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ClinicalTrials.gov public records Last synced Oct 8, 2026, 4:18 AM EDT

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Showing 73–96 of 122 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: neurofibromatosis1 (NF1) Clear all
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 8, 2026, 4:18 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 8, 2026, 4:18 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 8, 2026, 4:18 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 8, 2026, 4:18 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 8, 2026, 4:18 AM 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 8, 2026, 4:18 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 8, 2026, 4:18 AM EDT
Terminated Phase 2 Interventional Results available

AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine

NCT00326872
View directory record Official record
Conditions
Neurofibromatosis Type 1, Plexiform Neurofibroma, Spinal Cord Neurofibroma
Interventions
Cediranib Maleate
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2016
U.S. locations
9
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2017 · Synced Oct 8, 2026, 4:18 AM EDT
Recruiting No phase listed Observational

Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study

NCT06880991
View directory record Official record
Conditions
Neurofibromatosis Type 1, Neurofibroma
Interventions
Arm 1
Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
5 Years to 120 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 14, 2026 · Synced Oct 8, 2026, 4:18 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 8, 2026, 4:18 AM EDT
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 8, 2026, 4:18 AM EDT
Completed Phase 2 Interventional Results available

Bevacizumab in Treating Patients With Recurrent or Progressive Meningiomas

NCT01125046
View directory record Official record
Conditions
Acoustic Schwannoma, Adult Anaplastic Meningioma, Adult Ependymoma, Adult Grade I Meningioma, Adult Grade II Meningioma, Adult Meningeal Hemangiopericytoma, Adult Papillary Meningioma, Neurofibromatosis Type 1, Neurofibromatosis Type 2, Recurrent Adult Brain Tumor
Interventions
bevacizumab
Biological
Lead sponsor
Northwestern University
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2018
U.S. locations
5
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2021 · Synced Oct 8, 2026, 4:18 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 8, 2026, 4:18 AM 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 8, 2026, 4:18 AM 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 8, 2026, 4:18 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 8, 2026, 4:18 AM 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 8, 2026, 4:18 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 8, 2026, 4:18 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 8, 2026, 4:18 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 8, 2026, 4:18 AM EDT
Recruiting Not applicable Interventional

Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy

NCT07233408
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
NF1 Educational Videos, Peer Navigator, Letters about NF1 Care
Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Oct 8, 2026, 4:18 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)

NCT02544022
View directory record Official record
Conditions
Neurofibromatosis 1, Plexiform Neurofibromas
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
5 Years and older
Enrollment
476 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2027
U.S. locations
6
States / cities
Washington D.C., District of Columbia • Chicago, Illinois • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 8, 2026, 4:18 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 8, 2026, 4:18 AM EDT
Completed Phase 1 Interventional

Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors

NCT02415153
View directory record Official record
Conditions
Neurofibromatosis Type 1, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Visual Pathway Glioma, Recurrent Primary Central Nervous System Neoplasm, Refractory Primary Central Nervous System Neoplasm
Interventions
Laboratory Biomarker Analysis, Pharmacological Study, Pomalidomide
Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 20 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
12
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Oct 8, 2026, 4:18 AM EDT