A Clinical Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Patients With hATTR-PN

Study Overview
=================
Brief Summary
-----------------
The objective of the study is to characterize adverse events (AEs) occurring within one day of TEGSEDI administration to adult patients with hATTR-PN overall and in individual patients with respect to time course of AE onset, vital sign changes, preventive measures, treatment required, risk factors, and subsequent adverse outcomes.

Official Title
-----------------
A Phase 4 Safety Study Assessing the Adverse Events Occurring Within One Day of TEGSEDI Administration in Patients With Polyneuropathy of Hereditary Transthyretin-mediated Amyloidosis (hATTR-PN)

Conditions
-----------------
Hereditary Transthyretin Amyloidosis With Polyneuropthy

Intervention / Treatment
-----------------
* Procedure: Blood samples for immunoglobulins
* Procedure: Blood samples for inflammatory markers
* Procedure: Blood samples for anti-drug antibodies


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Satisfy one of the following: US Patients: Adult patients (≥ 18 years old) diagnosed with hATTR-PN and prescribed TEGSEDI according to the USPI Canadian Patients: Adult patients (≥ 18 years old) diagnosed with stage 1 or stage 2 hATTR PN and prescribed TEGSEDI according to the CPM Must have given written informed consent for participation in this study Must provide access to their previous medical records Are about to initiate or have recently initiated treatment with TEGSEDI and have not received more than 9 doses in total Be willing to complete required testing and report any AEs and/or changes in medications Satisfy one of the following: Females: Non-pregnant and non-lactating; abstinent, or if engaged in sexual relations of childbearing potential, patient is using an acceptable contraceptive method from time of signing the informed consent form (ICF) until 13 weeks after the last dose of TEGSEDI administration Males: Abstinent or if engaged in sexual relations with a female of childbearing potential, patient is utilizing an acceptable contraceptive method from the time of signing the ICF until 13 weeks after the last dose of TEGSEDI administration Exclusion Criteria: None

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Group 1<br>TEGSEDI | Procedure: Blood samples for immunoglobulins<br>* Following an injection of TEGSEDI as prescribed by their physician per the product label, patients will have additional blood tests at 1, 2, 4, 8 and 24 hours post injection for immunoglubulins at a clinic visit every 4 months for up to 2 years.<br>Procedure: Blood samples for inflammatory markers<br>* Following an injection of TEGSEDI as prescribed by their physician per the product label, patients will have additional blood tests at 1, 2, 4, 8 and 24 hours post injection for inflammatory markers at a clinic visit every 4 months for up to 2 years.<br>Procedure: Blood samples for anti-drug antibodies<br>* Patients taking TEGSEDI as prescribed by their physician per the product label, patients will have additional blood samples for anti-drug antibodies at various time-points as indicated in the event of hypersensitivity reactions.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To characterize AEs occurring within one day of TEGSEDI administration to adult patients with hATTR-PN. | All treatment emergent AEs potentially related to TEGSEDI will be summarized. | 1 year minimum to 2 years maximum | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Hereditary Transthyretin Amyloidosis, hATTR-PN, hATTR, Amyloidosis