Cure My Disease

Don't miss your cure!

 Log in / Sign up

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

NCT ID NCT04850105

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

First seen Jun 26, 2026 · Last updated Jun 26, 2026

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for HEREDITARY TRANSTHYRETIN AMYLOIDOSIS WITH POLYNEUROPTHY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Study Center

    RECRUITING

    Lille, Cedex, 59037, France

  • Study Center

    RECRUITING

    Nice, Romaine, 06001, France

  • Study Center

    RECRUITING

    Heidelberg, 69120, Germany

  • Study Center

    RECRUITING

    Athens, 115 28, Greece

  • Study Center

    RECRUITING

    Athens, 11528, Greece

  • Study Center

    RECRUITING

    Heraklion, 71500, Greece

  • Study Center

    RECRUITING

    Bologna, 40139, Italy

  • Study Center

    RECRUITING

    Genova, 16132, Italy

  • Study Center

    RECRUITING

    Messina, 98125, Italy

  • Study Center

    RECRUITING

    Milan, 20133, Italy

  • Study Center

    RECRUITING

    Naples, 80131, Italy

  • Study Center

    RECRUITING

    Pavia, 27100, Italy

  • Study Center

    RECRUITING

    Roma, 00133, Italy

  • Study Center

    RECRUITING

    Roma, 00168, Italy

  • Study Center

    RECRUITING

    Lisbon, 1649 035, Portugal

  • Study Center

    RECRUITING

    Huelva, Andalusia, 21005, Spain

  • Study Center

    RECRUITING

    Palma de Mallorca, Balearic Islands, 07198, Spain

  • Study Center

    RECRUITING

    Madrid, 28041, Spain

  • Study Centre

    RECRUITING

    Sofia, 1431, Bulgaria

  • Study Centre

    RECRUITING

    Égkomi, Nicosia, 2371, Cyprus

  • Study Centre

    RECRUITING

    Nantes, 44093, France

  • Study Centre

    RECRUITING

    Roma, Rome, 00189, Italy

  • Study Centre

    RECRUITING

    Oviedo, Avenida de Roma, 33011, Spain

  • Study Centre

    RECRUITING

    Villarreal, Barcelona, 08036, Spain

  • Study Centre

    RECRUITING

    Barcelona, Catalonia, 08035, Spain

  • Study Centre

    RECRUITING

    Madrid, Madrid, 28040, Spain