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

NCT ID NCT04850105

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

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.

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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