Clinical trials
NF1 clinical trials
Below are recruiting nf1 clinical trials, each written for real people, not researchers. We’re tracking 19 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06961565RecruitingPhase 1
Study of PAS-004 for adults with NF1 and plexiform neurofibromas
This trial tests an oral medication called PAS-004 for adults with Neurofibromatosis Type 1 who have at least one symptomatic plexiform neurofibroma that cannot be surgically removed. The goal is to see if the drug can shrink these tumors or improve symptoms, and to understand side effects.
Birmingham, AlabamaAges 18 years+ - NCT06159166RecruitingPhase 1/Phase 2
Mirdametinib for NF1 skin bumps
This study tests an oral drug called mirdametinib to see if it can shrink skin bumps caused by NF1. If you have at least 24 of these bumps on your skin and are in decent health, you may be able to join.
Baltimore, MarylandAges 18 years+ - NCT06735820RecruitingPhase 1/Phase 2
MEK and MDM2 inhibitors for NF1-related tumors
This trial tests two targeted drugs (selumetinib and milademetan) for people with neurofibromatosis type 1 (NF1) who have either a malignant peripheral nerve sheath tumor (MPNST) or an atypical neurofibromatous neoplasm with uncertain biologic potential (ANNUBP). The goal is to see if blocking certain proteins can shrink or control these tumors.
Washington D.C., District of ColumbiaAges 18 years+ - NCT07233408Recruiting
Online tool to help adults with neurofibromatosis 1 and low health literacy
This study tests an online platform designed to help adults with neurofibromatosis 1 (NF1) who have low health literacy. The goal is to make it easier to get evidence-based care. You may be a good fit if you have an upcoming checkup with your primary care doctor.
Boston, MassachusettsAges 18 years+ - NCT07102394RecruitingPhase 1
Testing talimogene laherparepvec for skin bumps in adults with NF1
This trial tests a drug called IMLYGIC (talimogene laherparepvec) to see if it is safe and tolerable for treating skin bumps (cutaneous neurofibromas) in adults with NF1. The drug is injected directly into the bumps, and the study will check if it can shrink them or improve symptoms.
Baltimore, MarylandAges 18 years+ - NCT05253131RecruitingPhase 2
Selumetinib plus durvalumab for advanced sarcoma
This Phase 2 study tests whether selumetinib and durvalumab can slow or shrink certain advanced sarcomas. It’s for adults with disease that has come back, progressed, or can’t be safely removed with surgery.
Birmingham, AlabamaAges 18–99 - NCT06693284RecruitingEarly Phase 1
Testing mirdametinib and vorinostat for NF1-related MPNST
This trial tests two targeted drugs, mirdametinib and vorinostat, to see if they can shrink MPNST tumors in people with NF1 before surgery. It is for patients whose tumors lack a specific protein marker (H3K27me3).
Minneapolis, MinnesotaAges 12 years+ - NCT02544022Recruiting
Testing and validating pain and symptom questionnaires for NF1
This study checks whether patient surveys (questionnaires) can reliably measure symptoms and pain in people with NF1 who have plexiform neurofibromas. It helps create better tools for tracking how you feel and how treatments may affect quality of life.
Washington D.C., District of ColumbiaAges 5 years+ - NCT04750928RecruitingPhase 1/Phase 2
Abemaciclib for NF1 tumors using pills and body scans
This trial studies whether the medicine abemaciclib can shrink or slow the growth of “atypical neurofibromas” in people with NF1. Participants take pills and undergo regular check-ups, including MRI scans to measure the tumor.
Bethesda, MarylandAges 12 years+ - NCT05735717RecruitingPhase 2
T-cell transplant using depleted receptors for blood cancers
This Phase 2 study tests a specialized stem cell transplant that removes certain immune “T-cell” receptors to help treat blood cancers like acute leukemia. It may help your body rebuild a healthier immune system after high-dose treatment.
Minneapolis, MinnesotaAges Up to 60 years - NCT05331105RecruitingPhase 2
Drug for adults with NF1 and non-surgically treatable nerve tumors
This Phase 2 trial tests HL-085 in adults with neurofibromatosis type 1 (NF1) who have a nerve tumor (plexiform neurofibroma) that cannot be removed by surgery and causes symptoms. It aims to shrink or control the tumor by measuring changes on MRI and monitoring side effects.
Shanghai, Shanghai MunicipalityAges 18–80 - NCT06222203Recruiting
Monitoring for tumor changes in people with NF1
This study tracks people with NF1 to watch for when benign nerve tumors turn cancerous. It uses regular MRI scans to catch changes early, which could help guide treatment decisions.
Bethesda, MarylandAges 3–120 - NCT06299839RecruitingPhase 1
Study of PAS-004 for advanced solid tumors
This phase 1 trial tests an oral drug called PAS-004 for people with advanced solid tumors that have certain gene changes. It aims to find a safe dose and see if the drug can stop tumor growth.
Austin, TexasAges 18 years+ - NCT06507748Recruiting
Measuring pain with a finger device in NF1
This study tests a new way to measure pain and other sensations in people with NF1 using a device that clips onto a finger or toe. The device looks at how your pupil reacts to mild pulses, which may help doctors better understand your pain.
Washington D.C., District of ColumbiaAges 1–120 - NCT07221331Recruiting
Neurofibromatosis type 1 study in Egypt
This study looks at how neurofibromatosis type 1 (NF1) affects people in Egypt, including how it progresses and how it is managed. Researchers will review medical records of patients diagnosed with NF1 between 2010 and 2023 to learn more about the condition.
Al MansurahAges Any age - NCT07237100RecruitingPhase 2
Mirdametinib for advanced NF1-mutant melanoma
This trial tests an experimental oral drug called mirdametinib for people with advanced melanoma that has a specific gene change (NF1 mutation). To join, you must have already had immunotherapy, but not a MEK/ERK/RAF targeted drug before.
San Francisco, CaliforniaAges 18 years+ - NCT02390752RecruitingPhase 1
Early drug study for children and young adults with hard-to-treat cancers
This Phase 1 trial tests pexidartinib (TURALIO) to see how safe it is and how well it works against refractory (hard-to-treat) leukemias and solid tumors, including tumors seen with NF1 and MPNST. It mainly helps researchers learn the best way to dose the medication and gather early signals of benefit.
Bethesda, MarylandAges 3–35 - NCT06300502Enrolling by invitationPhase 1
Monthly injections for NF1 skin bumps
This study tests if a monthly injection of deoxycholate can shrink or reduce the number of visible skin bumps (cutaneous neurofibromas) in people with NF1. It is designed for adults with at least 6 bumps that can be treated and monitored.
Boston, MassachusettsAges 18–85 - NCT05238909Enrolling by invitation
Tracking tumor burden in people with NF1
This study looks for better ways to measure how much tumor activity people with neurofibromatosis type 1 (NF1) have over time. The goal is to develop “biomarkers,” which are test results that may help doctors track disease more easily.
Chicago, IllinoisAges Any age
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Common questions
- Are there clinical trials for nf1?
- Yes. Clin2 currently lists 19 recruiting nf1 studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a nf1 trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a nf1 trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.