Clinical trials
Neurofibromatosis Type 1 clinical trials
Below are recruiting neurofibromatosis type 1 clinical trials, each written for real people, not researchers. We’re tracking 20 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06880991Recruiting
Study on visible tumors in neurofibromatosis type 1
This study talks with people who have NF1 (and caregivers of children with NF1) about how visible tumors affect them. The goal is to create questionnaires that measure these concerns, which could lead to better support and treatments.
Bethesda, MarylandAges 5–120 - NCT03050268Recruiting
Study of childhood cancer risk in families
This study looks at inherited (family) risk for childhood cancers, including families with early cancers or known cancer risk conditions. It may help researchers understand why some children get cancer and improve future risk detection and care.
Memphis, TennesseeAges Any age - NCT04763109Recruiting
MRI scan with AI to spot early changes in NF1 children
This study uses special MRI scans plus computer (AI) analysis to look for early, possibly cancer-related skin or tissue changes in children with neurofibromatosis type 1 (NF1). If it works, it could help doctors detect concerning changes earlier and plan care sooner.
Los Angeles, CaliforniaAges 5–17 - 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 - NCT06188741RecruitingPhase 2
Selumetinib to prevent tumor growth in NF1 children
This study tests whether the drug selumetinib can prevent the growth of plexiform neurofibromas (nerve tumors) in young children with NF1. Children are first monitored with MRI scans, and if a tumor appears or grows, they may receive treatment.
Birmingham, AlabamaAges 1–8 - 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 - 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 - NCT06541847RecruitingPhase 2
Testing HLX-1502 for NF1 with Plexiform Neurofibromas
This trial tests an experimental drug called HLX-1502 for people with NF1 who have a plexiform neurofibroma (a type of nerve tumor) that is growing or causing problems. It aims to see if the drug is safe and can shrink or stop the tumor from worsening.
Birmingham, AlabamaAges 12 years+ - 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+ - 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 - 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 - 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+ - NCT05199376Recruiting
Cryotherapy treatment for painful, hard-to-remove NF1 tumors
This trial tests whether freezing treatment done through the skin (cryotherapy) can reduce pain and discomfort from certain non-cancer NF1 tumors that can’t be safely removed. You may be able to join if you’re an adult with NF1, a painful or bothersome benign lesion, and can safely undergo procedures like MRI.
LyonAges 18 years+ - NCT05954416Recruiting
Study follow-up for rare skin and related genetic conditions
This study (a “cohort”) follows people with certain rare skin conditions over time. It helps doctors better understand these diseases and improve long-term care, and you may be asked to complete surveys.
BobignyAges Any age - 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 - NCT06523582Recruiting
Genetic study of neuroendocrine tumors in Mexican patients
This study aims to find genetic causes of neuroendocrine tumors in Mexican patients. By understanding your DNA, researchers hope to improve diagnosis and treatment for people with these rare conditions.
Mexico City, Mexico CityAges 18 years+ - NCT07407803RecruitingPhase 3
TQ-B3234 for adults with non-surgical neurofibromatosis type 1 tumors
This trial tests an oral capsule called TQ-B3234 for adults who have painful, inoperable nerve tumors from neurofibromatosis type 1. It aims to see if the drug can shrink these tumors or stop them from growing.
Hefei, AnhuiAges 18 years+ - NCT04374305RecruitingPhase 2
Study tests targeted treatments for NF2 tumors
This trial studies targeted medicines in people with neurofibromatosis type 2 (NF2) who have growing tumors on scans. The goal is to slow tumor growth and improve symptoms by testing different drug combinations in separate study groups.
Los Angeles, CaliforniaAges 12 years+ - NCT05849662RecruitingPhase 1/Phase 2
Trametinib plus azacitidine for newly diagnosed JMML
This trial studies whether trametinib and azacitidine can control juvenile myelomonocytic leukemia (JMML) soon after diagnosis. It’s designed to test safety and how well the treatment works in children and young adults who meet specific leukemia and genetic criteria.
Phoenix, ArizonaAges 1 month–21 years - 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
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Neurofibromatosis Type 1 trials by city
Studies with a site in or near these metro areas.
Neurofibromatosis Type 1 trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for neurofibromatosis type 1?
- Yes. Clin2 currently lists 20 recruiting neurofibromatosis type 1 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 neurofibromatosis type 1 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 neurofibromatosis type 1 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.