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ClinicalTrials.gov public records Last synced Oct 13, 2026, 5:38 PM EDT

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Showing 73–96 of 184 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurofibromatosis 1 (NF1) Clear all
Terminated Not applicable Interventional Results available

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

NCT01864291
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 Months to 17 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 2, 2020 · Synced Oct 13, 2026, 5:38 PM EDT
Available No phase listed Expanded access

Individual Patient Compassionate Use of Mirdametinib

NCT05028166
View directory record Official record
Conditions
Neurofibromatosis Type 1-Associated Plexiform Neurofibromas, Histiocytic Neoplasm, Other MAP-K Pathway Driven Diseases
Interventions
Mirdametinib (MEK Inhibitor)
Drug
Lead sponsor
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Industry
Eligibility
2 Years and older
U.S. locations
1
States / cities
Billerica, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 13, 2026, 5:38 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 Oct 13, 2026 · Synced Oct 13, 2026, 5:38 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 5, 2020 · Synced Oct 13, 2026, 5:38 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 6, 2023 · Synced Oct 13, 2026, 5:38 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 13, 2026, 5:38 PM EDT
Recruiting Phase 2 Interventional

Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas

NCT05253131
View directory record Official record
Conditions
MPNST, NF1, Sarcoma
Interventions
Selumetinib + ZEN-3694 ± Durvalumab
Combination Product
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years to 99 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 13, 2026, 5:38 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 18, 2025 · Synced Oct 13, 2026, 5:38 PM EDT
Completed Phase 1 Interventional

Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1)

NCT00901849
View directory record Official record
Conditions
Low-grade Gliomas
Interventions
Tarceva and Rapamycin
Drug
Lead sponsor
Roger Packer
Other
Eligibility
Up to 21 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Sep 16, 2014 · Synced Oct 13, 2026, 5:38 PM EDT
Completed Not applicable Interventional

Resiliency Training for Patients With NF2 Via Videoconferencing With Skype

NCT02811718
View directory record Official record
Conditions
Neurofibromatosis 2
Interventions
Stress Management Group 1, Stress Management Group 2
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 17, 2018 · Synced Oct 13, 2026, 5:38 PM EDT
Recruiting No phase listed Observational

Familial Investigations of Childhood Cancer Predisposition

NCT03050268
View directory record Official record
Conditions
Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Oct 13, 2026, 5:38 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 May 1, 2020 · Synced Oct 13, 2026, 5:38 PM EDT
Completed No phase listed Observational

Study of Plexiform Neurofibromas in Neurofibromatosis Type 1

NCT00006435
View directory record Official record
Conditions
Plexiform Neurofibromas, NF1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 100 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 4, 2020 · Synced Oct 13, 2026, 5:38 PM EDT
Terminated Not applicable Interventional Results available

Fludeoxyglucose F 18 Positron Emission Tomography and Magnetic Resonance Perfusion Imaging in Patients With Neurofibromatosis 1 and Plexiform Neurofibroma

NCT00060008
View directory record Official record
Conditions
Neurofibromatosis Type 1, Precancerous Condition
Interventions
fludeoxyglucose F 18, gadopentetate dimeglumine
Radiation
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 25 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2011
U.S. locations
2
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 4, 2014 · Synced Oct 13, 2026, 5:38 PM EDT
Recruiting No phase listed Observational

Natural History Study of Patients With Neurofibromatosis Type 2

NCT00598351
View directory record Official record
Conditions
Neurofibromatosis
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
8 Years to 75 Years
Enrollment
269 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 13, 2026, 5:38 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 18, 2017 · Synced Oct 13, 2026, 5:38 PM EDT
Recruiting Not applicable Interventional

Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence

NCT04763109
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Whole-body Magnetic Resonance Imaging
Other
Lead sponsor
Nicole Baca
Other
Eligibility
5 Years to 17 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 13, 2026, 5:38 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Analysis of Data Collected From Individuals Administered Neurobehavioral Assessments

NCT01445314
View directory record Official record
Conditions
HIV, Brain Tumors, Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month and older
Enrollment
598 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 16, 2024 · Synced Oct 13, 2026, 5:38 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 2, 2025 · Synced Oct 13, 2026, 5:38 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 30, 2019 · Synced Oct 13, 2026, 5:38 PM EDT
Recruiting Phase 2 Interventional

A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1

NCT06541847
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
HLX-1502
Drug
Lead sponsor
Healx Limited
Industry
Eligibility
12 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
14
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 13, 2026, 5:38 PM EDT
Completed No phase listed Observational

Neurofibromatosis Type 1 (NF1) and Tibial Dysplasia

NCT00303368
View directory record Official record
Conditions
Neurofibromatosis Type 1, Tibial Dysplasia
Interventions
Not listed
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
Not listed
Enrollment
395 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2016
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Feb 25, 2019 · Synced Oct 13, 2026, 5:38 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

NCT05309668
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Selumetinib granule formulation, Selumetinib capsule formulation
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
1 Year to 6 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
4
States / cities
Akron, Ohio • Philadelphia, Pennsylvania • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 13, 2026, 5:38 PM EDT
Active, not recruiting Phase 2 Interventional

MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas

NCT02407405
View directory record Official record
Conditions
Neurofibromatosis 1 (NF1), Plexiform Neurofibromas (PN)
Interventions
Selumetinib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 13, 2026, 5:38 PM EDT