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New drug shows promise for rare fat disorder in extended trial

NCT ID NCT05130450

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 1 time

Summary

This study is for people with familial chylomicronemia syndrome (FCS), a rare condition causing extremely high blood fats. It tests a drug called olezarsen, given as a shot under the skin, to see if it can lower triglycerides over months to years. Participants are 60 patients who already completed a prior olezarsen study. The goal is to check long-term safety and effectiveness.

What this could mean

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

Active substance
olezarsen (a drug injected under the skin)
What this could lead to
If it works, this could provide a long-term treatment option to control dangerously high fat levels in the blood for people with FCS.
What could go wrong
This is an extension study with no placebo group, so results may be less reliable. The drug may not work for everyone, and side effects are still being monitored.

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

60 people

The number who actually took part.

Started

Nov 2021

Expected to finish

Feb 2028

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: • Satisfactory completion of treatment with olezarsen in the index study (ISIS 678354-CS3, last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement. Exclusion Criteria: • Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the patient participating in or completing the study, including need for treatment with medications disallowed in the index study (ISIS 678354-CS3).

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Conditions

The condition(s) this trial relates to.

familial chylomicronemia syndrome Familial hyperchylomicronemia syndrome

As listed by the trial registrant

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

Familial chylomicronemia syndrome

Contacts and locations

Locations

  • Academisch Medisch Centrum Goederenontvangst Apotheek t.a.v. Kenniscentrum

    Amsterdam, Noor-Holland, 1105 AZ, Netherlands

  • Apokteket AB

    Malmö, 211 24, Sweden

  • Azienda Ospedaliero Universitaria Policlinico Umberto I

    Rome, 00161, Italy

  • CTSI Investigational Pharmacy

    New York, New York, 10016, United States

  • Clinique des Maladies Lipidiques de Quebec Inc.

    Québec, Quebec, G1V 4W2, Canada

  • Department of Pharmacy

    Park Ridge, Illinois, 60068, United States

  • Diabetes/Lipid Management & Research Center

    Huntington Beach, California, 92648, United States

  • Dra Margarida Falcao Centro Hospitalar Lisboa Ocidental Hospital Egas Moniz

    Lisbon, 1340-019, Portugal

  • Ecogene-21

    Chicoutimi, Quebec, G7H 7K9, Canada

  • Excel Medical Clinical Trials, LLC

    Boca Raton, Florida, 33434, United States

  • Groupement Hospitalier Est- Pharmacie Secteur Essais Clinique

    Bron, 69677, France

  • Hospital Clínic Barcelona C/ Villarroel

    Barcelona, 08036, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

  • Hospital Universitario Virgen del Rocío

    Seville, 41013, Spain

  • Hospital da Senhora da Oliveira Guimaraes

    Creixomil, 4835-044, Portugal

  • Hôpital Bicêtre

    Le Kremlin-Bicêtre, 94270, France

  • IDS Central

    Philadelphia, Pennsylvania, 19104, United States

  • Institute de Recherches Cliniques de Montreal

    Montreal, Quebec, H2W 1R7, Canada

  • Metabolicke centrum MU

    Bratislava, 83101, Slovakia

  • Milstein Hospital

    New York, New York, 10032, United States

  • Nathalie Saint-Pierre

    Montreal, Quebec, H2W 1R7, Canada

  • Oslo Hospital Pharmacy Rikshospitalet

    Oslo, 0372, Norway

  • Pharmacie Hopital de la Conception

    Marseille, 05 13385, France

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL, United Kingdom

  • UOC di Farmacia AOUP PAOLO GIACCONE

    Palermo, 90127, Italy

  • University of Kansas Medical Center (KUMC)

    Kansas City, Kansas, 66160, United States

  • Via Sergio Pansini 5

    Naples, 80131, Italy

More trials for these conditions

Other studies related to the condition(s) this trial covers.