Blood marker may predict nerve damage onset in rare genetic disease

NCT ID NCT06360289

First seen Jun 26, 2026 · Last updated Aug 13, 2026 · Updated 3 times

Summary

This observational study is looking at a protein called neurofilament light chain (NfL) in the blood of people who carry a gene variant for hereditary ATTR amyloidosis, both those without symptoms and those with nerve damage. Researchers want to see if NfL levels can help detect when the disease starts, how it progresses, and how well treatments work. About 346 participants are being followed over time, but no new drugs are being tested.

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 could show that a simple blood test (NfL) helps doctors detect when the disease starts and monitor how it progresses, leading to earlier treatment decisions.
What could go wrong
This is an observational study, not a treatment trial. It only measures a biomarker, so it cannot prove that NfL improves patient outcomes. Results may not change current care.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Centre Hospitalier Universitaire (CHU) Le Kremlin-Bicêtre Assistance Publique-Hôpitaux de Paris (APHP)

    Paris, Île-de-France Region, 94270, France

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