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New study tracks Real-World safety of TEGSEDI for rare nerve disease

NCT ID NCT04850105

Knowledge-focused Sponsor: Akcea Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jul 30, 2026 · Updated 2 times

Summary

This study follows 240 patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) to monitor the long-term safety of the drug TEGSEDI. Researchers will compare patients who have taken TEGSEDI with those who have not, focusing on side effects like low platelet counts. No extra tests or visits are required—data is collected from routine care.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
TEGSEDI (inotersen)
What this could lead to
If successful, this study could provide a clearer picture of the long-term safety of TEGSEDI, helping doctors manage risks like low platelet counts in patients with hATTR-PN.
What could go wrong
This is an observational study, not a treatment trial, so it won't test if TEGSEDI works. It only collects data from routine care, which may have gaps or inconsistencies.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

202 people

The number who actually took part.

Started

Sep 2021

Expected to finish

Mar 2036

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

The TEGSEDI-exposed cohort will consist of patients diagnosed with hATTR-PN who are receiving TEGSEDI. Data from the TEGSEDI-exposed cohort will be compared to data collected prospectively from a TEGSEDI-unexposed cohort which will consist of patients diagnosed with hATTR-PN who have not taken any dose of TEGSEDI within 25 weeks prior to enrollment and are eligible for TEGSEDI treatment per applicable product label. Patients in Europe, US, and Canada will be enrolled from centers that manage patients with hATTR-PN.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Either: 1. TEGSEDI Exposed Cohort: Patients diagnosed with hATTR-PN who have taken any dose of TEGSEDI within 25 weeks prior to enrollment 2. TEGSEDI Unexposed Cohort: Patients diagnosed with hATTR-PN who have not taken any dose of TEGSEDI within 25 weeks prior to enrollment and are eligible for TEGSEDI treatment per applicable product label. Patients may take other drugs to treat hATTR-PN. 2. Clinically managed in Canada, Europe, or the US 3. Have provided appropriate written informed consent Exclusion Criteria: * None

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Conditions

The condition(s) this trial relates to.

amyloidosis familial amyloid neuropathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hereditary transthyretin amyloidosis with polyneuropthy

Contacts and locations

Locations

  • Study Center

    Lille, Cedex, 59037, France

  • Study Center

    Nice, Romaine, 06001, France

  • Study Center

    Heidelberg, 69120, Germany

  • Study Center

    Athens, 115 28, Greece

  • Study Center

    Athens, 11528, Greece

  • Study Center

    Heraklion, 71500, Greece

  • Study Center

    Bologna, 40139, Italy

  • Study Center

    Genova, 16132, Italy

  • Study Center

    Messina, 98125, Italy

  • Study Center

    Milan, 20133, Italy

  • Study Center

    Naples, 80131, Italy

  • Study Center

    Pavia, 27100, Italy

  • Study Center

    Roma, 00133, Italy

  • Study Center

    Roma, 00168, Italy

  • Study Center

    Lisbon, 1649 035, Portugal

  • Study Center

    Huelva, Andalusia, 21005, Spain

  • Study Center

    Palma de Mallorca, Balearic Islands, 07198, Spain

  • Study Center

    Madrid, 28041, Spain

  • Study Centre

    Sofia, 1431, Bulgaria

  • Study Centre

    Égkomi, Nicosia, 2371, Cyprus

  • Study Centre

    Nantes, 44093, France

  • Study Centre

    Roma, Rome, 00189, Italy

  • Study Centre

    Oviedo, Avenida de Roma, 33011, Spain

  • Study Centre

    Villarreal, Barcelona, 08036, Spain

  • Study Centre

    Barcelona, Catalonia, 08035, Spain

  • Study Centre

    Madrid, Madrid, 28040, Spain