10-Year NF1 study aims to unlock secrets of rare genetic disorder

NCT ID NCT00924196

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This natural history study follows 259 children, adolescents, and adults with Neurofibromatosis Type 1 (NF1) for up to 10 years. Researchers will track tumor growth, monitor quality of life, and perform genetic testing to better understand how the disease changes over time. The goal is to gather information that could guide future treatments and improve care for people with NF1.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

What this could lead to

If successful, this study could provide crucial insights into how NF1 progresses, helping design future treatments and clinical trials.

What could go wrong

This is an observational study, not a treatment trial. It will not directly improve health, and results may take years to impact patient care.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

malignant peripheral nerve sheath tumor neurofibroma neurofibromatosis type 1 neurofibrosarcoma optic nerve glioma optic pathway glioma plexiform neurofibroma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States