Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Neurofibromatosis Type 1 clinical trials
5 of 18 indexed Neurofibromatosis Type 1 trials are currently recruiting.
18 US clinical trials · 5 currently recruiting
Neurofibromatosis Type 1 is the subject of 18 registered US clinical trials on ClinicalTrials.gov, 5 of them currently open to new participants. 2 are in Phase 3-4 (later-stage) and 8 in Phase 1-2 (earlier-stage). The most active sponsor is National Cancer Institute (NCI), running 7 of these trials.
The research picture
Neurofibromatosis Type 1 has 18 registered US clinical trials, 5 of them open to new participants right now, about 28% of the total.
- 5
- recruiting participants now
- 28%
- of trials open to enrollment
- 2
- in Phase 3–4 (later-stage)
- 7
- top sponsor: National Cancer Institute (NCI)
Active & Recent Trials
18 total, page 1 of 1
Neurofibromatosis Type 1 Tumor Early Detection Study
David Miller
NCT06515860
Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study
National Cancer Institute (NCI)
NCT06880991
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)
National Cancer Institute (NCI)
NCT06507748
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
Healx Limited
NCT06541847
Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence
Nicole Baca
NCT04763109
Natural History Study of Cutaneous Neurofibromas in People With NF1
Johns Hopkins University
NCT05581511
Natural History Study of Patients With Neurofibromatosis Type I
National Cancer Institute (NCI)
NCT00924196
A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma
National Cancer Institute (NCI)
NCT03871257
Interventions for Reading Disabilities in NF1
Vanderbilt University
NCT02964884
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
National Cancer Institute (NCI)
NCT01362803
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
AstraZeneca
NCT05309668
Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas
AstraZeneca
NCT05101148
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
National Cancer Institute (NCI)
NCT03190915
Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1
Kristina Hardy
NCT02944032
Targeting the Mechanisms Underlying Cutaneous Neurofibroma Formation in NF1: A Clinical Translational Approach.
Corewell Health West
NCT02777775
Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain
National Cancer Institute (NCI)
NCT02471339
Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors
Sarcoma Alliance for Research through Collaboration
NCT00304083
Vaccine Therapy in Treating Patients with Malignant Peripheral Nerve Sheath Tumor That is Recurrent or Cannot Be Removed by Surgery
Mayo Clinic
NCT02700230
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 4 |
| Phase 2 | 4 |
| Phase 3 | 2 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Neurofibromatosis Type 1 Trial Landscape
18 indexed Neurofibromatosis Type 1 studies · 5 recruiting (28%).
Phase mix: 2 late (III/IV) vs 8 early (I/II).
Top sponsor: National Cancer Institute (NCI) (7 trials), 9 others listed. 18 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 Type 1?
PlainTrial tracks 18 US clinical trials for Neurofibromatosis Type 1, of which 5 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Neurofibromatosis Type 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.