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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Neurofibromatosis I Clear all
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 17, 2017 · Synced Jul 23, 2026, 3:48 AM EDT
Completed Phase 2 Interventional Results available

NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

NCT04435665
View directory record Official record
Conditions
Neurofibromatosis 1, Cutaneous Neurofibroma
Interventions
NFX-179 Gel, Vehicle Gel
Drug
Lead sponsor
NFlection Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
6
States / cities
Fremont, California • New Brighton, Minnesota • Rochester, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 8, 2022 · Synced Jul 23, 2026, 3:48 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial

NCT04879160
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
7 Years and older
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 21, 2024 · Synced Jul 23, 2026, 3:48 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 24, 2022 · Synced Jul 23, 2026, 3:48 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 22, 2018 · Synced Jul 23, 2026, 3:48 AM EDT
Terminated Not applicable Interventional Results available

A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1)

NCT02718131
View directory record Official record
Conditions
NF1, Congenital Pseudarthrosis of Tibia
Interventions
INFUSE Bone Graft (BMP-2), Control Group
Device · Procedure
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
2 Years to 18 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
15
States / cities
Birmingham, Alabama • Los Angeles, California • Washington D.C., District of Columbia + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2021 · Synced Jul 23, 2026, 3:48 AM EDT
Completed Phase 1 Interventional

Vinblastine and Carboplatin in Treating Young Patients With Newly Diagnosed or Recurrent Low-Grade Glioma

NCT00352495
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Neurofibromatosis Type 1
Interventions
carboplatin, vinblastine sulfate
Drug
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 21 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
19
States / cities
Birmingham, Alabama • Orange, California • Washington D.C., District of Columbia + 16 more
Source: ClinicalTrials.gov public record
Updated Feb 12, 2014 · Synced Jul 23, 2026, 3:48 AM EDT
Completed Phase 2 Interventional Results available

A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas

NCT00634270
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Sirolimus
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
3 Years to 75 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
9
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Nov 16, 2017 · Synced Jul 23, 2026, 3:48 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 3, 2014 · Synced Jul 23, 2026, 3:48 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 4, 2026 · Synced Jul 23, 2026, 3:48 AM EDT
Completed No phase listed Observational

Neurofibromatosis Type 1 Brain Tumor Genetic Risk

NCT01707836
View directory record Official record
Conditions
Neurofibromatosis Type 1, Pediatric Brain Tumor
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 18 Years
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 8, 2017 · Synced Jul 23, 2026, 3:48 AM EDT
Active, not recruiting Phase 1 Interventional

NF-1, Nutraceutical Intervention

NCT05363267
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
curcumin, high phenolic extra virgin olive oil (HP-EVOO)
Dietary Supplement
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Jul 23, 2026, 3:48 AM 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 5, 2023 · Synced Jul 23, 2026, 3:48 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 Mar 4, 2026 · Synced Jul 23, 2026, 3:48 AM EDT
Completed No phase listed Observational

Internet Support Group for Parents of a Child With Neurofibromatosis Type 1

NCT02153931
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 30, 2019 · Synced Jul 23, 2026, 3:48 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 Jul 20, 2026 · Synced Jul 23, 2026, 3:48 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 Jul 16, 2026 · Synced Jul 23, 2026, 3:48 AM EDT
Terminated Phase 2 Interventional

Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

NCT01345136
View directory record Official record
Conditions
Neurofibromatosis Type 2, Neuroma, Acoustic
Interventions
RAD001, everolimus
Drug
Lead sponsor
Jonsson Comprehensive Cancer Center
Other
Eligibility
16 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Feb 12, 2024 · Synced Jul 23, 2026, 3:48 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 25, 2021 · Synced Jul 23, 2026, 3:48 AM EDT
No Longer Available No phase listed Expanded access

Compassionate Use Arm - ABI541 ABI for 10 NF2 Patients

NCT02589912
View directory record Official record
Conditions
Neurofibromatosis Type 2, Severe Profound Sensorineural Hearing Loss
Interventions
Nucleus Profile ABI541 Auditory Brainstem Implant
Device
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
12 Years to 80 Years
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 5, 2019 · Synced Jul 23, 2026, 3:48 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1

NCT05912400
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Blood draw, FACIT-F and Pain Scales
Diagnostic Test · Other
Lead sponsor
University of Arkansas
Other
Eligibility
18 Years and older
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated May 7, 2025 · Synced Jul 23, 2026, 3:48 AM EDT
Active, not recruiting No phase listed Observational

Natural History Study of Cutaneous Neurofibromas in People With NF1

NCT05581511
View directory record Official record
Conditions
Neurofibromatosis Type 1, Neurofibromatosis 1, Neurofibromatosis (Nonmalignant), Cutaneous Neurofibroma
Interventions
Evaluation of the natural history of cutaneous neurofibromas
Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
1 Year to 100 Years
Enrollment
500 participants
Timeline
2021 – 2029
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2025 · Synced Jul 23, 2026, 3:48 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 16, 2019 · Synced Jul 23, 2026, 3:48 AM EDT
Terminated Phase 2 Interventional Results available

A Phase II Trial of Poly-ICLC for Low-Grade Gliomas

NCT04544007
View directory record Official record
Conditions
NF1, Low-grade Glioma
Interventions
Poly ICLC
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Up to 22 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
12
States / cities
Birmingham, Alabama • Los Angeles, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Jul 23, 2026, 3:48 AM EDT