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 (part of Ionis Pharmaceuticals, Inc.) · 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.
82 studies on the registry are indexed under Amyloidosis, Hereditary, Transthyretin-Related; 28 are open to participants now.
This study's enrollment of 8 is below the median of 80 across 36 interventional studies indexed under Amyloidosis, Hereditary, Transthyretin-Related.
Browse Amyloidosis, Hereditary, Transthyretin-Related studies →Akcea Therapeutics is the lead sponsor of 15 studies on the registry; 1 is open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 8 (89%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Satisfy one of the following:
Satisfy one of the following:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is terminated, as verified in Mar 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Inotersen
Amyloidosis, Hereditary, Transthyretin-Related →