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Neurofibromatosis type 1

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Where research stands todayUpdated 15 Nov 2026
Trial-stage treatments you can join–open now
in United States
Studies published in the last 12 months00 in people · 0 in animals · 0 in the lab
Support organisations2organisations
in United States1 helpline
Offer tissue or dataGive a sample or your health data to research.Approved treatmentsMedicines approved for neurofibromatosis type 1 around the world.“Miracle cure” fact checkerSaw a cure online? Check it before you pay.

Trial-stage treatments and research studies you can join

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Get notified of new trialsUnited States, all cities, all phases
Your ageShows only trials that take people your age.
Phase
  1. Trial-stage treatmentPhase 3Taking people
    Phase 3 trial: Carboplatin, Selumetinib Sulfate, Vincristine Sulfate
    Akron, United StatesAlso Albany, Ann Arbor, Atlanta, Aurora, Austin, Baltimore, Bangor, Bethesda, …Age 2 to 21
  2. Trial-stage treatmentPhase 1/2Taking people
    Tests abemaciclib for nerve tumors in people with neurofibromatosis type 1

    Tests abemaciclib capsules in people 12 and older with neurofibromatosis type 1 and atypical neurofibromas.

    Bethesda, United StatesAge 12+
  3. Trial-stage treatmentPhase 1Taking people
    Tests pexidartinib in young people with refractory leukemias and solid tumors

    Tests pexidartinib in people aged 3 to 35 with leukemia or solid tumors.

    Bethesda, United StatesAge 3 to 35
  4. Trial-stage treatmentPhase 1Taking people
    Phase 1 trial: PAS-004 Tablets, PAS-004 Capsule
    Birmingham, United StatesAge 18+
  5. Trial-stage treatmentTaking people
    Measuring pain in neurofibromatosis type 1 using pupil response testing

    Tests a device that measures pain through pupil changes in people with NF1.

    Bethesda, United StatesAlso Washington D.C.Age 1 to 120
  6. Trial-stage treatmentPhase 2Soon in United States
    Mirdametinib, alone or with radiation, for high-grade brain tumors with NF1 changes

    Tests mirdametinib, alone or with radiation, in people with NF1-altered high-grade glioma.

    Birmingham, United StatesAge 12 to 89
  7. Trial-stage treatmentPhase 1Taking people
    Testing Cabozantinib and Selumetinib Together for Nerve Tumors in Neurofibromatosis Type 1

    Tests two cancer drugs together in people with neurofibromatosis type 1 and nerve tumors.

    Birmingham, United StatesAlso Boston, Cincinnati, Dallas, Indianapolis, Los Angeles, Philadelphia, Washington D.C.Age 16+
  8. Trial-stage treatmentPhase 1/2Taking people
    Tests trametinib and azacitidine in children with new juvenile myelomonocytic leukemia

    Tests trametinib and azacitidine, with or without chemotherapy, in children newly diagnosed with JMML.

    Ann Arbor, United StatesAlso Atlanta, Baltimore, Charlotte, Chicago, Cincinnati, Dallas, Denver, Indianapolis, …Age 0 to 21
  9. Trial-stage treatmentPhase 2Taking people
    Selumetinib, ZEN-3694 and durvalumab for hard-to-treat sarcomas, including MPNST

    Tests selumetinib with ZEN-3694 and durvalumab in adults with refractory sarcomas.

    Birmingham, United StatesAge 18 to 99
  10. Trial-stage treatmentPhase 2Taking people
    Tests HLX-1502 in people with neurofibromatosis type 1 and plexiform neurofibromas

    Tests HLX-1502 in people aged 12 and older with neurofibromatosis type 1.

    Aurora, United StatesAlso Baltimore, Birmingham, Chicago, Cincinnati, Dallas, Indianapolis, Los Angeles, New York, …Age 12+
  11. Trial-stage treatmentPhase 1/2Taking people
    Tests selumetinib with APG-115 in adults with NF1 nerve sheath tumors

    Tests selumetinib and APG-115 in adults with neurofibromatosis type 1 and nerve sheath tumors.

    Dallas, United StatesAlso Washington D.C.Age 18+
  12. Trial-stage treatmentPhase 2Taking people
    Tests selumetinib to prevent growth of nerve tumors in neurofibromatosis type 1

    Tests selumetinib early in children with neurofibromatosis type 1 and nerve tumors.

    Baltimore, United StatesAlso Bethesda, Birmingham, Boston, Chicago, Cincinnati, Dallas, Indianapolis, Los Angeles, …Age 1 to 8
  13. Trial-stage treatmentPhase 1Taking people
    Phase 1 trial: Imlygic
    Baltimore, United StatesAge 18+
  14. Trial-stage treatmentPhase 1
    Phase 1 trial: Kybella
    Boston, United StatesAge 18 to 85
  15. Trial-stage treatmentPhase 1/2Taking people
    Phase 1/2 trial: Mirdametinib
    Baltimore, United StatesAge 18+
  16. Trial-stage treatmentPhase 2Taking people
    Tests a stem cell transplant method for blood cancers

    Tests a T cell-depleted stem cell transplant in people with leukemia.

    Minneapolis, United StatesUp to age 60
  17. Research studyTaking people
    Tracking nerve tumors in people with neurofibromatosis type 1 over time

    Checks whether benign nerve tumors in neurofibromatosis type 1 patients become cancerous.

    Bethesda, United StatesAge 3 to 120
  18. Research studyTaking people
    Improving questionnaires about pain and daily life in neurofibromatosis 1

    Improves questionnaires measuring pain and daily life in people with plexiform neurofibromas.

    Baltimore, United StatesAlso Bethesda, Chicago, Cincinnati, Philadelphia, Washington D.C.Age 5+
  19. Research studyTaking people
    Registry collecting patient-reported data on neurofibromatosis and related conditions

    Collects patient-reported symptoms and treatments for people with neurofibromatosis and schwannomatosis.

    New York, United States
  20. Trial-stage treatmentTaking people
    Clinical trial: Neurofibromatosis 1
    Bethesda, United StatesAge 18+

Support organisations

Patient organisations and helplines in United States, for you and your family.

We have no organisations for this disease listed in United States yet. General support there is below.

General support in United States

  • 988 Suicide & Crisis Lifeline

    US national 24/7 free and confidential lifeline for anyone in suicidal crisis or emotional distress, reachable by calling or texting 988 or by chat.

    988lifeline.org
  • MedlinePlus

    Free, easy-to-read health information service of the US National Library of Medicine (NIH) on diseases, conditions, drugs and wellness.

    medlineplus.gov

International organisations

Patient organisation umbrella bodies

Offer tissue or data

There are three ways to help research move faster.

Give tissue or bloodA biobank stores your sample so many research teams can use it.No open programme yet
Share your health dataA patient registry collects your health data, without your name, for research.How to join
Take part as a healthy volunteerStudies also need people without the disease, for comparison. Family and friends can help.Volunteer

“Miracle cure” fact checker

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  1. Is there a published study in people?
  2. Can I have the full price in writing?
  3. What does my doctor say?

“Cures” others already fact-checked

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