Observational Study of Neurofilament Light Chain (NfL) Levels in Asymptomatic Carriers of the TTR Gene Variants and Patients With hATTR Amyloidosis With Polyneuropathy, Including Patients Who Undergo Treatment Change - NeuroFeeL Study

Status: Recruiting
Location: See location...
Intervention Type: Other
Study Type: Observational
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: t
View:

• \- 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

• OR

• 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.

Locations
Other Locations
France
Centre Hospitalier Universitaire (CHU) Le Kremlin-Bicêtre Assistance Publique-Hôpitaux de Paris (APHP)
RECRUITING
Paris
Contact Information
Primary
Alnylam Clinical Trial Information Line
clinicaltrials@alnylam.com
1-877-ALNYLAM
Time Frame
Start Date: 2024-04-25
Estimated Completion Date: 2027-12-01
Participants
Target number of participants: 500
Treatments
Asymptomatic Carriers
Participants who are asymptomatic carriers of a pathogenic TTR variant, and not diagnosed with hATTR amyloidosis with polyneuropathy will be enrolled in this cohort.
Participants with hATTR Amyloidosis with Polyneuropathy
Participants with a confirmed diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR variant will be enrolled in this cohort.
Related Therapeutic Areas
Sponsors
Leads: Alnylam Pharmaceuticals

This content was sourced from clinicaltrials.gov