Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.

2026 data Public-data reference. official source

Neurofibromatosis 1

Open-data reference.

23 US clinical trials · 11 currently recruiting

Active & Recent Trials

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

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 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
Early Phase 1 7
Phase 2 3
Phase 3 1

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Neurofibromatosis 1 Trial Landscape

ClinicalTrials.gov lists 23 US studies indexed under Neurofibromatosis 1, and 11 of those are currently open to recruitment — roughly 48% of the total volume on the registry. 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.

The phase distribution for Neurofibromatosis 1 shows 1 late-stage studies (Phase 3 and Phase 4 combined) alongside 10 earlier-phase entries (Phase 1 through Phase 2). 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.

Top sponsor activity for Neurofibromatosis 1 is led by National Cancer Institute (NCI) with 5 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 23 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. 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 23 US clinical trials for Neurofibromatosis 1, of which 11 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

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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 · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

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