NCT05879874Active, not recruiting
Prospective Evaluation of NfL as a Biomarker in ATTRv
Prospective Evaluation of Neurofilament Light Chains as a Biomarker in Hereditary Amyloidosis (ATTRv), Including Correlation With Various Measurements of Disease Severity and Progression.
Source: ClinicalTrials.gov · Last updated 14 Aug 2025
Brief summary (registry, in English)
ATTR amyloidosis is a rare and progressively disabling disease caused by the deposition of misfolded TTR protein in multiple tissues including the nerves, heart, and gastrointestinal tract. Polyneuropathy (PN) and cardiomyopathy (CM) are the two most frequent phenotypes and many patients presented a mixed picture of PN and CM. There are different methods to search for the existence and extent of PN and disability caused by ATTR amyloidosis (e.g. mNIS+7, Norfol, QoL-DN), these methods may not be sensitive enough to search for the onset of disease in patients carrying the pathogenic TTR variants or progression of PN in patients undergoing treatment. Also, some of the available methods can be difficult and time-consuming to perform. For this reason, there is a need for sensitive biomarkers that can aid in the investigation and follow-up of PN in patients with hATTR amyloidosis. NfL, a well-known biomarker of nerve damage due to both central and peripheral nervous system disorders, was recently evaluated as a potential biomarker of nerve damage in patients with hATTR amyloidosis. The results of this study can help understand the potential value of NfL in patients with PN of hATTR amyloidosis establishing i changes levels of this biomarker in response to different pathology-specific treatment options correlation between NfL levels and different ratings clinics. The primary objective of the study is to establish the potential of NfL as a biomarker of severity of polyneuropathy, progression and response to treatment in patients with symptomatic hATTR amyloidosis.
- Status (registry)
- Active, not recruiting
- Study type
- Observational
- Enrollment
- 40 (estimated)
- First submitted
- 9 May 2023
- Start date
- 1 Sept 2023
- Primary completion date
- Jun 2026
- Lead sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Diseases
- Amyloidosis
- NCT number
- NCT05879874
What you commit to
- Study type: Observational
- Planned participants: 40
- From 1 Sept 2023 to the primary completion date, Jun 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Age
- Patients aged \< 18 years
Other
- Pregnant women
- Patients who refused to give their consent to participate in the study.
Who the study is looking for
- Adult patients with hATTR amyloidosis with polyneuropathy with a confirmed TTR disease-causing mutation (NIS score between 5 and 130 points at baseline).
- Patients on hATTR amyloidosis therapy with gene silencers (patisiran or inotersen) and TTR stabilizers (tafamidis at doses of 20 or 61 mg).
- Patients who gave consent to participate in the study.
Age in the registry: 18 years to 100 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Fondazione Policlinico Gemelli IRCCS Coordinator of this site? Confirm its status | Rome | Italy | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.