Hereditary Transthyretin Amyloidosis With PolyneuropthyPhase 4NCT04306510
A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PN
A Phase 4 study testing TEGSEDI in people with Hereditary Transthyretin Amyloidosis With Polyneuropthy, run by Akcea Therapeutics at 2 sites in 2 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Transthyretin Amyloidosis With Polyneuropthy
- What is being tested?
- TEGSEDI
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 21 Jan 2021to 20 Mar 2024
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Show all 10Show fewer
- 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
Who cannot join
- None
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Phase 4 Safety Study Assessing the Adverse Events Occurring Within One Day of TEGSEDI Administration in Patients With Polyneuropathy of Hereditary Transthyretin-mediated Amyloidosis (hATTR-PN)
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.
- Being tested
- TEGSEDI
- Keywords
- Hereditary Transthyretin Amyloidosis, hATTR-PN, hATTR, Amyloidosis
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TEGSEDIDrug
Also called Inotersen
SC injection.
| Study group | Role | What they receive |
|---|---|---|
| TEGSEDI | Experimental | Participants received TEGSEDI 284 milligrams (mg), subcutaneously (SC) once weekly, as prescribed by their physician per the product label. Receives: Drug: TEGSEDI |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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).
Measured: 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.
Measured: Up to 2 years (24 hours post each TEGSEDI injection)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2020
- Study start21 Jan 2021
- Primary completion20 Mar 2024
- Study completion20 Mar 2024
Why it stopped: Withdrawal of TEGSEDI from sale was not related to any safety issues.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hereditary Transthyretin Amyloidosis With PolyneuropthyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Akcea Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04306510
- Sponsor's protocol ID
- TEG4004
- Status last verified
- Mar 2025
- First submitted
- 7 Feb 2020
- Last updated
- 10 Apr 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 4 Safety Study Assessing the Adverse Events Occurring Within One Day of TEGSEDI Administration in Patients With Polyneuropathy of Hereditary Transthyretin-mediated Amyloidosis (hATTR-PN)
- ClinicalTrials.gov ID
- NCT04306510
- Acronym / sponsor ID
- TEG4004
- Keywords
- Hereditary Transthyretin Amyloidosis, hATTR-PN, hATTR, Amyloidosis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Withdrawal of TEGSEDI from sale was not related to any safety issues.
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Akcea TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 21 Jan 2021 (actual)
- Primary completion
- 20 Mar 2024 (actual)
- Study completion
- 20 Mar 2024 (actual)
- First posted
- 13 Mar 2020 (actual)
- Results
- Posted 10 Apr 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TEGSEDI | Experimental | Drug: TEGSEDIParticipants received TEGSEDI 284 milligrams (mg), subcutaneously (SC) once weekly, as prescribed by their physician per the product label. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Satisfy one of the following:
- 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.
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TEGSEDIAlso: InotersenSC injection. | Drug | TEGSEDI |
Primary outcome
Number of Participants With Incident, Onset and Duration of Treatment Emergent Adverse Events (TEAEs) Occurring Within 24 Hours of Each TEGSEDI Administration
Time frame: Up to 2 years (24 hours post each TEGSEDI injection)
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.
Number of Participants With Clinically Significant Changes in Vital Signs
Time frame: Up to 2 years (24 hours post each TEGSEDI injection)
Vital signs including body temperature, heart rate, respiratory rate, and systolic/diastolic blood pressure (BP).
Number of Participants With Clinically Significant Changes in Cytokine Levels and Inflammatory Markers
Time frame: Up to 2 years (24 hours post each TEGSEDI injection)
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Hereditary Transthyretin Amyloidosis With Polyneuropthy
Active, not recruitingObservational study
A Non-interventional Cohort Safety Study of Patients With hATTR-PN
Hereditary Transthyretin Amyloidosis With Polyneuropthy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing TEGSEDI in people with Hereditary Transthyretin Amyloidosis With Polyneuropthy, run by Akcea Therapeutics at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 10 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Incident, Onset and Duration of Treatment Emergent Adverse Events (TEAEs) Occurring Within 24 Hours of Each TEGSEDI Administration (time frame: Up to 2 years (24 hours post each TEGSEDI injection)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Apr 2025.