Neurofibromatosis type 1: who works on it

One card per institution. Pick a target or a country to narrow the list.

• All targets
• NF1 15
• MEK 2
• BRAF 0
• MAPK 0
• ALK 0
• ERK 0
• FAK 0
• MDM2 1
• PD-L1 1
• ROS1 0

Funded research projects that name this disease in their title, terms or summary and have not ended yet: who is funded, where, how much and until when. Organisations only; the funder's page has the details.

No running funded project names this disease yet.

Sources: NIH RePORTER (U.S. National Institutes of Health, public domain) and CORDIS (European Commission, CC BY 4.0). Changes: filtered by disease, organisations matched to ROR.

Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

• Terminated

  Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

  NCT02728388· Donald Basel· Updated 10 Jul 2026

  “data analysis unequivocal - requires study methodology re-evaluation”Registry text, quoted unchanged. Source

• Terminated

  Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia

  NCT05891847· AstraZeneca· Updated 8 Jul 2026

  “Statistically significant failure to achieve key secondary endpoints. The current data do not demonstrate sufficient potential to achieve the planned scientific objectives. The need to optimize the allocation of company resources.”Registry text, quoted unchanged. Source

• Suspended

  Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1-associated Cutaneous Neurofibromas (cNFs)

  NCT06934928· Massachusetts General Hospital· Updated 4 Jun 2026

  “Study is currently not funded at this time.”Registry text, quoted unchanged. Source

• Terminated

  PF-07284892 in Participants With Advanced Solid Tumors

  NCT04800822· Pfizer· Updated 4 Jun 2026

  “The study was prematurely discontinued due to strategic reasons. The PF-07284892 program will continue”Registry text, quoted unchanged. Source

• Terminated

  US Selumetinib Registry

  NCT05683678· Alexion Pharmaceuticals, Inc.· Updated 4 Apr 2025

  “Sponsor decision.”Registry text, quoted unchanged. Source

• Terminated

  A Phase II Trial of Poly-ICLC for Low-Grade Gliomas

  NCT04544007· University of Alabama at Birmingham· Updated 21 Mar 2025

  “The closure of the study is due to the lack of enrollment and the lack of evaluable patients.”Registry text, quoted unchanged. Source

• Withdrawn

  Stem Cells in NF1 Patients With Tumors of the Central Nervous System

  NCT03332030· Roger Packer· Updated 7 Feb 2024

  “Suspended due to cessation of funding”Registry text, quoted unchanged. Source

• Withdrawn

  Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled Study

  NCT05825365· AstraZeneca· Updated 14 Aug 2023

  “Selumetinib launch approval achieved in advance,strategy changesa new study will be generated in place of this study.”Registry text, quoted unchanged. Source

Trials whose registry record says individual participant data (IPD) will be shared.

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

  NCT03871257· National Cancer Institute (NCI)· Source

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

  NCT04750928· National Cancer Institute (NCI)· Source

• Natural History Study of Patients With Neurofibromatosis Type I

  NCT00924196· National Cancer Institute (NCI)· Source

• A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma

  NCT04166409· National Cancer Institute (NCI)· Source

• Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

  NCT04924608· AstraZeneca· Source

• A Study of Avutometinib for People With Solid Tumor Cancers

  NCT06104488· Memorial Sloan Kettering Cancer Center· Source

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

  NCT06222203· National Cancer Institute (NCI)· Source

• Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...

  NCT02390752· National Cancer Institute (NCI)· Source

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

  NCT02544022· National Cancer Institute (NCI)· Source

• Combination of Avutometinib and Defactinib for Treatment of Relapsed, Refractory, Metastatic, or Unresectable Malignant Peripheral Nerve Sheath Tumor

  NCT07743554· Girish Dhall, MD· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

See which targets of this disease your abstract mentions, and go to the people working on them.