Trial Outcomes & Findings for A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PN (NCT NCT04306510)

Last Updated: 2025-04-10

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE4

Target enrollment

8 participants

Primary outcome timeframe

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

Results posted on

2025-04-10

Participant Flow

Participants took part at 2 clinical sites in the United States of America (USA) and Canada from 21 January 2021 to 20 March 2024.

A total of 8 participants with polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) were screened for the study, of which 7 participants were treated with TEGSEDI. The 8th participant was enrolled after the sponsor had notified the sites about the study close-out activities, therefore only 7 participants were included in the study.

Participant milestones

Participant milestones
Measure	TEGSEDI Participants received TEGSEDI 284 milligrams (mg), subcutaneously (SC) once weekly, as prescribed by their physician per the product label.
Overall Study STARTED	8
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	TEGSEDI Participants received TEGSEDI 284 milligrams (mg), subcutaneously (SC) once weekly, as prescribed by their physician per the product label.
Overall Study Voluntarily withdrawal	1
Overall Study Enrolled but Not Treated	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	TEGSEDI n=7 Participants Participants with hATTR-PN received TEGSEDI 284 mg, SC once weekly, as prescribed by their physician per the product label.
Age, Continuous	67.6 years STANDARD_DEVIATION 8.62 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	2 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

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

Population: The evaluable set included participants in the safety set having at least 1 post-dose assessment.

Outcome measures

Outcome measures
Measure	TEGSEDI n=7 Participants Participants received TEGSEDI 284 mg, SC once weekly, as prescribed by their physician per the product label.
Number of Participants With Incident, Onset and Duration of Treatment Emergent Adverse Events (TEAEs) Occurring Within 24 Hours of Each TEGSEDI Administration	3 Participants

PRIMARY outcome

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

Population: The evaluable set included participants in the safety set having at least 1 post-dose assessment.

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

Outcome measures

Outcome measures
Measure	TEGSEDI n=7 Participants Participants received TEGSEDI 284 mg, SC once weekly, as prescribed by their physician per the product label.
Number of Participants With Clinically Significant Changes in Vital Signs	0 Participants

PRIMARY outcome

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

Population: The evaluable set included participants in the safety set having at least 1 post-dose assessment.

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.

Outcome measures

Outcome measures
Measure	TEGSEDI n=7 Participants Participants received TEGSEDI 284 mg, SC once weekly, as prescribed by their physician per the product label.
Number of Participants With Clinically Significant Changes in Cytokine Levels and Inflammatory Markers	0 Participants

Adverse Events

TEGSEDI

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	TEGSEDI n=7 participants at risk Participants with hATTR-PN received TEGSEDI 284 mg, SC once weekly, as prescribed by their physician per the product label.
General disorders Chills	14.3% 1/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.
General disorders Influenza like illness	14.3% 1/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.
General disorders Injection site discomfort	14.3% 1/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.
General disorders Injection site pain	28.6% 2/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.
Musculoskeletal and connective tissue disorders Myalgia	14.3% 1/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.
Musculoskeletal and connective tissue disorders Pain in extremity	14.3% 1/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.
Gastrointestinal disorders Diarrhoea	14.3% 1/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.
Gastrointestinal disorders Nausea	14.3% 1/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.
Metabolism and nutrition disorders Decreased appetite	14.3% 1/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.
Nervous system disorders Dizziness	14.3% 1/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.
Skin and subcutaneous tissue disorders Hyperhidrosis	14.3% 1/7 • Up to 2 years (24 hours post each TEGSEDI injection) The evaluable set included participants in the safety set having at least 1 post-dose assessment.

Additional Information

Ionis Pharmaceuticals, Inc.

Phone: 760-603-2346

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60