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ClinicalTrials.gov public records Last synced Oct 6, 2026, 10:14 AM EDT

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Showing 25–48 of 182 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurofibromatosis Type 1 (NF1) Clear all
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 6, 2026, 10:14 AM EDT
Recruiting Phase 1 Interventional

Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas

NCT06502171
View directory record Official record
Conditions
Neurofibromatosis 1, Plexiform Neurofibroma
Interventions
Cabozantinib Oral Tablet, Selumetinib Oral Capsule
Drug
Lead sponsor
Girish Dhall, MD
Other
Eligibility
16 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2034
U.S. locations
8
States / cities
Birmingham, Alabama • Los Angeles, California • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 6, 2026, 10:14 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1

NCT04481048
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
N-Acetyl cysteine, Placebo
Drug · Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years to 16 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2024
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 6, 2026, 10:14 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Doxycycline in Cutaneous Schwannoma (NF2)

NCT05521048
View directory record Official record
Conditions
Neurofibromatosis Type 2
Interventions
Doxycycline Injection [Doxy]
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
8 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 8, 2025 · Synced Oct 6, 2026, 10:14 AM EDT
Recruiting Phase 2 Interventional

Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1

NCT06188741
View directory record Official record
Conditions
Neurofibromatosis 1, Plexiform Neurofibroma
Interventions
Selumetinib
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
1 Year to 8 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
15
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 11 more
Source: ClinicalTrials.gov public record
Updated May 29, 2026 · Synced Oct 6, 2026, 10:14 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 6, 2026, 10:14 AM EDT
Enrolling by invitation 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 Aug 31, 2026 · Synced Oct 6, 2026, 10:14 AM 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 Aug 1, 2012 · Synced Oct 6, 2026, 10:14 AM EDT
Recruiting Phase 2 Interventional

Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)

NCT04374305
View directory record Official record
Conditions
Neurofibromatosis Type 2, Vestibular Schwannoma, Non-vestibular Schwannoma, Meningioma, Ependymoma
Interventions
Brigatinib, Neratinib, Retifanlimab, Bevacizumab
Drug
Lead sponsor
Scott R. Plotkin, MD, PhD
Other
Eligibility
12 Years and older
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
6
States / cities
Los Angeles, California • Miami, Florida • Indianopolis, Indiana + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 6, 2026, 10:14 AM EDT
Recruiting No phase listed Observational

Neurofibromatosis (NF) Registry Portal

NCT01885767
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis
Interventions
Not listed
Lead sponsor
The Children's Tumor Foundation
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2050
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 30, 2023 · Synced Oct 6, 2026, 10:14 AM 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 6, 2026, 10:14 AM EDT
Withdrawn Phase 2 Interventional

Study of Imatinib in Children With Neurofibromatosis and Airway Tumors

NCT03688568
View directory record Official record
Conditions
Neurofibroma, Plexiform
Interventions
Imatinib Mesylate
Drug
Lead sponsor
Indiana University
Other
Eligibility
6 Months to 12 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 12, 2021 · Synced Oct 6, 2026, 10:14 AM EDT
Recruiting Phase 1 Interventional

Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...

NCT02390752
View directory record Official record
Conditions
Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Promyelocytic, Acute, Sarcoma
Interventions
TURALIO(R)
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 35 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 6, 2026, 10:14 AM 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 2, 2025 · Synced Oct 6, 2026, 10:14 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 6, 2026, 10:14 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 6, 2026, 10:14 AM EDT
Withdrawn Phase 1 Interventional

Feasibility Study of Auditory Brainstem Implant in Young Children

NCT01850225
View directory record Official record
Conditions
Bilateral Cochlear Aplasia, Bilateral Cochlear Nerve Deficiency, Bilateral Cochlear Ossification Secondary to Meningitis
Interventions
Auditory brainstem implant
Device
Lead sponsor
House Research Institute
Other
Eligibility
2 Years to 5 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 18, 2015 · Synced Oct 6, 2026, 10:14 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

NCT05849662
View directory record Official record
Conditions
Leukemia, Juvenile Myelomonocytic, JMML, JCML, Neurofibromatosis 1, CBL Syndrome
Interventions
Trametinib, Azacitidine, Fludarabine, Cytarabine
Drug
Lead sponsor
Therapeutic Advances in Childhood Leukemia Consortium
Other
Eligibility
1 Month to 21 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
21
States / cities
Phoenix, Arizona • Los Angeles, California • San Francisco, California + 18 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2026 · Synced Oct 6, 2026, 10:14 AM 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 6, 2026, 10:14 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 Aug 24, 2026 · Synced Oct 6, 2026, 10:14 AM EDT
Recruiting Phase 1Phase 2 Interventional

Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas

NCT04750928
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Abemaciclib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years and older
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 6, 2026, 10:14 AM EDT
Completed No phase listed Observational

Frameshift Peptides of Children With NF1

NCT04212351
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Frameshift Array blood sample test
Genetic
Lead sponsor
Children's National Research Institute
Other
Eligibility
1 Day to 30 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
3
States / cities
Washington D.C., District of Columbia • Atlanta, Georgia • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 31, 2022 · Synced Oct 6, 2026, 10:14 AM 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 13, 2022 · Synced Oct 6, 2026, 10:14 AM EDT
Completed No phase listed Observational

The Use of a Distress Thermometer in a Pediatric Research Setting: An Exploratory Pilot Study

NCT00824278
View directory record Official record
Conditions
Cancer, HIV Infections
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
7 Years to 21 Years
Enrollment
289 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 6, 2026, 10:14 AM EDT