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

October 31, 2023 updated by: Akcea Therapeutics

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)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

75

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M3K0A6
        • Recruiting
        • Study Center
  • United States
    • New York
      • Rosedale, New York, United States, 11422
        • Recruiting
        • Study Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Satisfy one of the following:

    1. US Patients: Adult patients (≥ 18 years old) diagnosed with hATTR-PN and prescribed TEGSEDI according to the USPI
    2. Canadian Patients: Adult patients (≥ 18 years old) diagnosed with stage 1 or stage 2 hATTR PN and prescribed TEGSEDI according to the CPM
  2. Must have given written informed consent for participation in this study
  3. Must provide access to their previous medical records
  4. Are about to initiate or have recently initiated treatment with TEGSEDI and have not received more than 9 doses in total
  5. Be willing to complete required testing and report any AEs and/or changes in medications

  6. Satisfy one of the following:

    1. 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
    2. 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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.

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.
Time Frame: 1 year minimum to 2 years maximum
All treatment emergent AEs potentially related to TEGSEDI will be summarized.
1 year minimum to 2 years maximum

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Akcea Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 21, 2021

Primary Completion (Estimated)

March 15, 2025

Study Completion (Estimated)

March 15, 2025

Study Registration Dates

First Submitted

February 7, 2020

First Submitted That Met QC Criteria

March 10, 2020

First Posted (Actual)

March 13, 2020

Study Record Updates

Last Update Posted (Actual)

November 2, 2023

Last Update Submitted That Met QC Criteria

October 31, 2023

Last Verified

October 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TEG4004

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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