New drug shows promise for rare fat disorder in extended trial

NCT ID NCT05130450

Ongoing Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

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.

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.

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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

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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