Neurofibromatosis Clinical Trials

34 recruitingLast updated: June 18, 2026

There are 34 actively recruiting neurofibromatosis clinical trials across 23 countries. Studies span Phase 2, Not Applicable, Phase 1, Phase 3, Phase 4. Top locations include Bethesda, Maryland, United States, Baltimore, Maryland, United States, Washington D.C., District of Columbia, United States. Updated daily from ClinicalTrials.gov.


Neurofibromatosis Trials at a Glance

34 actively recruiting trials for neurofibromatosis are listed on ClinicalTrialsFinder across 6 cities in 23 countries. The largest study group is Phase 2 with 8 trials, with the heaviest enrollment activity in Bethesda, Baltimore, and Washington D.C.. Lead sponsors running neurofibromatosis studies include National Cancer Institute (NCI), AstraZeneca, and Massachusetts General Hospital.

Browse neurofibromatosis trials by phase

Treatments under study

About Neurofibromatosis Clinical Trials

Looking for clinical trials for Neurofibromatosis? There are currently 4 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Neurofibromatosis trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Neurofibromatosis clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 34 trials

Recruiting

Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy)

Neurofibromatosis Type 1
AstraZeneca200 enrolled9 locationsNCT07221331
Recruiting

Familial Investigations of Childhood Cancer Predisposition

Pancreatic CancerHereditary Breast and Ovarian CancerHodgkin Lymphoma+43 more
St. Jude Children's Research Hospital1,500 enrolled1 locationNCT03050268
Recruiting

Natural History Study of Patients With Neurofibromatosis Type 2

Neurofibromatosis
National Institute of Neurological Disorders and Stroke (NINDS)269 enrolled1 locationNCT00598351
Recruiting
Phase 1Phase 2

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

Neurofibromatosis 1
National Cancer Institute (NCI)55 enrolled1 locationNCT04750928
Recruiting

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

Neurofibromatosis 1Plexiform Neurofibromas
National Cancer Institute (NCI)476 enrolled6 locationsNCT02544022
Recruiting
Not Applicable

A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)

Neurofibromatosis Type 1
National Cancer Institute (NCI)70 enrolled2 locationsNCT06507748
Recruiting
Phase 4

Pediatric Long-Term Follow-up and Rollover Study

Neurofibromatosis Type 1GlioblastomaAnaplastic Astrocytoma+20 more
Novartis Pharmaceuticals165 enrolled55 locationsNCT03975829
Recruiting

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

Neurofibromatosis 1Nerve Sheath Neoplasms
National Cancer Institute (NCI)225 enrolled1 locationNCT06222203
Recruiting

Real-World Treatment Study of Koselugo (Selumetinib)

Neurofibromatosis 1Neurofibroma, Plexiform
AstraZeneca200 enrolled12 locationsNCT06360406
Recruiting
Phase 2

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

Neurofibromatosis 1Plexiform Neurofibroma
University of Alabama at Birmingham200 enrolled15 locationsNCT06188741
Recruiting

Clinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsia

Neurofibromatosis
AstraZeneca200 enrolled13 locationsNCT07088991
Recruiting
Phase 2

A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1

Neurofibromatosis Type 1
Healx Limited25 enrolled14 locationsNCT06541847
Recruiting
Phase 1

Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1

NeurofibromatosisNF1Cutaneous Neurofibroma+3 more
Johns Hopkins University10 enrolled1 locationNCT07102394
Recruiting
Phase 2

Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)

Neurofibromatosis type 2MeningiomaEpendymoma+2 more
Scott R. Plotkin, MD, PhD109 enrolled6 locationsNCT04374305
Recruiting
Not Applicable

Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence

Neurofibromatosis Type 1
Nicole Baca15 enrolled1 locationNCT04763109
Recruiting

Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study

Neurofibromatosis Type 1Neurofibroma
National Cancer Institute (NCI)110 enrolled1 locationNCT06880991
Recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Cystic FibrosisHuntington DiseaseSickle Cell Disease+12 more
Assistance Publique - Hôpitaux de Paris550 enrolled1 locationNCT06147414
Recruiting
Phase 1Phase 2

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

Neurofibromatosis 1Leukemia, Juvenile MyelomonocyticJMML+2 more
Therapeutic Advances in Childhood Leukemia Consortium58 enrolled19 locationsNCT05849662
Recruiting
Phase 2

MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies

Acute Lymphoblastic LeukemiaAcute Myeloid LeukemiaAcute Leukemia+21 more
Masonic Cancer Center, University of Minnesota70 enrolled1 locationNCT05735717
Recruiting
Not Applicable

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

Neurofibromatosis 1
Massachusetts General Hospital50 enrolled1 locationNCT07233408