, 9:07 PM EDT

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Showing 1–24 of 122 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurofibromatosis-1 Clear all
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 8, 2025 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1

NCT04481035
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
N-acetylcysteine (NAC), Placebo
Drug · Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years to 16 Years
Enrollment
5 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2021
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Not applicable Interventional

Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosis Type 1

NCT05377008
View directory record Official record
Conditions
Neurocognitive Deficit, Mental Health Impairment, Neurofibromatosis 1
Interventions
OPTIMAL-SSI
Behavioral
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years to 16 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 22, 2023 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Phase 1 Interventional

Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1)

NCT00352599
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Lovastatin, placebo pill
Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
10 Years to 50 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 22, 2016 · Synced Oct 21, 2026, 9:07 PM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Stem Cells in NF1 Patients With Tumors of the Central Nervous System

NCT03332030
View directory record Official record
Conditions
Neurofibromatosis Type 1, Tumors of the Central Nervous System
Interventions
Collection of Stem Cells
Diagnostic Test
Lead sponsor
Roger Packer
Other
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Oct 21, 2026, 9:07 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 2, 2021 · Synced Oct 21, 2026, 9:07 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 10, 2026 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Phase 2 Interventional Results available

Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T

NCT02332902
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Everolimus
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 20, 2023 · Synced Oct 21, 2026, 9:07 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 4, 2025 · Synced Oct 21, 2026, 9:07 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 17, 2026 · Synced Oct 21, 2026, 9:07 PM EDT
Active, not recruiting No phase listed Observational

Natural History Study of Cutaneous Neurofibromas in People With NF1

NCT05581511
View directory record Official record
Conditions
Neurofibromatosis Type 1, Neurofibromatosis 1, Neurofibromatosis (Nonmalignant), Cutaneous Neurofibroma
Interventions
Evaluation of the natural history of cutaneous neurofibromas
Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
1 Year to 100 Years
Enrollment
500 participants
Timeline
2021 – 2029
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Oct 21, 2026, 9:07 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
11
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Oct 21, 2026, 9:07 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 30, 2015 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Phase 2 Interventional Results available

Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas

NCT00076102
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibroma, Plexiform
Interventions
Pirfenidone
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 21 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
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 23, 2018 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Phase 2 Interventional Results available

A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas

NCT00634270
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Sirolimus
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
3 Years to 75 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
9
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Nov 17, 2017 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Not applicable Interventional

Resiliency Training for Patients With Neurofibromatosis Via Videoconferencing With Skype

NCT02298270
View directory record Official record
Conditions
Neurofibromatosis, Neurofibromatosis I, Neurofibromatosis 2, Schwannomatosis
Interventions
The Relaxation Response Resiliency Program (3RP) via Skype
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 23, 2016 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Early Phase 1 Interventional

Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

NCT00657202
View directory record Official record
Conditions
Neurofibromatosis Type 1, Cutaneous Neurofibromas
Interventions
Ranibizumab
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 7, 2016 · Synced Oct 21, 2026, 9:07 PM EDT
Active, not recruiting Phase 1 Interventional

Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas

NCT05101148
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Selumetinib
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
12 Years to 17 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
2
States / cities
Rochester, Minnesota • Akron, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Neurobiology and Treatment of Reading Disability in NF-1

NCT00624234
View directory record Official record
Conditions
Neurofibromatosis Type 1, Reading Disabilities
Interventions
Tutoring Program I, Tutoring Program II
Behavioral
Lead sponsor
Vanderbilt University
Other
Eligibility
8 Years to 17 Years
Enrollment
184 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2017
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 8, 2025 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Bioavailability and Food Effect of Selumetinib (AZD6244) in Healthy Male Participants

NCT03649165
View directory record Official record
Conditions
Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs), Healthy Participants
Interventions
Treatment A, Treatment B, Treatment C, Treatment D, Acetaminophen
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 8, 2018 · Synced Oct 21, 2026, 9:07 PM EDT
Completed No phase listed Observational

Neurofibromatosis Type 1 Brain Tumor Genetic Risk

NCT01707836
View directory record Official record
Conditions
Neurofibromatosis Type 1, Pediatric Brain Tumor
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 18 Years
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 9, 2017 · Synced Oct 21, 2026, 9:07 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 6, 2026 · Synced Oct 21, 2026, 9:07 PM EDT
Completed No phase listed Observational

Spinal Abnormalities in Neurofibromatosis Type 1 (NF1)

NCT00844129
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
University of Utah
Other
Eligibility
6 Years to 9 Years
Enrollment
112 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 3, 2018 · Synced Oct 21, 2026, 9:07 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1

NCT00853580
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Lovastatin ™, placebo
Drug · Device
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
8 Years to 15 Years
Enrollment
146 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2016
U.S. locations
11
States / cities
Birmingham, Alabama • Los Angeles, California • Washington D.C., District of Columbia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2018 · Synced Oct 21, 2026, 9:07 PM EDT