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New drug shows promise for Long-Term control of rare nerve disease

NCT ID NCT05071300

Disease control Sponsor: Ionis Pharmaceuticals, Inc. 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study looks at the long-term safety and effects of a drug called eplontersen in people with a rare inherited nerve disease called hATTR-PN. About 151 participants who completed earlier studies will receive regular injections of eplontersen. The goal is to see if the drug remains safe over time and helps control nerve damage.

What this could mean

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

Active substance
Eplontersen (a drug given as a shot under the skin)
What this could lead to
If successful, this could confirm eplontersen as a safe long-term treatment option to slow nerve damage in people with hATTR-PN.
What could go wrong
This is an extension study, so participants already know the drug is generally safe, but long-term risks (like effects on platelets, kidneys, or liver) are still being checked. The trial is not designed to prove a cure, only to manage the disease.

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.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

151 people

The number who actually took part.

Started

Jan 2022

Expected to finish

Aug 2029

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.

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. Satisfactory completion of ION-682884-CS3 (NCT04136184) (Index Study) as judged by the Investigator and Sponsor, or diagnosis of hATTR-PN and satisfactory completion of either study ISIS 420915-CS101 or study 2018-P001436 (NCT03702829) (both are Investigator-Sponsored studies with inotersen - the unconjugated version of eplontersen) as judged by the Investigator and Sponsor. 2. Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements. 3. Satisfy the following: 1. Females: must be non-pregnant and non-lactating and either: * Surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy); * Post-menopausal (defined as 12 months of spontaneous amenorrhea in females \> 55 years of age or, in females ≤ 55 years, 12 months of spontaneous amenorrhea without an alternative medical cause and follicle-stimulating hormone (FSH) levels in the postmenopausal range for the laboratory involved; * Abstinent\*; * If engaged in sexual relations of child-bearing potential, agree to use highly effective contraceptive methods from the time of signing the informed consent form until at least 24 weeks after the last dose of eplontersen and agree to receive pregnancy tests per protocol. 2. Males: Surgically sterile (i.e., bilateral orchidectomy) or abstinent\*, if engaged in sexual relations with a woman of child-bearing potential (WOCBP), the participant or the participant's non-pregnant female partner must use a highly effective contraceptive method from the time of signing the informed consent form until at least 24 weeks after the last dose of eplontersen. \*Abstinence (i.e., refraining from heterosexual intercourse throughout the duration of study participation) is only acceptable as true abstinence, i.e., when this is in line with the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods), declaration of abstinence for the duration of a trial and withdrawal are not acceptable methods of contraception. 4. Willingness to adhere to vitamin A supplementation per protocol. Exclusion Criteria: 1\. Have any new condition or worsening of existing condition that in the opinion of the Investigator or Sponsor would make the participant unsuitable for enrollment or could interfere with the participant taking part in or completing the study.

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Conditions

The condition(s) this trial relates to.

familial amyloid neuropathy

As listed by the trial registrant

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

Hereditary transthyretin-mediated amyloid polyneuropathy

Contacts and locations

Locations

  • Boston University School of Medicine

    Boston, Massachusetts, 02118, United States

  • Centre Hospitalier Universitaire de Toulouse

    Toulouse, Haute-Garonne, 31059, France

  • Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria

    Lisbon, 1649-035, Portugal

  • Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio

    Porto, 4099-001, Portugal

  • Chang Gung Memorial Hospital

    Taoyuan, 333, Taiwan

  • China Medical University Hospital

    Taichung, 40447, Taiwan

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100, Italy

  • Hospital Clínico San Carlos

    Madrid, 28040, Spain

  • Hospital El Cruce

    San Juan Bautista, 1888, Argentina

  • Hospital Italiano de Buenos Aires

    Buenos Aires, C1199ABB, Argentina

  • Hospital Son Llàtzer

    Palma de Mallorca, 07198, Spain

  • Hospital Universitario Clementino Fraga Filho

    Botafogo, Rio de Janeiro, 22281-100, Brazil

  • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

    São Paulo, 14051-140, Brazil

  • Hôpital de la Timone

    Marseille, 13385, France

  • Indiana University Health University Hospital

    Indianapolis, Indiana, 46202, United States

  • Instituto Fleni

    Buenos Aires, C1428 AQK, Argentina

  • Instituto de Neurologia de Curitiba

    Curitiba, 81210-310, Brazil

  • Istanbul Üniversitesi - Istanbul Tip Fakültesi

    Istanbul, 34093, Turkey (Türkiye)

  • Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Fondazione Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Johns Hopkins University Neurology Research Office

    Baltimore, Maryland, 21287, United States

  • Mayo Clinic

    Scottsdale, Arizona, 85259, United States

  • National Taiwan University Hospital

    Taipei, 100, Taiwan

  • Norrlands Universitetssjukhus

    Umeå, 901 85, Sweden

  • Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • Perron Institute for Neurological and Translational Science

    Murdoch, Western Australia, 6150, Australia

  • Taipei Veterans General Hospital

    Taipei, 11217, Taiwan

  • The Cyprus Institute of Neurology and Genetics

    Égkomi, 2371, Cyprus

  • The Neurological Institute of New York

    New York, New York, 10032, United States

  • Toronto General Hospital

    Toronto, Ontario, M5G2C4, Canada

  • Universidade Estadual de Campinas

    Campinas, 13083-888, Brazil

  • University of North Carolina Hospitals - Neurology Clinic

    Chapel Hill, North Carolina, 27514, United States

  • University of Washington Medical Center

    Seattle, Washington, 98195, United States

  • Vancouver General Hospital

    Vancouver, British Columbia, V5Z 1M9, Canada