Ignite Creation Date:
2024-05-06 @ 8:23 PM
Last Modification Date:
2024-10-26 @ 3:26 PM
Study NCT ID:
NCT06360289
Status:
RECRUITING
Last Update Posted:
2024-06-27
First Post:
2024-04-08
Brief 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
Sponsor:
Alnylam Pharmaceuticals
Organization:
Alnylam Pharmaceuticals
Study Overview
Official Title:
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
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
Detailed Description:
In this study participants data will be extracted from their medical records or collected based on clinical and laboratory assessments during routine visits per the sites standard of care Blood samples collected from the participants during routine visits will also be used for analysis The study will be conducted in two parts Cross-Sectional part during which a single measurement of NfL levels will be performed using blood samples collected from asymptomatic carriers and symptomatic hATTR amyloidosis patients Longitudinal part during which measurements of NfL levels will be performed over time using blood samples already collected from the participants during routine visits from asymptomatic carriers and patients with symptomatic hATTR amyloidosis
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None