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Condition (MeSH)

Neurofibromatosis 1

ClinicalTrials.gov lists 172 trials for Neurofibromatosis 1 or a narrower condition beneath it. National Cancer Institute (NCI) has the most among industry and government lead sponsors, with 30.

MeSH D009456 Neoplasms Neoplasm C04 C10 C16 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 22 Phase 1/2 Phase 1/2 9 Phase 2 Phase 2 44 Phase 2/3 Phase 2/3 2 Phase 3 Phase 3 4 Phase 4 Phase 4 2 Not applicable Not applicable 32 Observational Observational 57 Phase 1 Phase 1 22 Phase 1/2 Phase 1/2 9 Phase 2 Phase 2 44 Phase 2/3 Phase 2/3 2 Phase 3 Phase 3 4 Phase 4 Phase 4 2 Not applicable Not applicable 32 Observational Observational 57

Trials per phase (log scale)

172 of the 172 trials are tagged with Neurofibromatosis 1 itself; the rest are tagged with a narrower condition beneath it.

Get the Neurofibromatosis 1 report as a PDF

The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Neurofibromatosis 1 trials start? (26)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 3 trials are registered to start in 2027 or later, and not listed here.

Trials over time, by phase

  • Phase 1
  • Phase 1/2
  • Phase 2
  • Phase 2/3
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational

Top of scale: 17

2000: 1 trials (Observational 1) 2001: 3 trials (Observational 1, Phase 2 2) 2002: 2 trials (Not applicable 1, Phase 1 1) 2004: 3 trials (Observational 1, Phase 4 1, Phase 2 1) 2005: 2 trials (Observational 1, Phase 2 1) 2006: 7 trials (Observational 3, Not applicable 1, Phase 2/3 1, Phase 2 1, Phase 1 1) 2007: 3 trials (Observational 2, Phase 2 1) 2008: 6 trials (Observational 2, Phase 2 2, Phase 1 2) 2009: 5 trials (Not applicable 2, Phase 2 1, Phase 1 2) 2010: 6 trials (Observational 3, Not applicable 1, Phase 2 1, Phase 1/2 1) 2011: 3 trials (Observational 1, Phase 2 1, Phase 1/2 1) 2012: 7 trials (Observational 5, Phase 2 2) 2013: 2 trials (Not applicable 2) 2014: 5 trials (Observational 2, Not applicable 1, Phase 2/3 1, Phase 2 1) 2015: 9 trials (Observational 5, Not applicable 1, Phase 3 1, Phase 2 1, Phase 1 1) 2016: 7 trials (Observational 3, Not applicable 1, Phase 2 3) 2017: 10 trials (Observational 2, Not applicable 2, Phase 2 5, Phase 1 1) 2018: 8 trials (Observational 1, Not applicable 4, Phase 2 2, Phase 1 1) 2019: 16 trials (Observational 5, Not applicable 4, Phase 4 1, Phase 2 5, Phase 1/2 1) 2020: 6 trials (Observational 1, Not applicable 1, Phase 3 1, Phase 2 1, Phase 1 2) 2021: 17 trials (Observational 6, Not applicable 2, Phase 3 1, Phase 2 3, Phase 1/2 2, Phase 1 3) 2022: 10 trials (Observational 3, Not applicable 3, Phase 1/2 1, Phase 1 3) 2023: 4 trials (Observational 1, Phase 3 1, Phase 2 2) 2024: 9 trials (Observational 3, Not applicable 2, Phase 2 2, Phase 1/2 1, Phase 1 1) 2025: 9 trials (Observational 2, Not applicable 1, Phase 2 3, Phase 1/2 1, Phase 1 2)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 9
2025 9
2024 9
2023 4
2022 10
2021 17
2020 6
2019 16
2018 8
2017 10

Showing 10 of 26 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 12 trials starting in 2026 and about 11 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 19 trials still running at the 2026-10-09 snapshot; registered median of 43 months across 98 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Neurofibromatosis 1 trials in? (8)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 22
Phase 1/2 9
Phase 2 44
Phase 2/3 2
Phase 3 4
Phase 4 2
Not applicable 32
Observational 57

How many Neurofibromatosis 1 trials are recruiting? (9)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 10
Recruiting 25
Enrolling by invitation 3
Active, not recruiting 21
Completed 80

Showing 5 of 9 statuses.

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Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Neurofibromatosis 1 trials? (3)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
National Cancer Institute (NCI) 30
AstraZeneca 10
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. 3

Which drugs are being tested for Neurofibromatosis 1? (6)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
Selumetinib 19
GSK1120212 5
Lovastatin 5
KYBELLA 3
EVEROLIMUS 3

Showing 5 of 6 drugs.

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Interventions as named in the registry (9)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Vinblastin 2
or carboplatin 2
Lamotrigine use 2
Pirfenidoneone 2
NFX-179 Gel 2

Showing 5 of 9 interventions.

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These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Neurofibromatosis 1 trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Headache 10 5
Seizure 10 4
Fatigue 9 1
Neutrophil count decreased 9 2
Neutropenia 8 2

Showing 5 of 10 adverse events.

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Which publications cover Neurofibromatosis 1? (180)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Lamotrigine for cognitive deficits associated with neurofibromatosis type 1: A phase II randomized placebo-controlled trial. Developmental medicine and child neurology 2025 Yes
Parent-child discrepancies in reports of child psychosocial functioning in neurofibromatosis type 1. Journal of family psychology : JFP : journal of the Division of Family Psychology of the American Psychological Association (Division 43) 2025 Yes
ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma. Journal of clinical oncology : official journal of the American Society of Clinical Oncology 2025 Yes
Precocious Puberty in Children with Neurofibromatosis Type 1. The Journal of pediatrics 2025 Yes
Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial. Nature medicine 2025 Yes

Showing 5 of 180 publications.

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Prevalence (30)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Neurofibromatosis type 1 Prevalence at birth 1-5 / 10 000 United Kingdom GB Unspecified
Neurofibromatosis type 1 Point prevalence 1-5 / 10 000 United Kingdom GB Exact
Neurofibromatosis type 1 Prevalence at birth 1-5 / 10 000 United Kingdom GB Exact
Neurofibromatosis type 1 Prevalence at birth 1-5 / 10 000 United Kingdom GB Exact
Neurofibromatosis type 1 Point prevalence 1-5 / 10 000 United Kingdom GB Exact

Showing 5 of 30 prevalence observations.

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Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Neurofibromatosis type 1 636 Exact Orphadata Science knowledge files, Product 1
Neurofibromatosis type 1 636 Exact Orphanet Rare Disease Ontology (ORDO)

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: sub-conditions in mesh.

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