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ClinicalTrials.gov public records Last synced Oct 22, 2026, 8:45 AM EDT

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Showing 73–96 of 122 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurofibromatosis-1 Clear all
Completed No phase listed Observational

Eligibility Screening for a NCI Pediatric Oncology Branch Research Study

NCT00026780
View directory record Official record
Conditions
Ewing Sarcoma, Osteosarcoma, Neuroblastoma, Acute Lymphoblastic Leukemia, Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 35 Years
Enrollment
1,720 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2020 · Synced Oct 22, 2026, 8:45 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial

NCT05361811
View directory record Official record
Conditions
Neurofibromatosis 1, Noonan Syndrome, Legius Syndrome, Cardiofaciocutaneous Syndrome, Costello Syndrome
Interventions
Waitlist, ACT Intervention
Behavioral
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 22, 2026, 8:45 AM EDT
Completed Phase 2 Interventional Results available

Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas

NCT00030264
View directory record Official record
Conditions
Neurofibromatosis Type 1, Precancerous Condition
Interventions
Methotrexate, Vinblastine
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 25 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2016
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 8, 2018 · Synced Oct 22, 2026, 8:45 AM 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 22, 2026, 8:45 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

NCT03190915
View directory record Official record
Conditions
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Interventions
Bone Marrow Aspiration and Biopsy, Trametinib
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
57
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 48 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Oct 22, 2026, 8:45 AM EDT
Completed Not applicable Interventional Results available

Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials

NCT03531814
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibroma, Plexiform
Interventions
Medication Event Monitoring System (MEMS^TM), Questionnaires
Behavioral · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 59 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 11, 2024 · Synced Oct 22, 2026, 8:45 AM 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 22, 2026, 8:45 AM EDT
Completed Phase 1 Interventional

Sorafenib to Treat Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

NCT00727233
View directory record Official record
Conditions
Neurofibromatosis Type I, Plexiform Neurofibroma
Interventions
Nexavar (BAY 43-9006) (Sorafenib), Toxicity, Pharmacokinetics, Pharmacodynamics, Radiographic Evaluation, QOL assessment, Neuropsychological, Bony Toxicity
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
2
States / cities
Birmingham, Alabama • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 12, 2019 · Synced Oct 22, 2026, 8:45 AM EDT
Completed No phase listed Observational

Incidence of Malignant Peripheral Nerve Sheath Tumor (MPNST) Development in Participants With Neurofibromatosis Type 1 (NF1) Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas (PN)

NCT04774289
View directory record Official record
Conditions
Neurofibromatosis 1, Peripheral Nerve Neoplasms, Malignant
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month and older
Enrollment
296 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 22, 2026, 8:45 AM 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 22, 2026, 8:45 AM 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 22, 2026, 8:45 AM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Pilot Randomized Control Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1

NCT04860362
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
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 26, 2021 · Synced Oct 22, 2026, 8:45 AM EDT
Recruiting No phase listed Observational

Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)

NCT06222203
View directory record Official record
Conditions
Neurofibromatosis 1, Nerve Sheath Neoplasms
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 120 Years
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 22, 2026, 8:45 AM EDT
Completed Phase 2 Interventional Results available

SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors

NCT03433183
View directory record Official record
Conditions
Malignant Peripheral Nerve Sheath Tumors, Neurofibromatosis 1
Interventions
Selumetinib, Sirolimus
Drug
Lead sponsor
Sarcoma Alliance for Research through Collaboration
Other
Eligibility
12 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 22, 2026, 8:45 AM 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 21, 2025 · Synced Oct 22, 2026, 8:45 AM 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 22, 2026, 8:45 AM 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 22, 2026, 8:45 AM EDT
Completed Phase 2 Interventional Results available

MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1

NCT02096471
View directory record Official record
Conditions
Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN
Interventions
PD-0325901
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
16 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
11
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2019 · Synced Oct 22, 2026, 8:45 AM 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 30, 2023 · Synced Oct 22, 2026, 8:45 AM EDT
Completed Phase 1 Interventional

Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas

NCT06120036
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Kybella, Asclera
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 85 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 21, 2025 · Synced Oct 22, 2026, 8:45 AM 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 22, 2026, 8:45 AM EDT
Active, not recruiting No phase listed Observational

Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1

NCT05677594
View directory record Official record
Conditions
Neurofibromatosis 1, Malignant Peripheral Nerve Sheath Tumor (MPNST) of Soft Tissue (Diagnosis), Atypical Neurofibroma
Interventions
Whole Body Magnetic Resonance Imaging
Diagnostic Test
Lead sponsor
Johns Hopkins University
Other
Eligibility
6 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Oct 22, 2026, 8:45 AM EDT
Completed Not applicable Interventional

A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1

NCT06262113
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Letters about NF1 Care (Content Type 1), Letters about NF1 Care (Content Type 2)
Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
Not listed
Enrollment
294 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 22, 2026, 8:45 AM EDT
Active, not recruiting Phase 3 Interventional

A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma

NCT03871257
View directory record Official record
Conditions
Low Grade Glioma, Neurofibromatosis Type 1, Visual Pathway Glioma
Interventions
Carboplatin, Magnetic Resonance Imaging, Quality-of-Life Assessment, Questionnaire Administration, Selumetinib Sulfate, Vincristine Sulfate
Drug · Procedure · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 21 Years
Enrollment
165 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
125
States / cities
Birmingham, Alabama • Mesa, Arizona • Phoenix, Arizona + 101 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 22, 2026, 8:45 AM EDT