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ClinicalTrials.gov 12 recruiting now official registry

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)
Phase 17Phase 24Phase 31

Active & Recent Trials

25 total, page 1 of 1

RECRUITING 20,000 participants

Neurofibromatosis (NF) Registry Portal

The Children's Tumor Foundation

NCT01885767

RECRUITING 1,000 participants

Neurofibromatosis Type 1 Tumor Early Detection Study

David Miller

NCT06515860

RECRUITING 1,000 participants

RASopathy Biorepository

Children's Hospital Medical Center, Cincinnati

NCT04395495

RECRUITING 476 participants

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

National Cancer Institute (NCI)

NCT02544022

RECRUITING NA 294 participants

A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1

Massachusetts General Hospital

NCT06262113

RECRUITING 225 participants

Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)

National Cancer Institute (NCI)

NCT06222203

RECRUITING Phase 2 200 participants

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

University of Alabama at Birmingham

NCT06188741

RECRUITING NA 70 participants

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

RECRUITING Phase 1 58 participants

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

Therapeutic Advances in Childhood Leukemia Consortium

NCT05849662

RECRUITING Phase 1 55 participants

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

National Cancer Institute (NCI)

NCT04750928

RECRUITING NA 50 participants

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

Massachusetts General Hospital

NCT07233408

RECRUITING Phase 2 30 participants

Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

Donald Basel

NCT02728388

ACTIVE NOT RECRUITING 500 participants

Natural History Study of Cutaneous Neurofibromas in People With NF1

Johns Hopkins University

NCT05581511

ACTIVE NOT RECRUITING Phase 1 160 participants

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

ACTIVE NOT RECRUITING Phase 3 145 participants

Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

AstraZeneca

NCT04924608

ACTIVE NOT RECRUITING Phase 1 99 participants

AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors

National Cancer Institute (NCI)

NCT01362803

ACTIVE NOT RECRUITING 80 participants

Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1

Johns Hopkins University

NCT05677594

ACTIVE NOT RECRUITING Phase 2 58 participants

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

Children's Hospital Medical Center, Cincinnati

NCT04481048

ACTIVE NOT RECRUITING Phase 1 23 participants

NF-1, Nutraceutical Intervention

Masonic Cancer Center, University of Minnesota

NCT05363267

ACTIVE NOT RECRUITING NA 10 participants

Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1

Children's National Research Institute

NCT03820778

COMPLETED Phase 2 199 participants

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

NFlection Therapeutics

NCT05005845

COMPLETED 55 participants

Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1

University of Arkansas

NCT05912400

COMPLETED 30 participants

PET/MRI in CNS and Extra-CNS Tumors of Patients With Neurofibromatosis-1 (NF1)

UNC Lineberger Comprehensive Cancer Center

NCT01800032

COMPLETED Phase 1 20 participants

Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas

Massachusetts General Hospital

NCT06120036

COMPLETED Phase 1 13 participants

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

Massachusetts General Hospital

NCT06132165

Phase Distribution

PhaseTrial count
Phase 1 7
Phase 2 4
Phase 3 1

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.

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.

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.

Verify with NIH →

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.