Condition

Neurofibromatosis 1 clinical trials and research

• 14Clinical trials
• 2NIH research projects
• 4Countries

Newest Neurofibromatosis 1 clinical trials

All recruiting trials (14)

• Online program providing personalized NF1 care letters plus videos or peer navigator support for adults with low health literacy

  This study will test whether giving adults with NF1 and low health literacy personalized care letters plus either short, plain-language videos or a call with a trained peer navigator helps them better understand NF1 symptoms and care before their primary care visit.

  Recruiting Massachusetts General Hospital · Boston, Massachusetts · NCT07233408

• Understanding genetic causes of neuroendocrine tumors in Mexican patients

  This study is trying to find out the genetic changes that cause neuroendocrine tumors in Mexican patients to help develop better treatments.

  Recruiting at 3 sites Universidad Nacional Autonoma de Mexico · Mexico City, Mexico City and 2 other locations · NCT06523582

• Combination therapy to shrink plexiform neurofibromas in NF1 patients

  This trial tests whether the drugs cabozantinib and selumetinib can be safely given together to see if they help shrink progressive or painful plexiform neurofibromas in adolescents and adults with NF1.

  Recruiting at 8 sites Phase 1 University of Alabama at Birmingham · Birmingham, Alabama and 7 other locations · NCT06502171

• Study on the use of Koselugo for treating Neurofibromatosis 1

  This study is testing how well Koselugo works and how safe it is for people with Neurofibromatosis 1 in everyday treatment settings in South Korea.

  Recruiting at 9 sites AstraZeneca · Busan and 11 other locations · NCT06360406

• Monitoring for cancer risk in neurofibromatosis type 1 tumors

  This study is trying to find out which harmless tumors in people with neurofibromatosis type 1 might turn into cancer so that doctors can better monitor patients at risk.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT06222203

• Early selumetinib to try to prevent plexiform neurofibromas in young children with NF1

  This trial will see if giving selumetinib to young children with NF1 who are at high risk can prevent or slow the development of plexiform neurofibromas.

  Recruiting at 15 sites Phase 2 University of Alabama at Birmingham · Birmingham, Alabama and 14 other locations · NCT06188741

• Combining trametinib and azacitidine for treating juvenile myelomonocytic leukemia

  This study is testing a new combination of medications to see if they can safely help children with juvenile myelomonocytic leukemia feel better, depending on their risk level.

  Recruiting at 21 sites Phase 1/2 Therapeutic Advances in Childhood Leukemia Consortium · Phoenix, Arizona and 20 other locations · NCT05849662

• T cell receptor depletion for blood cancer treatment

  This study is testing a new way to treat blood cancers by using a special treatment before a stem cell transplant to see if it helps both kids and adults do better after the procedure.

  Recruiting Phase 2 Masonic Cancer Center, University of Minnesota · Minneapolis, Minnesota · NCT05735717

• Therapy to help caregivers of children with genetic disorders manage stress

  This study tests whether a remote therapy program can help caregivers of children with genetic disorders manage their stress better.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT05361811

• Treatment of Neurofibromatosis Type 1 with HL-085

  This study is testing a new treatment called HL-085 to see if it can safely help adults with Neurofibromatosis Type 1 and inoperable tumors.

  Recruiting Phase 2 Shanghai Kechow Pharma, Inc. · Shanghai, Shanghai Municipality · NCT05331105

• Using Abemaciclib to treat atypical neurofibromas in patients with Neurofibromatosis Type 1

  This study is testing if the drug abemaciclib can safely help people with Neurofibromatosis Type 1 who have hard-to-remove atypical neurofibromas.

  Recruiting Phase 1/2 National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT04750928

• Collection of bio-specimens from patients with RASopathies

  This study is collecting samples from people with RASopathies to help researchers learn more about these rare genetic disorders and how they affect the body.

  Recruiting Children's Hospital Medical Center, Cincinnati · Cincinnati, Ohio · NCT04395495

• Improving questionnaires for pain and daily life in neurofibromatosis 1 patients

  This study is trying to improve pain questionnaires for people with neurofibromatosis 1 to better understand how their condition affects their daily lives.

  Recruiting at 2 sites National Institutes of Health Clinical Center (CC) · Washington D.C., District of Columbia and 5 other locations · NCT02544022

• Registry for patients with neurofibromatosis

  This study is creating a registry for people with neurofibromatosis to share their symptoms and experiences, helping connect them with clinical trials and improving understanding of the condition.

  Recruiting The Children's Tumor Foundation · New York, New York · NCT01885767

NIH-funded research (2)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Understanding variations in Neurofibromatosis type 1 to improve risk and care

  This project uses genetic, cellular, and computer models to learn why Neurofibromatosis type 1 causes different problems in different people and to guide better care for children and adults with NF1.

  NIH research Washington University · Saint Louis, United States · NIH-11327379

• Understanding the varied effects of neurofibromatosis type 1 to improve treatment and risk assessment

  This study is looking at neurofibromatosis type 1 (NF1) to understand how different genes and other factors affect the symptoms people experience, so we can create better, personalized treatments just for you.

  NIH research Washington University · Saint Louis, United States · NIH-11134859

Related topics

Quick answers

How many Neurofibromatosis 1 clinical trials are recruiting right now?

14, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 4 countries. The most sites are in United States (11 trials), China (1 trial), South Korea (1 trial), Mexico (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 3, Phase 1/2: 2, Phase 1: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.

Describe your situation →