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ClinicalTrials.gov 12 recruiting now early-phase tilt

Neurofibromatosis 1: early-phase trial registry

12 of 25 indexed Neurofibromatosis 1 trials are currently recruiting, with more weight in Phase 1-2.

Corpus placement: #652 of 28,707 conditions by registered trial count.

25 US clinical trials · 12 currently recruiting · #652 of 28,707 conditions by registered trial count

Neurofibromatosis 1: 25 US clinical trials tracked, 12 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.

Enrollment posture brief

Registry condition neurofibromatosis-1

OPEN 12 | RECR 48% | LATE 1 | EARLY 11 | RANK #652 | SPON 5

Nearest open-share peer: Diabetes Type 2 (48% recruiting · 25 trials)

Alpha-1 Antitrypsin Deficien48%Neurofibromatosis 1 (this)48%Alcohol Use40%Acute Lymphocytic Leukemia36%Alcohol-Related Disorders36%
Open-enrollment share among volume-matched conditions near Neurofibromatosis 1

Neurofibromatosis 1 lists 12 open slots (48% of 25 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →

Key findings

What ClinicalTrials.gov does not surface for Neurofibromatosis 1 on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.

  1. Neurofibromatosis 1 is recruiting above its size-band peers.

    12 of 25 indexed trials (48%) are open to enrollment, against a 39.7% average across 762 conditions with 20-99 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.

  2. One sponsor concentrates Neurofibromatosis 1 research.

    National Cancer Institute (NCI) accounts for 5 of 25 trials (20%). 16 distinct sponsors appear on at least one study for this condition label.

  3. Typical Neurofibromatosis 1 enrolment sits around 80 participants.

    Median target enrolment is 80 among the 25 trials that report a genuine target (sentinel values excluded). 14 of those (56%) plan for fewer than 100 participants.

  4. Neurofibromatosis 1 splits between interventional and observational designs.

    16 interventional and 9 observational studies make up this index (64% interventional). Study type is sponsor-reported on ClinicalTrials.gov.

Phase 17Phase 24Phase 31

Open and 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

Named interventions

Most-linked intervention names on Neurofibromatosis 1 records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.

Selumetinib Drug
2 trials
Placebo Other
2 trials
FACIT-F and Pain Scales Other
1 trials
NFX-179 gel Drug
1 trials
1064nm Nd:YAG laser Device
1 trials
Polidocanol Drug
1 trials
Deoxycholic Acid Drug
1 trials
Asclera Drug
1 trials

US sites by state

Facility locations filed on Neurofibromatosis 1 trials. A trial with sites in several states counts in each.

Maryland 10 trials
Ohio 7 trials
Massachusetts 7 trials
Pennsylvania 4 trials
California 4 trials
New York 3 trials
Minnesota 3 trials

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

What to do with this Neurofibromatosis 1 page

25 registered trials, 12 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.

Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Neurofibromatosis 1 condition card totals 25 registered trials; most-linked intervention Selumetinib; top US site state Maryland. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.