A Phase 4 interventional study of TEGSEDI in Hereditary Transthyretin Amyloidosis With Polyneuropthy, sponsored by Akcea Therapeutics. Terminated at 2 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-04-10.
Sponsored by Akcea Therapeutics · Phase 4, Interventional, and Other
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.
Satisfy one of the following:
Satisfy one of the following:
Exclusion Criteria:
Participants received TEGSEDI 284 milligrams (mg), subcutaneously (SC) once weekly, as prescribed by their physician per the product label.
Drug: TEGSEDI
SC injection.
Also known as: Inotersen
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)
Participants took part at 2 clinical sites in the United States of America (USA) and Canada from 21 January 2021 to 20 March 2024.
| Milestone | TEGSEDI |
|---|---|
| Started | 8 |
| Completed | 6 |
| Not completed | 2 |
| Withdrew: Voluntarily withdrawal | 1 |
| Withdrew: Enrolled but not treated | 1 |
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.
| Participants | TEGSEDI |
|---|---|
| Number of Participants With Incident, Onset and Duration of Treatment Emergent Adverse Events (TEAEs) Occurring Within 24 Hours of Each TEGSEDI Administration | 3 |
Vital signs including body temperature, heart rate, respiratory rate, and systolic/diastolic blood pressure (BP).
| Participants | TEGSEDI |
|---|---|
| Number of Participants With Clinically Significant Changes in Vital Signs | 0 |
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.
| Participants | TEGSEDI |
|---|---|
| Number of Participants With Clinically Significant Changes in Cytokine Levels and Inflammatory Markers | 0 |
Collected over Up to 2 years (24 hours post each TEGSEDI injection). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| TEGSEDI | 0/7 (0%) | 0/7 (0%) | 6/7 (85.7%) |
| Event | TEGSEDI |
|---|---|
| Injection site painGeneral disorders | 2/7 |
| ChillsGeneral disorders | 1/7 |
| Influenza like illnessGeneral disorders | 1/7 |
| Injection site discomfortGeneral disorders | 1/7 |
| MyalgiaMusculoskeletal and connective tissue disorders | 1/7 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 1/7 |
| DiarrhoeaGastrointestinal disorders | 1/7 |
| NauseaGastrointestinal disorders | 1/7 |
| Decreased appetiteMetabolism and nutrition disorders | 1/7 |
| DizzinessNervous system disorders | 1/7 |
The safety set included all participants who received at least 1 dose of TEGSEDI while enrolled in this study protocol.
| Age, Continuous(years) | TEGSEDI |
|---|---|
| Mean | 67.6 ± 8.62 |
| Sex: Female, Male(Participants) | TEGSEDI |
|---|---|
| Female | 4 |
| Male | 3 |
| Ethnicity (NIH/OMB)(Participants) | TEGSEDI |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 6 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | TEGSEDI |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 2 |
| Black or African American | 4 |
| White | 1 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
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Amyloid Neuropathies, Familial→
Akcea Therapeutics