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

NCT04306510 | Terminated Phase 4 Results FDA Drug

• 1 other identifier: TEG4004
• Study Type: interventional
• Target: 8
• Locations: 2 countries
• Sites: 2

Brief Summary

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

Conditions

Hereditary Transthyretin Amyloidosis With Polyneuropthy

Keywords

Hereditary Transthyretin Amyloidosis; hATTR-PN; hATTR; Amyloidosis

Outcome Measures

Primary Outcomes (3)

• Number of Participants With Incident, Onset and Duration of Treatment Emergent Adverse Events (TEAEs) Occurring Within 24 Hours of Each TEGSEDI Administration

  An adverse event (AE) is defined as any unfavorable and unintended sign, symptom, or disease temporally associated with the use of medicinal product, whether or not the AE is considered related to the medicinal product. TEAEs are defined as AEs with an onset date/time on or after the date/time of the first on study administration of TEGSEDI.

  Up to 2 years (24 hours post each TEGSEDI injection)

• Number of Participants With Clinically Significant Changes in Vital Signs

  Vital signs including body temperature, heart rate, respiratory rate, and systolic/diastolic blood pressure (BP).

  Up to 2 years (24 hours post each TEGSEDI injection)

• Number of Participants With Clinically Significant Changes in Cytokine Levels and Inflammatory Markers

  Cytokines and inflammatory markers including the following parameters: Immunoglobulin (Ig)E, IgG, IgM, C-reactive protein, erythrocyte sedimentation rate, interferon-alpha, interferon beta, chemokines (macrophage inflammatory protein-1a and membrane cofactor protein-1, granulocyte-macrophage colony-stimulating factor, Interleukin (IL)-1alpha (α), IL-1 beta (β), IL-6, IL-8, IL-12, and tumor necrosis factor-α.

  Up to 2 years (24 hours post each TEGSEDI injection)

Study Arms (1)

TEGSEDI

EXPERIMENTAL

Participants received TEGSEDI 284 milligrams (mg), subcutaneously (SC) once weekly, as prescribed by their physician per the product label.

Drug: TEGSEDI

Interventions

TEGSEDI DRUG

SC injection.

Also known as: Inotersen

TEGSEDI

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Satisfy one of the following:
• United States (US) Participants: Adult participants (≥ 18 years old) diagnosed with hATTR-PN and prescribed TEGSEDI according to the United States Prescribing Information (USPI).
• Canadian participants: Adult participants (≥ 18 years old) diagnosed with stage 1 or stage 2 hATTR-PN and prescribed TEGSEDI according to the Canadian Product Monograph (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, participant 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, participant is utilizing an acceptable contraceptive method from the time of signing the ICF until 13 weeks after the last dose of TEGSEDI administration

You may not qualify if:

• None

Sponsors & Collaborators

• Akcea Therapeutics: lead

Study Sites (2)

Study Center

Rosedale, New York, 11422, United States

Location

Study Center

Toronto, Ontario, M3K0A6, Canada

Location

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-Related; Amyloidosis

Interventions

Inotersen

Condition Hierarchy (Ancestors)

Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Limitations and Caveats

This study was subject to limitations due to the rarity of hATTR-PN, which limits the enrollment of potentially eligible patients. Additionally, the availability of concurrent clinical trials and newer therapies targeting the same patient population further complicated efforts to achieve the planned enrollment.

Results Point of Contact

• Title: Ionis Pharmaceuticals, Inc.
• Organization: Ionis Pharmaceuticals, Inc.

globalregulatoryaffairs@ionis.com

760-603-2346

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 7, 2020
• First Posted: March 13, 2020
• Study Start: January 21, 2021
• Primary Completion: March 20, 2024
• Study Completion: March 20, 2024
• Last Updated: April 10, 2025
• Results First Posted: April 10, 2025

Record last verified: 2025-03

Locations

US (1); CA (1)