Actively Recruiting
Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy
Led by Alnylam Pharmaceuticals · Updated on 2025-12-24
500
Participants Needed
1
Research Sites
187 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a single-center observational study evaluating the potential value of NfL as a biomarker for diagnosis, detection of disease onset, monitoring of disease progression, and treatment response in asymptomatic carriers of TTR variants and symptomatic hATTR amyloidosis patients with polyneuropathy.
CONDITIONS
Official Title
Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Carrier of a documented pathogenic TTR variant confirmed with genotyping with predicted disease onset within 5 years and not diagnosed with hATTR amyloidosis with polyneuropathy
- Confirmed diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR variant confirmed with genotyping
- Participant is able to understand the study and does not oppose participating in the study after reviewing the content of the PIS provided.
You will not qualify if you...
- A known condition (other than hATTR amyloidosis) that can cause nerve damage and affect NfL levels
- Estimated glomerular filtration rate (eGFR) <45 milliliters per minute per 1.73 meters squared (mL/min/1.73 m²)
- Currently enrolled in a clinical study for any investigational agent.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Centre Hospitalier Universitaire (CHU) Le Kremlin-Bicêtre Assistance Publique-Hôpitaux de Paris (APHP)
Paris, Île-de-France Region, France, 94270
Actively Recruiting
Research Team
A
Alnylam Clinical Trial Information Line
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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