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Clinical Trials in France / NCT06360289
Active, not recruiting Observational

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

NCT06360289 · tracked via the Priya Life Science France tracker
Sponsor
Alnylam Pharmaceuticals
Phase
Observational
Started
2024-04-25
Last updated
2026-08-12

Condition(s) studied

Hereditary Amyloidosis, Transthyretin-RelatedAsymptomatic Carrier State

Investigational drug(s) / intervention(s)

Standard of Care

Standard of Care: This is an observational study. No study drug will be administered as a part of the study, participants will be treated according to the decision of the treating physician.

Study 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

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
Accepted
Inclusion Criteria: \- 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. Exclusion Criteria: * 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 m\^2) * Currently enrolled in a clinical study for any investigational agent.

Primary outcome measure(s)

  • NfL Levels in Asymptomatic Carriers of a TTR Variant and Symptomatic hATTR Patients With Polyneuropathy at Baseline — Baseline
  • Change in NfL Levels Over Time in Asymptomatic Carriers of a TTR Variant and Symptomatic hATTR Patients With Polyneuropathy — Up to 24 months

Trial sites (1)

FacilityCityRegionStatus
Centre Hospitalier Universitaire (CHU) Le Kremlin-Bicêtre Assistance Publique-Hôpitaux de Paris (APHP) Paris Île-de-France Region

More Alnylam Pharmaceuticals trials in France

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Active, not recruiting
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Enrolling by invitation
BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1) Active, not recruiting
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT) Active, not recruiting
TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06360289 on ClinicalTrials.gov ↗ ← All trials in France