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

Sponsor

Akcea Therapeutics (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04306510

Collaborator

(none)

Enrollment

Locations

Arm

49.7

Anticipated Duration (Months)

18.8

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Hereditary Transthyretin Amyloidosis With Polyneuropthy	Procedure: Blood samples for immunoglobulins Procedure: Blood samples for inflammatory markers Procedure: Blood samples for anti-drug antibodies	Phase 4

Study Design

Study Type:

Interventional

Anticipated Enrollment :

75 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Other

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)

Actual Study Start Date :

Jan 21, 2021

Anticipated Primary Completion Date :

Mar 15, 2025

Anticipated Study Completion Date :

Mar 15, 2025

Arms and Interventions

Arm	Intervention/Treatment
Other: Group 1 TEGSEDI	Procedure: Blood samples for immunoglobulins 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. Procedure: Blood samples for inflammatory markers 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. Procedure: Blood samples for anti-drug antibodies 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.

Arm

Intervention/Treatment

Other: Group 1

TEGSEDI

Procedure: Blood samples for immunoglobulins

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.

Procedure: Blood samples for inflammatory markers

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.

Procedure: Blood samples for anti-drug antibodies

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.

Outcome Measures

Primary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Study Center	Rosedale	New York	United States	11422
2	Study Center	Portland	Oregon	United States	97239
3	Study Center	Philadelphia	Pennsylvania	United States	19104
4	Study Center	Toronto	Ontario	Canada	M3K0A6