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2026 data Public-data reference. official source

Neurofibromatosis Type 1

Open-data reference.

18 US clinical trials · 5 currently recruiting

Active & Recent Trials

RECRUITING 1,000 participants

Neurofibromatosis Type 1 Tumor Early Detection Study

David Miller

NCT06515860

RECRUITING 110 participants

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

National Cancer Institute (NCI)

NCT06880991

RECRUITING NA 70 participants

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

RECRUITING Phase 2 25 participants

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

Healx Limited

NCT06541847

RECRUITING NA 15 participants

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

ACTIVE NOT RECRUITING 500 participants

Natural History Study of Cutaneous Neurofibromas in People With NF1

Johns Hopkins University

NCT05581511

ACTIVE NOT RECRUITING 259 participants

Natural History Study of Patients With Neurofibromatosis Type I

National Cancer Institute (NCI)

NCT00924196

ACTIVE NOT RECRUITING Phase 3 165 participants

A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma

National Cancer Institute (NCI)

NCT03871257

ACTIVE NOT RECRUITING Phase 2 120 participants

Interventions for Reading Disabilities in NF1

Vanderbilt University

NCT02964884

ACTIVE NOT RECRUITING Phase 1 99 participants

AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors

National Cancer Institute (NCI)

NCT01362803

ACTIVE NOT RECRUITING Phase 1 36 participants

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

AstraZeneca

NCT05309668

ACTIVE NOT RECRUITING Phase 1 24 participants

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

ACTIVE NOT RECRUITING Phase 2 10 participants

Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

National Cancer Institute (NCI)

NCT03190915

COMPLETED NA 103 participants

Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1

Kristina Hardy

NCT02944032

COMPLETED 71 participants

Targeting the Mechanisms Underlying Cutaneous Neurofibroma Formation in NF1: A Clinical Translational Approach.

Corewell Health West

NCT02777775

COMPLETED Phase 3 66 participants

Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain

National Cancer Institute (NCI)

NCT02471339

COMPLETED Phase 2 48 participants

Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors

Sarcoma Alliance for Research through Collaboration

NCT00304083

COMPLETED Phase 1 9 participants

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

PhaseTrial count
Early Phase 1 4
Phase 2 4
Phase 3 2

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

Reading the Neurofibromatosis Type 1 Trial Landscape

ClinicalTrials.gov lists 18 US studies indexed under Neurofibromatosis Type 1, and 5 of those are currently open to recruitment — roughly 28% 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 Type 1 shows 2 late-stage studies (Phase 3 and Phase 4 combined) alongside 8 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 Type 1 is led by National Cancer Institute (NCI) with 7 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 18 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 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

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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