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ClinicalTrials.gov public records Last synced Oct 2, 2026, 6:56 AM EDT

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Showing 1–24 of 181 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurofibromatosis Type 1 (NF1) Clear all
Completed Phase 2 Interventional Results available

R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas

NCT00021541
View directory record Official record
Conditions
Neurofibroma, Plexiform, Neurofibromatosis Type I
Interventions
tipifarnib, placebo
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 25 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
11
States / cities
Birmingham, Alabama • Los Angeles, California • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 17, 2018 · Synced Oct 2, 2026, 6:56 AM 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 2, 2026, 6:56 AM 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 2, 2026, 6:56 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 2, 2026, 6:56 AM 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 2, 2026, 6:56 AM 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 2, 2026, 6:56 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 2, 2026, 6:56 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1

NCT04954001
View directory record Official record
Conditions
Neurofibromatosis 1, Plexiform Neurofibroma, NF1
Interventions
FCN-159
Drug
Lead sponsor
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Industry
Eligibility
2 Years to 70 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • St. Petersburg, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 12, 2024 · Synced Oct 2, 2026, 6:56 AM EDT
Recruiting No phase listed Observational

Glioma Developmental and HyperActive Ras Tumor (DHART) Board

NCT05489783
View directory record Official record
Conditions
Glioma
Interventions
Not listed
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
7
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 2, 2026, 6:56 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 2, 2026, 6:56 AM 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 25, 2022 · Synced Oct 2, 2026, 6:56 AM 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 4, 2025 · Synced Oct 2, 2026, 6:56 AM EDT
Active, not recruiting Phase 2 Interventional Accepts healthy volunteers

Interventions for Reading Disabilities in NF1

NCT02964884
View directory record Official record
Conditions
Neurofibromatosis Type 1, Learning Disability, Reading Disability, NF1
Interventions
Lovastatin, reading tutoring intervention, Placebo Oral Tablet, Other Academic "sham" tutoring
Drug · Behavioral
Lead sponsor
Vanderbilt University
Other
Eligibility
8 Years to 20 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 2, 2026, 6:56 AM EDT
Completed Phase 2 Interventional Results available

Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma

NCT00589784
View directory record Official record
Conditions
CNS Cancer, Meningioma, Intracranial Hemangiopericytoma, Hemangioblastoma, Neurofibromatosis
Interventions
Sunitinib
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
6
States / cities
Boston, Massachusetts • Basking Ridge, New Jersey • Commack, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2016 · Synced Oct 2, 2026, 6:56 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 2, 2026, 6:56 AM EDT
Completed No phase listed Observational

Multi-center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients

NCT00667836
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
3 Years to 18 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Feb 25, 2019 · Synced Oct 2, 2026, 6:56 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Neurofibromatosis Type 1 Patient Registry

NCT01410006
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Not listed
Enrollment
2,391 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2017
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 25, 2017 · Synced Oct 2, 2026, 6:56 AM EDT
Completed Phase 1 Interventional

Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas

NCT06132165
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Deoxycholic Acid, Polidocanol, 1064nm Nd:YAG laser, 755nm Alexandrite Laser
Drug · Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 80 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 21, 2025 · Synced Oct 2, 2026, 6:56 AM EDT
Completed No phase listed Observational

Using Positron Emission Tomography to Predict Intracranial Tumor Growth in Neurofibromatosis Type II Patients

NCT01222728
View directory record Official record
Conditions
Neoplasms, Nervous System Disease, Vestibular Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 50 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 2, 2026, 6:56 AM EDT
Completed No phase listed Observational

Relationship Stressors in Parents of Children With Cancer or Neurofibromatosis Type 1 (NF1)

NCT01702922
View directory record Official record
Conditions
Parents of Children With Cancer, Parents of Children With NF1, Parents of Children With Neurofibromatosis Type I
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
262 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
2
States / cities
Bethesda, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 31, 2024 · Synced Oct 2, 2026, 6:56 AM 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 19, 2025 · Synced Oct 2, 2026, 6:56 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 2, 2026, 6:56 AM EDT
Enrolling by invitation Phase 1 Interventional

Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)

NCT06300502
View directory record Official record
Conditions
Neurofibromas, Cutaneous, Neurofibromatosis 1
Interventions
Kybella
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 85 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Oct 2, 2026, 6:56 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 2, 2026, 6:56 AM EDT