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

Phase 4TerminatedNCT04306510

Akcea Therapeutics8 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

OTHER

Masking

NONE

Enrollment

Conditions

Hereditary Transthyretin Amyloidosis With Polyneuropthy

Interventions

TEGSEDIDRUG

SC injection.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Satisfy one of the following: 1. United States (US) Participants: Adult participants (≥ 18 years old) diagnosed with hATTR-PN and prescribed TEGSEDI according to the United States Prescribing Information (USPI). 2. 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). 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, 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 2. 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 Exclusion Criteria: * None

Locations (2)

Study Center

Rosedale, New York, United States

Study Center

Toronto, Ontario, Canada

Outcomes

Primary Outcomes

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.

Time frame: 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).

Time frame: 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.

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

Data from ClinicalTrials.gov

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