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Neurofibromatosis 1 clinical trials
12 of 25 indexed Neurofibromatosis 1 trials are currently recruiting.
25 US clinical trials · 12 currently recruiting
Neurofibromatosis 1 is the subject of 25 registered US clinical trials on ClinicalTrials.gov, 12 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 11 in Phase 1-2 (earlier-stage). The most active sponsor is National Cancer Institute (NCI), running 5 of these trials.
The research picture
Neurofibromatosis 1 has 25 registered US clinical trials, 12 of them open to new participants right now, about 48% of the total.
- 12
- recruiting participants now
- 48%
- of trials open to enrollment
- 1
- in Phase 3–4 (later-stage)
- 5
- top sponsor: National Cancer Institute (NCI)
Active & Recent Trials
25 total, page 1 of 1
Neurofibromatosis (NF) Registry Portal
The Children's Tumor Foundation
NCT01885767
Neurofibromatosis Type 1 Tumor Early Detection Study
David Miller
NCT06515860
RASopathy Biorepository
Children's Hospital Medical Center, Cincinnati
NCT04395495
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
National Cancer Institute (NCI)
NCT02544022
A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1
Massachusetts General Hospital
NCT06262113
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
National Cancer Institute (NCI)
NCT06222203
Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
University of Alabama at Birmingham
NCT06188741
Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial
National Cancer Institute (NCI)
NCT05361811
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
Therapeutic Advances in Childhood Leukemia Consortium
NCT05849662
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
National Cancer Institute (NCI)
NCT04750928
Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy
Massachusetts General Hospital
NCT07233408
Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II
Donald Basel
NCT02728388
Natural History Study of Cutaneous Neurofibromas in People With NF1
Johns Hopkins University
NCT05581511
Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
Shanghai Fosun Pharmaceutical Industrial Development Co.
NCT04954001
Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
AstraZeneca
NCT04924608
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
National Cancer Institute (NCI)
NCT01362803
Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1
Johns Hopkins University
NCT05677594
Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1
Children's Hospital Medical Center, Cincinnati
NCT04481048
NF-1, Nutraceutical Intervention
Masonic Cancer Center, University of Minnesota
NCT05363267
Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1
Children's National Research Institute
NCT03820778
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
NFlection Therapeutics
NCT05005845
Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1
University of Arkansas
NCT05912400
PET/MRI in CNS and Extra-CNS Tumors of Patients With Neurofibromatosis-1 (NF1)
UNC Lineberger Comprehensive Cancer Center
NCT01800032
Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas
Massachusetts General Hospital
NCT06120036
Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas
Massachusetts General Hospital
NCT06132165
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 7 |
| Phase 2 | 4 |
| Phase 3 | 1 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Neurofibromatosis 1 Trial Landscape
25 indexed Neurofibromatosis 1 studies · 12 recruiting (48%).
Phase mix: 1 late (III/IV) vs 11 early (I/II).
Top sponsor: National Cancer Institute (NCI) (5 trials), 9 others listed. 25 shown below, recruiting first.
How to read this condition's trial landscape
That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Neurofibromatosis 1?
PlainTrial tracks 25 US clinical trials for Neurofibromatosis 1, of which 12 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Neurofibromatosis 1?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology - how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.