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

Hereditary Transthyretin Amyloidosis With Polyneuropthy Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04306510
• Other study ID #: TEG4004
• Status: Recruiting
• Phase: Phase 4
• Start date: January 21, 2021
• Est. completion date: March 15, 2025

Study information

• Verified date: October 2023
• Source: Akcea Therapeutics
• Contact: Ionis Pharmaceuticals
• Phone: (844) 742-0280
• Email: ionisNCT04306510study[@]clinicaltrialmedia.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 75
• Est. completion date: March 15, 2025
• Est. primary completion date: March 15, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Amyloid Neuropathies, Familial
• Amyloidosis
• Hereditary Transthyretin Amyloidosis With Polyneuropthy

Intervention

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.
• 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.
• 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.

Locations

Country	Name	City	State
Canada	Study Center	Toronto	Ontario
United States	Study Center	Rosedale	New York

Sponsors (1)

Lead Sponsor	Collaborator
Akcea Therapeutics

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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