Clinical trials
Neurofibromatosis clinical trials
Below are recruiting neurofibromatosis clinical trials, each written for real people, not researchers. We’re tracking 37 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06502171RecruitingPhase 1
Cabozantinib and selumetinib for plexiform neurofibromas
This trial tests a combination of two targeted drugs (cabozantinib and selumetinib) for people with NF1 who have plexiform neurofibromas that are growing or causing significant problems. The goal is to see if this combination can shrink or slow the growth of these tumors.
Birmingham, AlabamaAges 16 years+ - NCT06360406Recruiting
Real-world study of Koselugo (selumetinib) in South Korea
This study looks at how patients with certain conditions are doing while taking Koselugo (selumetinib) in real life. It's a way to gather information about the treatment's effectiveness and safety outside of a controlled clinical trial.
BusanAges 3–99 - 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 - 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 - 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+ - NCT06147414Recruiting
Blood test for genetic disorders in pregnancy
This study tests a safer blood test to check for certain genetic disorders during pregnancy, instead of an invasive procedure like amniocentesis. It's for pregnant women who are at risk of passing on a known genetic condition.
ParisAges 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 - 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 - 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 - NCT05361811Recruiting
Therapy coaching for parents of children with rare genetic syndromes
This trial tests an “Acceptance and Commitment Therapy” program for parents or legal guardians who feel moderate parenting stress. It aims to help caregivers cope in healthier ways and may also improve how they manage stress while supporting their child.
Bethesda, MarylandAges 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+ - NCT03893643Recruiting
Skin and mouth symptoms of NF2 in children under 15
This study looks at skin and mouth problems that children with NF2 may have. It helps doctors learn more about NF2 symptoms in kids, which may lead to better care.
NiceAges birth–15 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
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Common questions
- Are there clinical trials for neurofibromatosis?
- Yes. Clin2 currently lists 37 recruiting neurofibromatosis 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 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 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.